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
Aptiom for partial seizures
Aptiom (eslicarbazepine acetate) - Sunovion - Aptiom is approved for the treatment of partial seizures, the most common type of seizure seen in people with epilepsy. Seizures can cause a wide range of symptoms, including repetitive limb movements, unusual behavior and generalized convulsions with loss of consciousness. Seizures can have serious consequences, including injury and death. Three clinical studies in which participants with partial epilepsy were randomly assigned to receive Aptiom or placebo. Patients treated with Aptiom demonstrated statistically significant reductions in standardized seizure frequency versus placebo, and significantly more Aptiom treated patients experienced seizure frequency reduction of 50% or more from baseline (41% compared to 22% for placebo-treated patients). The most common side effects reported by patients receiving Aptiom in clinical trials included dizziness, drowsiness, nausea, headache, double-vision, vomiting, fatigue and loss of coordination.
Approved Nov. 8, 2013
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.
Gazyva for chronic lymphocytic leukemia
Gazyva (obinutuzumab)-Genentech - The FDA approved Gazyva (obinutuzumab) for use in combination with chlorambucil to treat patients with previously untreated chronic lymphocytic leukemia (CLL). Gazyva’s approval is based on a study of 356 participants in a randomized open-label multicenter trial comparing Gazyva in combination with chlorambucil to chlorambucil alone in participants with previously untreated CLL. Participants receiving Gazyva in combination with chlorambucil demonstrated a significant improvement in progression free survival: an average of 23 months compared with 11.1 months with chlorambucil alone. The most common side effects observed in participants receiving Gazyva in combination with chlorambucil were infusion-related reactions, a decrease in infection-fighting white blood cells (neutropenia), a low level of platelets in the blood (thrombocytopenia), low red blood cells (anemia), pain in the muscles and bones (musculoskeletal pain), and fever (pyrexia).
Approved: Nov. 1, 2013
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
Imbruvica (ibrutinib) for with mantle cell lymphoma (MCL)
Imbruvica (ibrutinib) - Janssen - Imbruvica is intended for patients with Mantle Cell Lymphoma (MCL) who have received at least one prior therapy. Imbruvica’s accelerated approval was based on a study where 111 participants were given Imbruvica daily until their disease progressed or side effects became intolerable. Results showed nearly 66 percent of participants had their cancer shrink or disappear after treatment (overall response rate). An improvement in survival or disease-related symptoms has not been established. The most common side effects are low levels of platelets in the blood (thrombocytopenia), diarrhea, a decrease in infection-fighting white blood cells (neutropenia), anemia, fatigue, musculoskeletal pain, swelling (edema), upper respiratory infection, nausea, bruising, shortness of breath (dyspnea), constipation, rash, abdominal pain, vomiting, and decreased appetite.
Approved: Nov. 13, 2013
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
Olysio for chronic hepatitis C
Olysio (simeprevir) - Janssen - Olysio has been approved for adults with Hepatitis C who have a diseased liver that is still functioning, including cirrhosis, who have not received treatment for their infection (treatment naïve) or for whom previous treatment has not been effective (treatment experienced). Olysio is a protease inhibitor that blocks a specific protein needed by the hepatitis C virus to replicate. Results showed 80 percent of treatment-naive participants given Olysio plus peginterferon-alfa and ribavirin achieved sustained virologic response, compared to 50 percent of participants receiving peginterferon-alfa and ribavirin alone. In one of the studies with treatment-experienced participants whose infection returned (prior relapsers), 79 percent receiving Olysio plus peginterferon-alfa and ribavirin achieved sustained virologic response compared to 37 percent of participants receiving peginterferon-alfa and ribavirin alone. Another study examined Olysio’s safety and effectiveness in treatment-experienced participants, including prior relapsers, those who partially responded to prior therapy (partial responders) and those who did not respond to prior therapy (null responders). Adding Olysio improved response rates in each of these subgroups compared to peginterferon-alfa and ribavirin alone. The most common side effects in patients treated with Olysio in combination with peginterferon-alfa and ribavirin were rash (including photosensitivity), itching (pruritis) and nausea. Serious photosensitivity reactions resulting in hospitalization were reported. Patients will be advised to limit sun exposure and to use sun protective measures during treatment with Olysio in combination with peginterferon alfa and ribavirin. Olysio should not be used alone to treat chronic hepatitis C infection.
Approved: Dec. 6, 2013
Opsumit to treat pulmonary arterial hypertension
Opsumit (macitentan) - Actelion - This medication has recently been approved for treatment adults with pulmonary arterial hypertension (PAH), a chronic, progressive and debilitating disease that can lead to death or the need for lung transplantation. Opsumit belongs to a class of drugs called endothelin receptor blockers, which act to relax the pulmonary arteries, decreasing blood pressure in the lungs. Opsumit’s safety and effectiveness were established in a clinical trial where 742 participants were randomly assigned to take Opsumit or placebo. The average treatment duration was about two years. In the study, Opsumit was effective in delaying disease progression, a finding that included a decline in exercise ability, worsening symptoms of PAH or need for additional PAH medication.Common side effects observed in those treated with Opsumit include low red blood cell count (anemia), common cold-like symptoms (nasopharyngitis), sore throat, bronchitis, headache, flu and urinary tract infection. Because of the possibility of fetal injury this medication is highly restricted in the Unites States.
Approved: October 18, 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
Sovaldi for chronic hepatitis C
Sovaldi (sofosbuvir) - Gilead - The FDA has approved Sovaldi (sofosbuvir) to treat chronic hepatitis C virus (HCV) infection. Sovaldi is the first drug that has demonstrated safety and efficacy to treat certain types of HCV infection without the need for co-administration of interferon.Sovaldi is a nucleotide analog inhibitor that blocks a specific protein needed by the hepatitis C virus to replicate. Sovaldi is to be used as a component of a combination antiviral treatment regimen for chronic HCV infection Sovaldi’s effectiveness was evaluated in clinical trials consisting of 1,947 participants who had not previously received treatment for their disease (treatment- naive) or had not responded to previous treatment (treatment-experienced), The trials end-point was whether the hepatitis C virus was no longer detected in the blood at least 12 weeks after finishing treatment (sustained virologic response). Results showed a treatment regimen containing Sovaldi was effective in treating multiple types of the hepatitis C virus. Additionally, Sovaldi demonstrated efficacy in participants who could not tolerate or take an interferon-based treatment regimen and in participants with liver cancer awaiting liver transplantation, addressing unmet medical needs in these populations. The most common side effects were fatigue and headache.
Approved: Dec. 6, 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) - Actelion - 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
Velphoro for hyperphosphatemia in patients with chronic kidney disease (CKD)
Velphoro (sucroferric oxyhydroxide) - Fresenius - This mdication has been approved for treatment of hyperphosphatemia in patients with chronic kidney disease (CKD) who are receiving dialysis. Velphoro controls hyperphosphatemia over the long term and has a lower pill burden (3 tablets/day) than sevelamer carbonate, the current standard of care in patients with CKD who are receiving dialysis.
Approved: Nov. 28, 2013
Xiaflex for Peyronie’s disease
Xiaflex (collagenase clostridium histolyticum) - Auxilium - Xiaflex is the first FDA-approved non-surgical treatment option for men with this condition, who have a plaque (lump) in the penis that results in a curvature deformity of at least 30 degrees upon erection. Peyronie’s disease is caused by scar tissue that develops under the skin of the penis. This scar tissue causes an abnormal bend during erection and can cause problems such as bothersome symptoms during intercourse. Previously approved for the treatment of Dupuytren’s contracture, Xiaflex is believed to work by breaking down the buildup of collagen (a structural protein in connective tissue) that causes the curvature deformity. The safety and effectiveness of Xiaflex for the treatment of Peyronie’s disease were established in two randomized double- blind, placebo-controlled studies in 832 men with Peyronie’s disease with penile curvature deformity of at least 30 degrees. Participants were given up to four treatment cycles of Xiaflex or placebo and were then followed 52 weeks. Xiaflex treatment significantly reduced penile curvature deformity and related bothersome effects compared with placebo.
Approved: Dec. 6, 2013