New Drugs 2013
Adempas for pulmonary hypertension.
Adempas (riociguat) - Bayer - Recently approved for pulmonary hypertension Adempas belongs to a class of drugs called soluble guanylate cyclase stimulators that help arteries relax to increase blood flow and decrease blood pressure. It is intended for patients with chronic thromboembolic pulmonary hypertension (CTEPH) after surgery or patients who cannot undergo surgery, to improve their ability to exercise. Adempas is also indicated for patients with pulmonary arterial hypertension (PAH) of unknown causes, inherited or associated with connective tissue diseases.. The effectiveness of Adempas to treat CTEPH were established in a trial with 261 participants randomized to Adempas,or a placebo. The study was designed to measure the change in the distance a patient could walk in six minutes. After 16 weeks of treatment, the average improvement in a 6-minute walk distance in participants treated with Adempas was 46 meters (about 150 feet) more than in those treated with placebo. The trial evaluating the effectiveness of Adempas to treat PAH included 443 participants randomly assigned to take Adempas or placebo. After 12 weeks of treatment, the 6-minute walk distance in patients treated with Adempas improved by an average of 36 meters (about 118 feet) more than in patients treated with placebo. Adempas carries a Boxed Warning that the drug should not be used in pregnant women because it can harm the developing fetus. Common side effects include: headache, dizziness, indigestion (dyspepsia), tissue swelling (peripheral edema), nausea, diarrhea and vomiting.This medication is highly restricted by Bayer.
Approved: October 8
Brintellix for major depression.
Brintellix (vortioxetine) - Takeda - Brintellix has been approved for the treatment of major depressive disorders (MDD) A person with MDD experiences mood changes and other symptoms that interfere with a person's ability to work, sleep, study, eat and enjoy once-pleasurable activities. Episodes of depression often recur throughout a person's lifetime, although some may experience a single occurrence. Six clinical studies in which adults with MDD were randomly assigned to receive Brintellix or placebo demonstrated that Brintellix is effective in treating depression. An additional study showed Brintellix decreased the likelihood of participants becoming depressed again after treatment of their MDD episode. These studies were conducted in the United States and other countries. The most common side effects included: nausea,constipation and vomiting.
Approved: Sept. 30
Clinolipid for intravenous feeding in adult patients.
Clinolipid (Lipid) - Baxter - The FDA recently approved this intravenous lipid preparation as a source of calories and essential fatty acids for adult patients who are unable to eat or drink. Clinolipid is a lipid emulsion that contains a mixture of refined olive oil and refined soybean oil. The fatty acids contained in Clinolipid serve as an important source of energy in patients receiving parenteral nutrition. The omega-3: omega-6 fatty acid ratio in Clinolipid has not been shown to improve clinical outcomes compared to other lipid emulsion products. The safety and effectiveness of Clinolipid were evaluated in clinical studies comparing Clinolipid with a soybean oil-based lipid emulsion found that Clinolipid is an effective source of energy in adults. The most common side effects in patients included infectious complications, nausea and vomiting, excess fat (lipids) in the blood, high blood sugar, low levels of protein in the blood and abnormal liver function tests.
Approved: October 3
Diclegis for morning sickness
Diclegis (doxylamine succinate and pyridoxine hydrochloride) - Duchesnay - the FDA has approved Diclegis to treat pregnant women experiencing nausea and vomiting. It is intended for women who have not adequately responded to conservative management of nausea and vomiting during pregnancy, such as dietary and lifestyle modifications. Diclegis was studied in 261 women experiencing nausea and vomiting due to pregnancy. Women were randomly assigned to receive two weeks of treatment with Diclegis or a placebo. The study results showed that women taking Diclegis experienced greater improvement in nausea and vomiting than those taking placebo. The most common side effect is drowsiness or sleepiness, which can be severe.
Gilotrif (afatinib) for late stage non-small cell lung cancer
Gilotrif (afatinib) - Boehringer Ingelheim - Gilotrif, a tyrosine-kinase inhibitor, has been approved for the treatment of non-small cell lung cancer (NSCLC) in tumors that express specific types of epidermal growth factor receptor (EGFR) gene mutations, as detected by an FDA-approved test. About 85 percent of lung cancers are NSCLC, making it the most common type of lung cancer. EGFR gene mutations are present in about 10 percent of NSCLC, with the majority of these gene mutations expressing EGFR exon 19 deletions or exon 21 L858R substitution. It is intended for patients whose tumors express the EGFR exon 19 deletions or exon 21 L858R substitution gene mutations. Gilotrif is being approved concurrently with the therascreen EGFR RGQ PCR Kit, a companion diagnostic that helps determine if a patient’s lung cancer cells express the EGFR mutations. Gilotrif’s safety and effectiveness were established in a clinical study of 345 participants with metastatic NSCLC whose tumors harbored EGFR mutations. Participants were randomly assigned to receive Gilotrif or up to six cycles of the chemotherapy drugs pemetrexed and cisplatin. Participants receiving Gilotrif had a delay in tumor growth (progression-free survival) that was 4.2 months later than those receiving chemotherapy. There was no statistically significant difference in survival. Common side effects include: diarrhea, skin breakouts that resemble acne, dry skin, itching (pruritus), inflammation of the mouth, skin infection around the nails (paronychia), decreased appetite, decreased weight, inflammation of the bladder (cystitis), nose bleed, runny nose, fever, eye inflammation and low potassium levels in the blood (hypokalemia). Serious side effects include severe diarrhea that can result in kidney failure and severe dehydration, severe rash, lung inflammation and liver toxicity.
Approved: July 12
Invokana for type 2 diabetes.
Invokana (Canagliflozin) – Janssen - this recently approved drug is the first in a new class of drugs known as sodium-glucose co-transporter 2 (SGLT2) inhibitors. Invokana 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. Its safety and effectiveness were evaluated in nine trials involving over 10,285 patients with type 2 diabetes. These showed improvement in hemoglobin A1c levels (a measure of blood sugar control) and fasting plasma glucose (blood sugar) levels. The FDA is requiring five postmarketing studies for Invokana: a cardiovascular outcomes trial; an enhanced pharmacovigilance program to monitor for malignancies, serious cases of pancreatitis, severe hypersensitivity reactions, photosensitivity reactions, liver abnormalities, and adverse pregnancy outcomes; a bone safety study; and two pediatric studies under the Pediatric Research Equity Act (PREA), including a pharmacokinetic and pharmacodynamic study and a safety and efficacy study. The most common side effects are: vaginal yeast infections and urinary tract infections. Because Invokana is associated with a diuretic effect, it can cause a reduction in intravascular volume leading to orthostatic or postural hypotension (a sudden fall in blood pressure when standing up). This may result in symptoms such as dizziness or fainting, and is most common in the first three months of therapy.
Approved: March 29
Kadcyla for HER2-positive, late-stage (metastatic) breast cancer
Kadcyla (Ado-trastuzumab Emtansine) - Genentech - This drug reprents a new therapy for patients with HER2-positive, late-stage (metastatic) breast cancer. Kadcyla is intended for patients who were previously treated with trastuzumab, another anti-HER2 therapy, and taxanes, a class of chemotherapy drugs commonly used for the treatment of breast cancer. Kadcyla is trastuzumab connected to a drug called DM1 that interferes with cancer cell growth. Kadcyla delivers the drug to the cancer site to shrink the tumor, slow disease progression and prolong survival. It is the fourth approved drug that targets the HER2 protein. Results showed that patients treated with Kadcyla had a median progression-free survival of 9.6 months compared to 6.4 months in patients treated with lapatinib plus capecitabine. The median overall survival was 30.9 months in the Kadcyla group and 25.1 months in the lapatinib plus capecitabine group. The most common side effects reported were nausea, fatigue, pain in the muscles or joints, low levels of platelets in the blood (thrombocytopenia), increased levels of liver enzymes, headache, and constipation.
There is a BOXED WARNING that Kadcyla can cause liver toxicity, heart toxicity and death. The drug can also cause severe life-threatening birth defects, and pregnancy status should be verified prior to starting Kadcyla treatment.
Approved: Febuary 22, 2013
Kynamro for familial hypercholesterolemia
Kynamro (Mipomersen) - Genzyme - Approved for use in treating homozygeous familial hypercholesterolemia (HoFH) Kynamro must be given by injection once weekly. In one clinical trial, after 26 weeks of injections the average level of LDL-C "bad" cholesterol fell by 25%. Side effects were: flu-like symptoms, nausea, headache, and elevations in liver enzymes. This medication carries a BOXED WARNING of the possibility of serious liver toxicity.
Approved: January 29, 2013-02-20
Mekinist (trametinib), for melanoma.
Mekinist (trametinib) - GSK - Similar to Tafinlar Mckinest,, a MEK inhibitor, is approved to treat patients whose tumors express the BRAF V600E or V600K gene mutations. Approximately half of melanomas arising in the skin have a BRAF gene mutation. Mekinist was studied in 322 patients with metastatic or unresectable melanoma with the BRAF V600E or V600K gene mutation. Patients were randomly assigned to receive either Mekinist or chemotherapy. Patients receiving Mekinist had a delay in tumor growth that was 3.3 months later than those on chemotherapy. Patients who previously used Tafinlar or other inhibitors of BRAF did not appear to benefit from Mekinist. The most serious side effects reported: included heart failure, lung inflammation, skin infections and loss of vision. Common side effects included rash, diarrhea, tissue swelling (peripheral edema) and skin breakouts that resemble acne.
Approved: May 29
Nesina for Type 2 diabetes
Nesina (Alogliptin) - Takeda - This drug was approved for the treatment of Type 2 diabetes. In extensive clinical studies Nesina lowered the HbA1c of 0.4% to 0.6% over 26 weeks of use. Common side effects were; runny nose, headache, and upper respiratory tract infections.
Approved: January 25, 2013
Osphena for moderate to severe dyspareunia
Osphena (Ospemifene) - Shionogi - This drug has been approved for treating moderate to severe dyspareunia in postmenopausal women. Dyspareunia is associated with declining levels of estrogen during menopause, which leads to vulvovaginal atrophy, often resulting in pain during sexual intercourse. Ospemifene is a novel selective estrogen receptor modulator that makes vaginal tissue thicker and less fragile, resulting in a reduction in the amount of pain women experience with sexual intercourse. Ospemifene is taken orally with food once daily. The safety and effectiveness of ospemifene for dyspareunia were established in 1889 postmenopausal women with symptoms of vulvar and vaginal atrophy who were randomly assigned to receive ospemifene or a placebo. After 12 weeks of treatment, results showed a statistically significant improvement of dyspareunia in ospemifene-treated women compared with placebo-treated women. Common adverse effects of ospemifene reported during clinical trials included hot flushes/flashes, vaginal discharge, muscle spasms, genital discharge, and excessive sweating.
There is a BOXED WARNING - Ospemifene is being approved with a boxed warning alerting women and healthcare professionals that the drug, which acts like estrogen on vaginal tissues, has been shown to stimulate the endometrium and cause it to thicken. Women should see their physician if they experience any unusual bleeding as it may be a sign of endometrial cancer or a condition that can lead to it. It should be prescribed for the shortest duration consistent with treatment goals and risks for the individual woman, The boxed warning also states the incidence rates of thrombotic and hemorrhagic strokes (0.72 and 1.45 per 1000 women, respectively) and the incidence rate of deep vein thrombosis (1.45 per 1000 women).
Approved: February 26, 2013
Pomalyst for multiple myeloma
Pomalyst (Pomalid) - Celgene - Approved for the treatment of progressive multiple myeloma after treatment failure on other anti-cancer drugs. Pomalyst is a tablet that modulates the body's immune system to destroy cancerous cells and inhibit their growth. It is intended for patients who have received at least two prior therapies, including lenalidomide and bortezomib, and whose disease did not respond to treatment and progressed within 60 days of the last treatment (relapsed and refractory). Clinical results showed 7.4 percent of patients treated with Pomalyst alone achieved objective response rate (ORR). The median duration of response has not yet been reached in these patients. In patients treated with Pomalyst plus low-dose dexamethasone, 29.2 percent achieved ORR with a 7.4-month median duration of response.
Pomalyst carries a BOXED WARNING that the drug should not be used in pregnancy because it can cause severe life-threatening birth defects, and that the drug may cause blood clots.
Common side effects include a decrease in white blood cells (neutropenia), fatigue and weakness, low red blood cell count (anemia), constipation, diarrhea, low levels of platelets in the blood (thrombocytopenia), upper respiratory tract infections, back pain and fever.
Approved: February 8, 2013
Procysbi for the management of nephropathic cystinosis.
Procysbi (cysteamine bitartrate) - Raptor - The FDA has approved Procysbi to prevent the buildup of cystine in the kidney. Cystinosis may lead to slow body growth and small stature, weak bones and developing and worsening kidney failure. There are three types of cystinosis, the most severe being nephropathic cystinosis, which severely damages the kidneys. The major study supporting Procysbi’s safety and effectiveness involved 43 adult and pediatric patients with nephropathic cystinosis. Patients received Cystagon (a currently available oral product) or Procysbi for three weeks before being switched to the other product for an additional three weeks. Blood testing showed Procysbi was as effective as Cystagon in controlling cystine levels. The most common side effects include: nausea, bad breath, abdominal pain, constipation, indigestion or upset stomach, headache, drowsiness and dizziness. Other uncommon but serious side effects include ulcers or bleeding of the stomach or intestine, altered mental state, seizures, severe skin rashes and allergic reactions.
Approved: April 30
Ravicti for urea cycle disorders
Ravicti (Glycerol Phenylbutyrate) - Hyperion - This is the 2nd drug approved for the treatment of Urea Cycle disorders (UCD's). The ability of this drug to control blood levels of ammonia was found comparable to the only other drug approved for this indication, sodium phenylbutrate (Buphenyl). UCDs are genetic disorders that involve deficiencies of specific enzymes involved in the urea cycle, a series of biochemical steps normally required to remove ammonia from the blood. When protein is absorbed and broken down by the body, it produces nitrogen as a waste product. The urea cycle removes nitrogen from the blood and converts it to urea, which is removed from the body through urine. In people with UCDs, nitrogen accumulates and remains in the body as ammonia, which can travel to the brain and cause brain damage, coma or death. Ravicti, a liquid taken three times a day with meals, helps dispose of ammonia in the body. It is intended for patients whose UCD cannot be managed by a protein-restricted diet or amino acid supplements alone. Ravicti must be used with a protein-restricted diet and, in some cases, dietary supplements. The most common side effects in patients treated with Ravicti include diarrhea, flatulence and headache.
Approved: February 1, 2013
Tafinlar (dabrafenib)- GSK - Tafinlar, a BRAF inhibitor, is approved to treat patients with melanoma whose tumors express the BRAF V600E gene mutation. The FDA approved Tafinlar with a genetic test called the THxID BRAF test, a companion diagnostic that will help determine if a patient’s melanoma cells have the V600E or V600K mutation in the BRAF gene. Tafinlar was studied in 250 patients with BRAF V600E gene mutation-positive metastatic or unresectable melanoma. Patients were randomly assigned to receive Tafinlar or the chemotherapy drug dacarbazine. Patients who took Tafinlar had a delay in tumor growth that was 2.4 months later than those receiving dacarbazine. The most serious side effects reported in patients receiving Tafinlar included an increased risk of skin cancer, fevers that may be complicated by hypotension (low blood pressure), severe rigors (shaking chills), dehydration, kidney failure and increased blood sugar levels. The most common side effects reported in patients receiving Tafinlar included thickening of the skin (hyperkeratosis), headache, fever, joint pain, non-cancerous skin tumors, hair loss and hand-foot syndrome.
Approved: May 29
Tecfidera (dimethyl fumarate) for relapsing forms of multiple sclerosis (MS).
Tecfidera (dimethyl fumarate) - Biogen Idac - this new drug has been approved for the treatment of Multiple sclerosis. Results from two clinical trials showed that those taking Tecfidera had fewer MS relapses compared to people taking an inactive pill (placebo). One of the trials showed that those taking Tecfidera experienced a worsening of disability less often than patients taking a placebo. Adverse effects include: a decrease in white blood cell count (lymphocytes) which can raise the risk of infection. Before starting treatment, and annually thereafter, the FDA recommends that the patient’s white blood cell count be assessed by their health care provider. Other side effects include: flushing (warmth and redness) and stomach problems (nausea, vomiting, and diarrhea).
Approved: March 27
Tivicay for HIV-1 infections
Tivicay (dolutegravir) - ViiV Healthcare - This new drug has a unique mechanism in treating the HIV virus. Tivicay is an integrase strand transfer inhibitor that interferes with one of the enzymes necessary for HIV to multiply. It is a pill taken daily in combination with other antiretroviral drugs. Tivicay has been approved for use in a broad population of HIV-infected patients. It can be used to treat HIV-infected adults who have never taken HIV therapy (treatment-naïve) and HIV-infected adults who have previously taken HIV therapy (treatment-experienced), Tivicay is also approved for children ages 12 years and older weighing at least 40 kilograms (kg) who are treatment-naïve or treatment-experienced but have not previously taken other integrase strand transfer inhibitors. Tivicay’s safety and efficacy in adults was evaluated in 2,539 participants enrolled in four clinical trials. Depending on the trial, participants were randomly assigned to receive Tivicay or Isentress (raltegravir), each in combination with other antiretroviral drugs, or Atripla, a fixed-dose combination of efavirenz, emtricitabine and tenofovir. Results showed Tivicay-containing regimens were effective in reducing viral loads. Side effects include difficulty sleeping (insomnia) and headache. Serious side effects include hypersensitivity reactions and abnormal liver function in participants co-infected with hepatitis B and/or C.
Approved: August 12
Uceris for Ulcerative Colitis
Uceris (Budesonide) - Santarus - This is a substantially improved method for delivering a steroid to the colon, the location of ulcerative colitis. Uceris, given in tablet form is taken once daily, does not release its steroid component until it reaches the colon. Clinical studies which compared Uceris with a placebo tablet resulted in 17-18% of patients experiencing a remission fo the colitis compared with 4-7% of patients treated with placebo. Side effects consited of nausea, decreased blood cortisol, upper abdominal pain, fatigue, abdominal distension, acne and urinary tract infections. It should be noted that the overall incidence of side effects did not differ from placebo.
Approved: January 15, 2013
Valchlor for Mycosis Fungoides
Valchlor (Mechlorethamine) - Actilion - this has been approved for the topical (skin) treatment of mycosis fungoides, a manifestation of T-cell lymphomas. When applied to the skin once a day 60% of patients had a response defined as a reduction of 50% or more in lesion severity. Complete responses were rare.
Approved: August 26