New Drugs 2014

NewAkynzeo to treat nausea and vomiting in patients undergoing cancer chemotherapy.

Akynzeo (netupitant and palonosetrot prevents nausea and vomiting during both the acute and delayed phase after cancer chemotherapy. Oral palonosetron, which was approved in 2008, prevents nausea and vomiting during the acute phase (within first 24 hours) after the start of cancer chemotherapy. Netupitant, a new drug, prevents nausea and vomiting during both the acute phase and delayed phase (from 25 to 120 hours) after the start of cancer chemotherapy. The effectiveness was established in 2 clinical trials of 1,720 patients receiving cancer chemotherapy. Patients were randomly assigned to receive Akynzeo or the single agent palonosetron. The trials were designed to measure whether the study drugs prevented any vomiting episodes in the acute, delayed and n) – Eisai - Akynzeo is indicated for the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of cancer chemotherapy, including, but not limited to, highly emetogenic chemotherapy. Akynzeo is an oral fixed combination of palonosetron and netupitant: palonosetron prevents nausea and vomiting during the acute phase and netupitanoverall phases after the start of cancer chemotherapy. Results of the first trial showed that 98.5 percent, 90.4 percent and 89.6 percent of Akynzeo treated patients did not experience any vomiting or require rescue medication for nausea during the acute, delayed and overall phases, respectively. In contrast, 89.7 percent, 80.1 percent and 76.5 percent of patients treated with oral palonosetron did not experience any vomiting or require rescue medication for nausea during the acute, delayed and overall phases, respectively. The second trial showed similar results. Common side effects of Akynzeo were headache, weakness, fatigue, dyspepsia and constipation. For highly emetogenic chemotherapy, including Cisplatin based chemotherapy, the recommended dosage in adults is one capsule of Akynzeo administered approximately 1 hour prior to the start of chemotherapy with dexamethasone 12 mg administered orally 30 minutes prior to chemotherapy on day 1 and 8 mg orally once daily on days 2 to 4.For chemotherapy not considered as highly emetogenic (anthracyclines and cyclophosphamide based), the recommended dosage is 1 capsule of Akynzeo approximately 1 hour prior to the start of chemotherapy with dexamethasone 12 mg administered orally 30 minutes prior to chemotherapy on day 1. Administration of dexamethasone on days 2 to 4 is not necessary. Akynzeo can be taken with or without food. It is available in 1 white-caramel hard gelatin capsule which contains 3 tablets each containing 100 mg netupitant and 1 gelatin capsule containing 0.56 mg palonosetron hydrochloride (equivalent to 0.50 mg palonosetron).
Approved: October 10, 2014.

Beleodaq to treat a rare, aggressive form of non-Hodgkin lymphoma.

Beleodaq (belinostat) - Spectrum - is approved for the treatment of patients with peripheral T-cell lymphoma (PTCL), a rare and fast-growing type of non-Hodgkin lymphoma (NHL). Beleodaq is a histone deacetylase inhibitor indicated for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma (PTCL). Beleodaq works by stopping enzymes that contribute to T-cells becoming cancerous. Beleodaq was evaluated in a clinical study involving 129 patients with relapsed or refractory PTCL. All patients were treated with Beleodaq until their disease progressed or side effects became intolerable. Results showed 25.8 percent of participants had their cancer disappear or shrink after treatment. In clinical trials, BELEODAQ has shown that it may be well-tolerated, which allows for combination with traditional chemotherapy without causing excessive bone marrow toxicity. The most common side effects were nausea, fatigue, fever, anemia, and vomiting. Beleodaq is supplied in single 30 ml vial containing lyophilized powder equivalent to 500 mg. The recommended dosage of Beleodaq is 1,000 mg/m2 administered over 30 minutes by IV infusion once daily on Days 1-5 of a 21-day cycle. Cycles can be repeated every 21 days until disease progression or unacceptable toxicity.
Approved: July 3, 2014.

NewBelsomra for insomnia.

Belsomra (suvorexant) - Merck - Belsomra has been approved for use as needed to treat insomnia. Belsomra is an orexin receptor antagonist and is the first approved drug of this type. Orexins are chemicals that are involved in regulating the sleep-wake cycle and play a role in keeping people awake. Belsomra alters the action of orexin in the brain. Belsomra is taken no more than once per night, within 30 minutes of going to bed, with at least 7 hours remaining before the planned time of waking. The total maximum dose should not exceed 20 mg once daily. It is available in 5, 10, 15, and 20 milligrams strengths. The most common adverse effect of Belsomra was drowsiness. Clinical tests have shown impaired driving performance in both male and female participants when the 20 mg strength was taken. Patients using the 20 mg strength should be cautioned against next-day driving or activities requiring full mental alertness. The effectiveness of Belsomra was shown in 3 clinical trials with more than 500 patients. The studies showed that patients taking the drug fell asleep faster and spent less time awake during the remainder of the night compared with placebo. Belsomra will be dispensed with an FDA-approved patient Medication Guide that provides instructions for its use and important safety information. Belsomra is a Schedule IV controlled substance.
Approved: August 13, 2014.

Cerdelga for the long-term treatment of adult patients with the Type 1 form of Gaucher disease.

Cerdelga (eliglustat) - Genzyme - Cerdelga has been approved for the long-term treatment of adult patients with the Type 1 form of Gaucher disease, a rare genetic disorder. Gaucher disease occurs in individuals who do not produce enough of the enzyme, glucocerebrosidase. It is a genetic disease in which fatty substances (sphingolipids) accumulate in cells and certain organs. The disorder is characterized by bruising, fatigue, anemia, low blood platelets, and enlargement of the liver and spleen. The symptoms of Gaucher disease includes liver and spleen enlargement, anemia, low platelet counts and bone problems. Cerdelga is available in a hard gelatin capsule containing eliglustat that is taken orally. In patients with Gaucher disease Type 1, the drug slows down the production of the fatty materials by inhibiting the metabolic process that forms them. The safety and effectiveness of Cerdelga were evaluated in 2 clinical trials with 199 participants. Compared with placebo, Cerdelga showed a greater reduction in spleen volume from baseline at week 39 and also resulted in greater improvement in liver volume, blood platelet count, and hemoglobin level. The most commonly adverse effects were fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain.
Approved: August 19, 2014.

Cyramza for treatment of advanced or metastatic gastric or gastroesophageal junction adenocarcinoma.

Cyramza (ramucirumab) - Lilly - Cyramza is a recombinant monoclonal antibody of the IgG1 class that binds to vascular endothelial growth factor receptor-2 (VEGFR2) and blocks the activation of the receptor. It is indicated in the treatment of gastric cancer (advanced gastric cancer or gastro-esophageal junction adenocarcinoma), as a single agent after prior fluoropyrimidine or platinum containing chemotherapy. The approval is based on the improved overall survival in multinational, randomized, double-blind, multicenter study (I4T-IE-JVBD) who enrolled 355 patients with previously treated advanced or metastatic, gastric or GEJ adenocarcinoma. They were randomized to receive Cyramza or placebo. The median overall survival was 5.2 months with Cyramza and 3.8 months with placebo. The most common adverse effects were hypertension and diarrhea and this was at a rate of ≥10% and ≥2% higher than placebo. The most common serious adverse events with Cyramza were intestinal obstruction (2.1%) and anemia (3.8%). Cyramza increased the risk of hemorrhage, including severe and sometimes fatal hemorrhagic events. Permanently discontinue Cyramza in patients who experience severe bleeding. Other important risks included in labeling are hemorrhage, arterial thrombotic events, impaired wound healing, clinical deterioration in patients with cirrhosis, and reversible posterior leukoencephalopathy. The recommended dose and schedule is 8mg/kg administered as a 60 minute IV infusion every 2 weeks.
Approved: April 2, 2014

Dalvance to treat skin infections.

Dalvance (dalbavancin) - Durata Therapeutics - Dalvance is intended to treat acute bacterial skin and skin structure infections (ABSSSI) caused by certain susceptible bacteria like Staphylococcus aureus (including methicillin-susceptible and methicillin-resistant strains) and Streptococcus pyogenes. Dalvance is the first drug designated as a Qualified Infectious Disease Product (QIDP) to receive FDA approval. As part of its QIDP designation, Dalvance was given priority review, which provides an expedited review of the drug’s application. Dalvance’s QIDP designation also qualifies it for an extra 5 years of marketing exclusivity to be added to certain exclusivity periods already provided by the FDA. Dalvance’s safety and efficacy were evaluated in 2 clinical trials with a total of 1,289 adults with ABSSSI. Participants were randomly assigned to receive Dalvance or vancomycin. Results showed Dalvance was as effective as vancomycin for the treatment of ABSSSI. The most common adverse effects were nausea, headache and diarrhea. In the trials, more participants in the Dalvance group had elevations in 1 of their liver enzyme tests. The Dalvance drug label provides recommendations on dosage adjustment in patients with renal impairment. The recommended dose is 1000 mg followed one week later by 500 mg and should be administered over 30 minutes by intravenous infusion. DALVANCE is supplied in 500 mg single-dose vials containing sterile powder for reconstitution.
Approved: May 23, 2014.

Entyvio for ulcerative colitis and crohn’s disease.

Entyvio (vedolizumab) - Takeda - Entyvio is an injection for the treatment of adults with moderate to severe ulcerative colitis and moderate to severe Crohn‘s disease. Entyvio is approved to treat those conditions when 1 or more standard therapies (corticosteroids, immunomodulators, or tumor necrosis factor blockers) have not resulted in an adequate response. The safety and effectiveness of Entyvio for ulcerative colitis were established in 2 clinical trials involving approximately 900 patients who had not responded adequately to 1 or more standard therapies. Evaluations of patients included measures of stool frequency, rectal bleeding, endoscopic findings and a physician‘s overall assessment. Results showed a greater percentage of patients treated with Entyvio compared to a placebo achieved and maintained clinical response, achieved and maintained clinical remission, achieved corticosteroid-free clinical remission. The safety and effectiveness of Entyvio for Crohn‘s disease were established in 3 clinical trials involving approximately 1,500 patients. Results showed a greater percentage of patients treated with Entyvio compared to a placebo achieved clinical response, achieved clinical remission, and achieved corticosteroid-free clinical remission. Entyvio is an integrin receptor antagonist. Integrin receptors are proteins expressed on the surface of certain cells and they function as bridges for cell-cell interactions. Entyvio blocks the interaction of a specific integrin receptor with a specific protein and thereby blocks the migration of those circulating inflammatory cells across those blood vessels and into areas of inflammation in the gastrointestinal tract. The most common side effects in patients treated with Entyvio include headache, joint pain, nausea, and fever. The most serious risks associated with Entyvio include serious infections, hypersensitivity and infusion-related reactions; and hepatotoxicity.
Approved: May 20, 2014

NewEsbriet to treat idiopathic pulmonary fibrosis.

Esbriet (pirfenidone) - InterMune - Esbriet has been approved for the treatment of idiopathic pulmonary fibrosis. Idiopathic pulmonary fibrosis is a condition in which the lungs become progressively scarred over time and as a result, patients with IPF experience shortness of breath, cough, and have difficulty participating in everyday physical activities. Current treatments for IPF include oxygen therapy, pulmonary rehabilitation, and lung transplant. Esbriet acts on multiple pathways that may be involved in the scarring of lung tissue. Its safety and effectiveness were established in 3 clinical trials of 1,247 patients with IPF. The decline in forced vital capacity – the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible – was significantly reduced in patients receiving Esbriet compared to patients receiving placebo. Esbriet is not recommended for patients who have severe liver problems, end-stage kidney disease, or who require dialysis. Esbriet should be taken with food to minimize the potential for nausea and dizziness. Patients should avoid or minimize exposure to sunlight and sunlamps and wear sunscreen and protective clothing, as Esbriet may cause patients to sunburn more easily. The most common side effects are nausea, rash, abdominal pain, upper respiratory tract infection, diarrhea, fatigue, headache, dyspepsia, dizziness, vomiting, decreased/loss of appetite, GERD, sinusitis, insomnia, decreased weight, and arthralgia.
Approved: October 15, 2014.

Farxiga to treat adult type 2 diabetes.

Farxiga (dapaglifozin) - Bristol Meyers - Fargixa is a sodium-glucose cotransporter 2 (SGLT2) inhibitor indicated as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus. SGLT2 inhibitors block the reabsorption of glucose by the kidney, increase glucose excretion, and lower blood glucose levels. The safety and effectiveness were evaluated in 16 clinical trials including more than 9,400 patients with type 2 diabetes. The trials showed improvement in HbA1c which is a measure of blood sugar control. Farxiga can be used alone or in combination with other type 2 diabetic medications. Farxiga should not be used to treat people with type 1 diabetes; those with diabetic ketoacidosis; or those with moderate or severe renal impairment, end stage renal disease, or patients on dialysis. An increased number of bladder cancers were diagnosed among Farxiga users in clinical trials, so Farxiga is not recommended for patients with active bladder cancer. Patients with a history of bladder cancer should talk to their physician before taking Farxiga. Farxiga can cause dehydration, leading to hypotension that can result in dizziness and/or fainting and a decline in renal function. The elderly, patients with impaired renal function, and patients on diuretics to treat other conditions appear to be more susceptible to this risk. Due to these adverse reactions, the FDA is requiring 6 post marketing studies for Farxiga. Farxiga is available in 5mg and 10mg tablets. The recommended starting dose is 5 mg once daily, taken in the morning, with or without food. The dose can be increased to 10 mg once daily in patients tolerating Farxiga who need additional glycemic control. Renal function needs to be assessed before therapy with Farxiga is initiated. Do not initiate Farxiga if eGFR is below 60 mL/min and discontinue use if eGFR falls persistently below 60. The most common side effects were female genital mycotic infections, nasopharyngitis, and urinary tract infections.

NewHarvoni to treat chronic hepatitis C virus (HCV) genotype 1 infection.

Harvoni (ledipasvir and sofosbuvir) - Gilead - Harvoni is the first combination pill approved to treat chronic hepatitis C virus genotype 1 infection. It is also the first approved regimen that does not require administration with interferon or ribavirin, 2 FDA-approved drugs also used to treat HCV infection. Both drugs in Harvoni interfere with the enzymes needed by HCV to multiply. Sofosbuvir has been previously approved under the brand name Sovaldi. Harvoni also contains a new drug called ledipasvir. Harvoni is a two-drug fixed-dose combination product that contains 90 mg of ledipasvir and 400 mg of sofosbuvir in a single tablet. The recommended dosage of HARVONI is one tablet taken orally once daily with or without food. The efficacy was evaluated in 3 clinical trials with 1,518 patients who had not previously received treatment for their infection or had not responded to previous treatment, including patients with cirrhosis. Patients were randomly assigned to receive Harvoni with or without ribavirin. The trials were designed to measure whether the hepatitis C virus was no longer detected in the blood at least 12 weeks after finishing treatment (sustained virologic response, or SVR), indicating that a patient's HCV infection has been cured. The most common side effects reported in clinical trial patients were fatigue and headache.
Approved: October 10, 2014.

 

Hetlioz to treat 24-hour sleep-wake disorder in totally blind individuals.

Hetlioz (tasimelteon) - Vanda - Hetlioz, a melatonin receptor agonist, has been approved to treat non-24- hour sleep-wake disorder (“non-24”) in totally blind individuals. This is the first FDA approved medication for the disorder. Non-24 is a chronic circadian rhythm (body clock) disorder in the blind that causes problems with the timing of sleep. Light does not enter their eyes and they cannot synchronize their body clock to the 24-hour light-dark cycle. Those with the disorder may have difficulty falling asleep or staying asleep, and may wake up groggy or feeling as if they need more rest. People with non-24 may find their sleep patterns reversed, therefore needing to sleep during the day and to be awake at night. The effectiveness of Hetlioz was evaluated in 104 participants in 2 clinical trials of totally blind individuals with non-24 disorder. The trials showed treatment with Hetlioz resulted in significant improvement compared to placebo, both in increasing nighttime sleep and decreasing daytime sleep duration. The dose is 20 mg prior to bedtime at same time every night. It is available as a 20mg capsule and should be taken without food. After taking Hetlioz, patients should limit their activity to preparing for going to bed, because Hetlioz can impair the performance of activities requiring complete mental alertness. The most common side effects were headache, increased alanine aminotransferase, nightmares or unusual dreams, and upper respiratory or urinary tract infection. Avoid use of Hetlioz in combination with strong CYP1A2 inhibitors because of increased exposure and strong CYP3A4 inducers because of decreased exposure.
Approved: January 31, 2014

Impavido to treat leishmaniasis.

Impavido (miltefosine) - Paladin - Impavido (miltefosine) is used to treat a tropical disease called leishmaniasis. Leishmaniasis is a disease caused by a parasite, Leishmania, which is transmitted to humans through sand fly bites. The disease occurs predominantly in people who live in the tropics and subtropics. Most U.S. patients acquire leishmaniasis while overseas. Impavido is an oral medicine approved to treat the 3 main types of leishmaniasis: visceral (affects internal organs), cutaneous (affects the skin) and mucosal (affects the nose and throat) in patients 12 years of age and older. Impavido is the first FDA approved drug to treat cutaneous or mucosal leishmaniasis, therefore the FDA granted Paladin with fast track designation, priority review, and orphan product designation. Impavido’s safety and efficacy were evaluated in 4 clinical trials with a total of 547 patients receiving Impavido and 183 patients receiving either a comparator drug or a placebo. Results from these trials demonstrated that Impavido is safe and effective in treating visceral, cutaneous and mucosal leishmaniasis. The labeling for Impavido contains a boxed warning to alert patients and health care professionals that the drug can cause fetal harm and therefore should not be given to pregnant women. Health care professionals should advise women to use effective contraception during and for five months after Impavido therapy. The most common side effects in clinical trials were nausea, vomiting, diarrhea, headache, decreased appetite, dizziness, abdominal pain, itching, drowsiness and elevated levels of liver enzymes (transaminases) and creatinine. Impavido is available as a 50 mg capsule and should be given with food to improve GI side effects. For 30 to 44 kg give 50 mg twice daily for 28 days and for 45 kg or > give 50 mg 3 times daily for 28 days.
Approved: March 19, 2014

NewJardiance to improve gylcemic control in adults with type 2 diabetes.

Jardiance (empagliflozin) - Boehringer - Jardiance is approved as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes. Jardiance is a sodium glucose co-transporter 2 (SGLT2) inhibitor. It works by blocking the reabsorption of glucose by the kidney, increasing glucose excretion, and lowering blood glucose levels in diabetics who have elevated blood glucose levels. The safety and effectiveness were evaluated in 7 clinical trials with 4,480 patients and they showed that Jardiance improved hemoglobin A1C levels as compared with placebo. Jardiance has been studied as mono-therapy and in combination with other type 2 diabetes therapies including metformin, sulfonylureas, pioglitazone, and insulin. Jardiance can cause dehydration, leading to hypotension that can result in dizziness and/or fainting and a decline in renal function. The most common side effects are urinary tract infections and female genital infections.
Approved: August 1, 2014.

Jublia to treat fungal infection.

Jublia (efinaconazole) - Valeant - Jublia is a 10% topical solution for the treatment of onychomycosis of the toenails due to Trichophyton rubrum and Trichophyton mentagrophytes. Apply Jublia to the affected toenails once daily for 48 weeks using the integrated flow-through brush applicator. When applying make sure the toenail, the toenail folds, toenail bed, hyponychium, and the undersurface of the toenail plate, are completely covered. The most common adverse effects were application site reaction and ingrown toenails. It is available in a 4 ml and 8 ml 10 % topical solution containing 100 mg of efinaconazole in each gram of clear, colorless to pale yellow solution.
Approved: June 6, 2014.

Kerydin to treat onychomycosis of the toenails.

Kerydin (tababorole) - Anacor - is an oxaborole antifungal indicated for the topical treatment of onychomycosis of the toenails due to Trichophyton rubrum or Trichophyton mentagrophytes. The mechanism of action is inhibition of fungal protein synthesis. Tavaborole inhibits protein synthesis by inhibition of an aminoacyl-transfer ribonucleic acid (tRNA) synthetase (AARS). KERYDIN should be applied to the entire toenail surface and under the tip of each affected toenail for 48 weeks. KERYDIN may cause irritation at the treatment site. The most common side effects include skin peeling, ingrown toenail, redness, itching, and swelling. Kerydin is a 5% clear topical solution that is supplied in a 12-mL amber glass bottle with 10 mls of medication.
Approved: July 7, 2014.

NewKeytruda for treatment of advanced or unresectable melanoma who are no longer responding to other drugs.

Keytruda (pembrolizumab) - Merck - Keytruda is the first approved drug that blocks a cellular pathway known as PD-1, which restricts the body's immune system from attacking melanoma cells. Keytruda is intended for use following treatment with ipilimumab, a type of immunotherapy. For melanoma patients whose tumors express a gene mutation called BRAF V600, Keytruda is intended for use after treatment with ipilimumab and a BRAF inhibitor, a therapy that blocks activity of BRAF gene mutations. The efficacy was shown in 173 clinical trial patients with advanced melanoma whose disease progressed after prior treatment. All participants were treated with Keytruda, either at the recommended dose of 2 mg/kg or at a higher dose of 10 mg/kg. In the half of the participants who received Keytruda at the recommended dose of 2 mg/kg, approximately 24 percent had their tumors shrink. This effect lasted at least 1.4 to 8.5 months and continued beyond this period in most patients. The most common side effects of Keytruda were fatigue, cough, nausea, pruritus, rash, decreased appetite, constipation, arthralgia, and diarrhea. Keytruda also has the potential for severe immune-mediated side effects. In the 411 participants with advanced melanoma, severe immune-mediated side effects involving healthy organs, including the lung, colon, hormone-producing glands and liver, occurred uncommonly. Keytruda should be administered by intravenous infusion 2 mg/kg over 30 minutes every 3 weeks. Keytruda is supplied in a carton containing one 50 mg single-use vials with sterile lyophilized powder. Vials should be stored at 2°C to 8°C (36°F to 46°F).
Approved: September 4, 2014.

NewMovantik for opioid-induced constipation.

Movantik (naloxegol) - Astrazeneca - The FDA approved Movantik for the treatment of opioid-induced constipation in adults with chronic non-cancer pain. Movantik belongs to a class of drugs called peripherally acting opioid receptor antagonists, which are used to decrease the constipating effects of opioids. Movantik's safety and effectiveness were established in 2 clinical trials of 1,352 participants who had taken opioids for at least four weeks. Results of the first trial showed that 44 % of participants receiving 25 mg of Movantik and 41 % of participants receiving 12.5 mg of Movantik experienced an increase in bowel movements per week, compared to 29 % of participants receiving placebo. The 2nd trial showed similar results. Common side effects of Movantik include abdominal pain, diarrhea, headache and the experience of excessive gas in the stomach or intestinal area. The FDA is requiring a post marketing study to further evaluate the potential risk of cardiovascular adverse events in patients taking Movantik. Movantik is currently a schedule II controlled substance because it is structurally related to noroxymorphone. During the review of the New Drug Application, the FDA evaluated the abuse potential of MOVANTIK and the approved labeling indicates that it has no risk of abuse or dependency. It is available in 12.5 and 25 mg tablets.
Approved: September 16, 2014.

Myalept to treat complications of leptin deficiency.

Myalept (metreleptin) - Amylin - Myalept is a leptin analog used as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy. Lipodystrophy is a condition associated with the lack of fat tissue. Patients with congenital lipodystrophy are born with little or no fat tissue and they generally lose fat tissue over a period of time. Because the hormone leptin is made by fat tissue, patients with lipodystrophy have very low leptin levels. Leptin regulates food intake and other hormones, such as insulin. These patients often develop severe insulin resistance at a young age and may have diabetes mellitus that is difficult to control or hypertriglyceridemia that can lead to inflammation of the pancreas. The safety and effectiveness of Myalept was evaluated in an open-label, single-arm study that included 48 patients with congenital or acquired generalized lipodystrophy who also had diabetes mellitus, hypertriglyceridemia, and/or elevated levels of fasting insulin. The trial showed reductions in HbA1c, fasting glucose, and triglycerides. Myalept is available as an injection used once daily after reconstitution. Because of the risks associated with the development of neutralizing antibodies and lymphoma, Myalept is available only through the Myalept Risk Evaluation and Mitigation Strategy (REMS) Program. Under this REMS program, prescribers must be certified and complete training. Pharmacies must be certified also and only dispense Myalept after receipt of the Myalept REMS Prescription Authorization Form for each new prescription. Myalept is contraindicated in patients with general obesity. Myalept is not approved for patients with HIV-related lipodystrophy or in patients with metabolic disease, including diabetes mellitus and hypertriglyceridemia, without concurrent evidence of generalized lipodystrophy. The recommended daily dose is based on body weight: <= 40 kg start at 0.06 mg/kg/day and increase or decrease by 0.02 mg/kg to maximum daily dose of 0.13 mg/kg. Males > 40 kg body weight start at a dose of 2.5 mg/day and increase or decrease by 1.25 mg to 2.5 mg/day to a maximum dose of 10 mg/day. Females > than 40 kg body weight start at a dose of 5 mg/day and increase or decrease by 1.25 mg to 2.5 mg/day to a maximum dose of 10 mg/day. The most common side effects include low blood sugar, headache, decreased weight, and abdominal pain.
Approved: February 24, 2014

Neuraceq used for PET imaging of the brain.

Neuraceq (florbetaben F 18) - Piramal Imaging - Neuraceq is a radioactive diagnostic agent indicated for Positron Emission Tomography (PET) imaging of the brain to estimate β-amyloid neuritic plaque density in adult patients with cognitive impairment who are being evaluated for Alzheimer’s Disease (AD) and other causes of cognitive decline. Following intravenous administration, Neuraceq crosses the blood brain barrier and shows differential retention in brain regions that contain β-amyloid deposits. Differences in signal intensity between brain regions showing specific and nonspecific Neuraceq uptake form the basis for the image interpretation method. A negative Neuraceq scan indicates sparse to no neuritic plaques and is inconsistent with a neuropathological diagnosis of AD at the time of image acquisition; a negative scan result reduces the likelihood that a patient’s cognitive impairment is due to AD. A positive Neuraceq scan indicates moderate to frequent amyloid neuritic plaques; neuropathological examination has shown this amount of amyloid neuritic plaque is present in patients with AD, but may also be present in patients with other types of neurologic conditions as well as older people with normal cognition. Neuraceq is used in adjunct to other diagnostic evaluations. Neuraceq is supplied in a 30 mL glass vial containing up to 30 mL of clear solution at a strength of 50 to 5000 MBq/mL florbetaben F18 at EOS. Each vial contains multiple doses and is enclosed in a shielded container to minimize external radiation exposure. No serious adverse reactions related to Neuraceq administration have been reported. The most common side effects were injection site reactions consisting of erythema, irritation and pain. All adverse reactions were mild to moderate in severity and of short duration.
Approved: March 19, 2014

Northera to treat neurogenic orthostatic hypotension.

Northera (droxidopa) - Chelsea Therapeutics - Northera has been approved for the treatment of neurogenic orthostatic hypotension. NOH is a rare, chronic and often debilitating drop in blood pressure that occurs upon standing and is associated with Parkinson's disease, multiple-system atrophy, and pure autonomic failure. The FDA has approved Northera under the accelerated approval program which provides patient access to new drugs while the company conducts post-approval clinical trials to prove the drug’s clinical benefit. Northera also received orphan-product designation from the FDA because it is intended to treat a rare disease or condition. The effectiveness of Northera was shown in 2 clinical trials in patients with NOH through a 2 week period. Patients taking Northera reported a decrease in dizziness, lightheadedness, feeling faint, or feeling as if they might black out as compared with those taking placebo. Improvement in patient symptoms beyond two weeks has not been demonstrated. Northera is available in a 100mg, 200mg, and 300mg capsule. The starting dose is 100 mg 3 times daily to titrate by 100 mg 3 times daily, up to a maximum dose of 600 mg 3 times daily. Northera can be taken with or without food. Northera has a boxed warning to alert health care professionals and patients about the risk of supine hypertension, a common problem that affects people with primary autonomic failure and can cause stroke. It is essential that patients be reminded that they must sleep with their head and upper body elevated. Supine blood pressure should be monitored prior to and during treatment and more frequently when increasing doses. The most common side effects include headache, dizziness, nausea, hypertension and fatigue.
Approved: February 18, 2014

NewOfev to treat idiopathic pulmonary fibrosis.

Ofev (nintedanib) - Boehringer - Ofev was also approved for the treatment of idiopathic pulmonary fibrosis (IPF). Ofev is a kinase inhibitor that blocks multiple pathways that may be involved in the scarring of lung tissue. Its safety and effectiveness were established in 3 clinical trials of 1,231 patients with IPF. The decline in forced vital capacity – the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible – was significantly reduced in patients receiving Ofev compared to patients receiving placebo. Ofev is not recommended for patients who have moderate to severe liver problems. Ofev can cause birth defects or death to an unborn baby, so women should not become pregnant while taking Ofev. Women who are able to get pregnant should use adequate contraception during and for at least 3 months after the last dose of Ofev. The most common side effects are diarrhea, nausea, abdominal pain, vomiting, liver enzyme elevation, decreased appetite, headache, decreased weight, and high blood pressure.
Approved: October 15, 2014.

NewOrbactiv for treatment of ABSSSI.

Orbactiv (oritavancin) - Medicines Company - Orbactiv for injection is indicated for the treatment of adult patients with acute bacterial skin and skin structure infections (ABSSSI) caused by susceptible isolates of the following Gram-positive microorganisms. This includes Staphylococcus aureus (including methicillin-susceptible and methicillin-resistant strains), various Streptococcus species and Enterococcus faecalis. Orbactiv is the 1st and only antibiotic approved to treat ABSSSIs with a single, once-only administration infused over 3 hours. Orbactiv is also the 3rd new drug designated as a Qualified Infectious Disease Product (QIDP) to receive FDA approval. The safety and efficacy were evaluated in 2 clinical trials with a total of 1,987 adults with ABSSSI. Participants were randomly assigned to receive Orbactiv or vancomycin and the results showed Orbactiv was as effective as vancomycin for the treatment of ABSSSI. The most common side effects identified in the clinical trials were headache, nausea, vomiting, the formation of skin and soft tissue abscesses on arms and legs and diarrhea. Orbactiv's label also includes a warning regarding interference with coagulation tests and a drug interaction with warfarin.
Approved: August 6, 2014.

Otezla to treat psoriatic arthritis.

Otezla (apremilast) - Celgene - Otezla is an inhibitor of phosphodieasterase-4 and it is approved to treat adults with active psoriatic arthritis. The safety and effectiveness of Otezla were evaluated in 3 clinical trials involving almost 1500 patients with active psoriatic arthritis. Patients treated with Otezla showed improvement in their signs and symptoms, including tender and swollen joints and physical function, as compared to placebo. Patients treated with Otezla should be weighed by their physician regularly, and if there is unexplained or clinically significant weight loss the discontinuation of treatment should be considered. Treatment with Otezla was also associated with an increase in occurrence of depression compared to placebo. The FDA is requiring a pregnancy exposure registry as a post-marketing requirement to assess the risk to pregnant women related to Otezla exposure. The most common side effects were diarrhea, nausea, and headache. To reduce the risk of GI symptoms, titrate over a 6 day period to recommended dose of 30 mg twice daily. The dosage in severe renal impairment is decreased to just 30 mg once daily. Otezla is available in 10 mg, 20 mg, and 30 mg tablets.
Approved: March 21, 2014

NewPlegridy approved for patients with relapsing forms of multiple sclerosis.

Plegridy (peginterferon beta-1A) - Biogen - Plegridy is indicated for the treatment of patients with relapsing forms of multiple sclerosis. The recommended dosage of Plegridy is 125 mcg injected subcutaneously every 14 days. Patients should start treatment with 63 mcg on day 1 and on day 15, the dose is increased to 94 mcg, reaching the full dose of 125 mcg on day 29. Patients continue with the full dose of 125 mcg every 14 days thereafter. The most common side effects were injection site erythema, influenza-like illness, pyrexia, headache, myalgia, chills, injection site pain, asthenia, injection site pruritus, and arthralgia.
Approved: August 15, 2014.

Sixextro to treat adults with skin infections.

Sivextro (tedizolid phosphate) - Cubist - Sivextro is approved to treat patients with acute bacterial skin and skin structure infections (ABSSSI) caused by certain susceptible bacteria, including Staphylococcus aureus (including methicillin-resistant strains (MRSA) and methicillin-susceptible strains), various Streptococcus species, and Enterococcus faecalis. The application for Sivextro was designated as a qualified infectious disease product (QIDP) and received an expedited review. Sivextro’s QIDP designation also qualifies it for an extra 5 years of marketing exclusivity to be added to certain exclusivity periods already provided by the FDA. Sivextro’s safety and efficacy were evaluated in 2 clinical trials with 1,315 adults with ABSSSI. Participants were randomly assigned to receive Sivextro or linezolid, another antibacterial drug approved to treat ABSSSI and the results showed Sivextro was as effective as linezolid for the treatment of ABSSSI. Sivextro is available for IV and oral use. The most common side effects were nausea, headache, diarrhea, vomiting and dizziness. The safety and efficacy of Sivextro have not been evaluated in patients with decreased levels of white blood cells (neutropenia), so alternative therapies should be considered. The recommended dose of SIVEXTRO is 200 mg given once daily for 6 days either orally or IV. It is available in a 200 mg tablet and as a 200 mg single dose vial for reconstitution.
Approved: June 20, 2014.

NewStriverdi Respimat to treat patients with COPD.

Striverdi (olodaterol) - Boehringer - Striverdi inhalation spray is used to treat patients with COPD, including chronic bronchitis and/or emphysema that are experiencing airflow obstruction. Striverdi Respimat is a long-acting beta-adrenergic agonist that helps the muscles around the airways in the lungs stay relaxed to prevent symptoms. The safety and effectiveness of Striverdi Respimat was evaluated in 3,104 people diagnosed with COPD and was shown to have improved lung function as compared to placebo. Striverdi Respimat should not be used in patients with acutely deteriorating COPD and may cause serious side effects, including narrowing and obstruction of the respiratory airway (paradoxical bronchospasm) and cardiovascular effects. The drug carries a boxed warning that it may increase the risk of asthma-related death. The most common side effects were nasopharyngitis, upper respiratory tract infection, bronchitis, cough, urinary tract infection, dizziness, rash, diarrhea, back pain and arthralgia. Striverdi Respimat should be used with caution in patients with convulsive disorders, thyrotoxicosis, diabetes mellitus, ketoacidosis, in patients with known or suspected prolongation of the QT interval, and in patients who are unusually responsive to sympathomimetic amines.
Approved: July 31, 2014.

Sylvant for treatment of Castleman’s disease.

Sylvant (siltuximab) - Janssen - Sylvant has been approved to treat patients with multicentric Castleman’s disease (MCD), a rare disorder similar to lymphoma. MCD causes an abnormal overgrowth of immune cells in lymph nodes and related tissues in the body. The disease usually affects adults who suffer from fever, night sweats, weight loss and weakness or fatigue because their body’s immune system is weakened and cannot fight infections. Sylvant is an injection that works by blocking a protein that stimulates abnormal growth of immune cells. It is intended for patients with MCD who do not have HIV or human herpes virus 8 (HHV-8). The FDA reviewed Sylvant under its priority review program, which provides an expedited review for drugs that demonstrate the potential to be a significant improvement in safety or effectiveness in the treatment of a serious condition. Sylvant was also granted orphan product designation because it is being used to treat a rare disease or condition. Sylvant’s safety and effectiveness were evaluated in a clinical trial of 79 patients with MCD who were HIV and HHV-8 negative. Patients were randomly assigned to receive a combination of Sylvant and best supportive care, or placebo and best supportive care. Results showed 34 percent of patients treated with Sylvant and best supportive care experienced tumor response, while no patient treated with placebo and best supportive care did. Common side effects include pruritis, weight gain, rash, increased levels of uric acid in the blood and upper respiratory tract infection. Sylvant is for intravenous infusion only and administered as an 11 mg/kg dose given over 1 hour every 3 weeks. Sylvant is available in a 100 mg or 400 mg single dose vial.
Approved: April 23, 2014

Tanzeum for treatment of type 2 diabetes.

Tanzeum (albiglutide) - GSK - Tanzeum is approved for subcutaneous injection to improve glycemic control, along with diet and exercise, in adults with type 2 diabetes. Tanzeum is a glucagon-like-peptide-1 receptor agonist, which is a hormone that helps regulate blood sugar levels. The drug’s safety and effectiveness were evaluated in 8 clinical trials, which included more than 2,000 patients with type 2 diabetes. The trials showed that the patients had an improvement in their HbA1c level. Tanzeum can be used alone or in combination with other drugs, including metformin, glimepiride, pioglitazone, and insulin. It should not be used to treat type 1 diabetes; in those who have diabetic ketoacidosis; or as first line therapy for patients adequately controlled on diet and exercise. Do not use Tanzeum in patients with a history of pancreatitis or pre-existing severe gastrointestinal disease. Tanzeum is administered once weekly at any time of day, without regard to meals. It is injected SQ into the abdomen, thigh, or upper arm with an initial dose of 30 mg. Dose can be increased to 50 mg once weekly in patients requiring additional glycemic control. It is available in a 30 or 50 mg single-dose pen. The most common side effects include upper respiratory tract infection, diarrhea, nausea, and injection site reaction. Tanzeum has a black box warning included for a risk of thyroid C-cell tumors. The FDA is requiring post-marketing studies for: use in pediatrics; a medullary thyroid carcinoma (MTC) case registry for 15 years; and a cardiovascular outcomes trial (CVOT) to evaluate cardiovascular risks in patients with high baseline risk of cardiovascular disease.
Approved: April 15, 2014

New Trulicity to treat type 2 diabetes.

Trulicity (dulaglutide) - Eli Lilly- The FDA approved Trulicity once-weekly subcutaneous injection to improve glycemic control, along with diet and exercise, in adults with type 2 diabetes. Trulicity is a glucagon-like peptide-1 (GLP-1) receptor agonist, a hormone that helps normalize blood sugar levels. The drug's safety and effectiveness were evaluated in 6 clinical trials in which 3,342 patients with type 2 diabetes received Trulicity. Patients receiving Trulicity had an improvement in their blood sugar control as observed with reductions in HbA1c level. Trulicity has been studied as a stand-alone therapy and in combination with other type 2 diabetes therapies, including metformin, sulfonylurea, thiazolidinedione, and prandial insulin. Trulicity should not be used to treat people with type 1 diabetes; those who have increased ketones in their blood or urine (diabetic ketoacidosis); those with severe stomach or intestinal problems; or as first-line therapy for patients who cannot be managed with diet and exercise. Trulicity has a boxed warning that tumors of the thyroid gland have been observed in rodent studies with Trulicity but that it is unknown whether Trulicity causes thyroid tumors in humans. The FDA approved Trulicity with a Risk Evaluation and Mitigation Strategy (REMS), which consists of a communication plan to inform health care professionals about the serious risks associated with Trulicity. In clinical trials, the most common side effects were nausea, diarrhea, vomiting, abdominal pain, and decreased appetite. It is available in a carton of 4 single dose or prefilled syringes in 2 strengths; 0.75 mg/0.5 mL solution or 1.5 mg/0.5 mL and must be stored under refrigeration. The recommended initiating dose of Trulicity is 0.75 mg once weekly and may be increased to 1.5 mg once weekly for additional glycemic control. The maximum recommended dose is 1.5 mg once weekly.
Approved: September 18, 2014.

Vimizim for treatment of mucopolysaccharidosis type IVA.

Vimizim (elosulfase alfa) - BioMarin - Vimizim is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). The safety and efficacy of Vimizim were evaluated in a 24 week, randomized, double-blind, placebo-controlled clinical trial of 176 patients with MPS IVA. The majority of the patients (82%) presented with a medical history of musculoskeletal conditions, which included knee deformity (52%), kyphosis (31%), hip dysplasia (22%), prior spinal fusion surgery (22%) and arthralgia (20%). At baseline, all enrolled patients could walk more than 30 meters but less than 325 m in 6 minutes. Patients received Vimizim 2 mg/kg once per week, Vimizim 2 mg/kg once every other week, or placebo. The primary endpoint was the change from baseline in the distance walked in 6 minutes at Week 24. The other endpoints included changes from baseline in the rate of stair climbing in 3 minutes and changes from baseline in urine KS levels at Week 24. The treatment effect in the distance walked in 6 minutes, compared to placebo, was 22.5 m in patients who received Vimizim 2 mg/kg once per week. There was no difference in the rate of stair climbing between patients who received Vimizim 2 mg/kg once per week and those who received placebo. Patients who received Vimizim 2 mg/kg once every other week performed similarly in the 6-MWT and 3-MSCT as those who received placebo. The reduction in urinary KS levels from baseline, a measure of pharmacodynamic effect, was greater in the Vimizim treatment groups compared to placebo. The relationship between urinary KS and other measures of clinical response has not been established. The most common side effects were pyrexia, vomiting, headache, nausea, abdominal pain, chills, and fatigue. The recommended dose is 2 mg per kg given IV over 3.5 to 4.5 hours, based on infusion volume, once a week. Pre-treatment with antihistamines with or without antipyretics is recommended 30 to 60 minutes prior to the infusion. Vimizim is available in a (1 mg/ml) 5 ml single dose vial and should be stored under refrigeration at 2°C to 8°C. Must be diluted prior to use.
Approved: February 14, 2014

NewZontivity for cardiovascular risk reduction.

Zontivity (vorapaxar) - Merck-Zontivity is approved for high risk patients (previous heart attack or peripheral artery disease) to reduce the risk of heart attack, stroke, cardiovascular death, and need for procedures to restore blood flow to the heart. Zontivity is the first in a new class of drug, called a protease-activated receptor-1 antagonist (PAR-1). It is an anti-platelet drug, designed to decrease the tendency of platelets to clump together and form a clot, which will decrease the risk of heart attack and stroke. Zontivity will increase the risk of bleeding which can be life threatening. This will be included on the label as a Boxed Warning to alert health care professionals. It is contraindicated in people who have had a stroke, TIA, or bleeding in the head, because the risk of bleeding in the head is increased. In clinical trials, Zontivity lowered the risk of heart attack, stroke, and cardiovascular death in high risk patients from 9.5% to 7.9% over a 3 year period. The most common side effects are bleeding, GI bleeding, anemia, and depression.
Approved: May 8, 2014.

Zydelig to treat patients with 3 types of blood cancers.

Zydelig (idelalisib) - Gilead - Zydelig is being granted traditional approval to treat patients whose chronic lymphocytic leukemia (CLL) has relapsed. Used in combination with Rituxan, Zydelig is to be used in patients for whom Rituxan alone would be considered appropriate therapy due to other existing medical conditions. Zydelig has also been granted accelerated approval to treat patients with relapsed follicular B-cell non-Hodgkin lymphoma and relapsed small lymphocytic lymphoma. The safety and effectiveness to treat relapsed CLL was established in a clinical trial of 220 participants who were randomly assigned to receive Zydelig and Rituxan or placebo and Rituxan. The trial was stopped for effectiveness following the first interim analysis point, due to patients being treated with Zydelig and Rituxan had the possibility of living at least 10.7 months without disease progressing compared to about 5.5 months for patients treated with placebo and Rituxan. Results from a 2nd interim analysis continued to show a significant improvement for Zydelig and Rituxan over placebo and Rituxan. Safety and effectiveness to treat relapsed FL and relapsed SLL were established in a clinical trial with 123 participants with slow-growing non-Hodgkin lymphomas. All patients were treated with Zydelig and were evaluated for complete or partial disappearance of their cancer after treatment. Results showed 54 percent of participants with relapsed FL and 58 percent of patients with SLL experienced ORR. Zydelig has a boxed warning for fatal and serious toxicities including liver toxicity, diarrhea and colitis, pneumonitis and intestinal perforation that can occur in Zydelig-treated patients. Zydelig is also being approved with a Risk Evaluation and Mitigation Strategy (REMS) comprised of a communication plan to ensure healthcare providers who are likely to prescribe Zydelig are fully informed about these risks. Side effects include diarrhea, fever, fatigue, nausea, cough, pneumonia, abdominal pain, chills and rash. Laboratory abnormalities include neutropenia, hypertriglyceridemia, hyperglycemia and elevated liver enzymes. The recommended maximum starting dose of Zydelig is 150 mg administered twice daily. Zydelig is available in a 100 mg and 150 mg tablet.
Approved: July 23, 2014

Zykadia for treatment of metastatic non-small cell lung cancer.

Zykadia (ceritinib) - Novartis -Zykadia is approved as a kinase inhibitor indicated for the treatment of patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib. This indication is approved under accelerated approval based on tumor response rate and duration of response. An improvement in survival or disease-related symptoms has not been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. The safety of Zykadia was based on 255 patients with ALK positive tumors who received Zykadia at a dose of 750 mg orally once daily. The most common adverse reactions (>or=25%) were diarrhea, nausea, transaminitis, vomiting, abdominal pain, fatigue, decreased appetite and constipation. The most common CTCAE grade 3-4 adverse reactions (>or=5%) were diarrhea, fatigue, transaminitis, hyperglycemia, hypophosphatemia, increased lipase levels, and anemia. Other serious adverse reactions include interstitial lung disease and QT prolongation. The recommended dose of Zykadia is 750 mg once daily on an empty stomach until disease progression or unacceptable toxicity. Zykadia is available in a 150 mg capsule.
Approved: April 29, 2014