New Drugs 2015
Repatha to treat certain patients with high cholesterol.
Repatha (evolocumab) – Amgen - The FDA has approved Repatha injection for some patients who are unable to get their low-density lipoprotein cholesterol under control with current treatment options. Repatha, the second drug approved in a new class of drugs known as PCSK9 inhibitors, is approved for use in addition to diet and maximally-tolerated statin therapy in adult patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, or clinical atherosclerotic cardiovascular disease, such as heart attacks or strokes, who require additional lowering of LDL cholesterol. Repatha is an antibody that targets a specific protein, called PCSK9. PCSK9 reduces the number of receptors on the liver that remove LDL cholesterol from the blood. By blocking PCSK9’s ability to work, more receptors are available to get rid of LDL cholesterol from the blood and, as a result, lower LDL cholesterol levels. The efficacy and safety of Repatha were evaluated in one 52-week placebo-controlled trial and eight 12-week placebo-controlled trials in patients with primary hyperlipidemia, including two that specifically enrolled patients with HeFH and one that enrolled participants with HoFH. In one of the 12-week studies, 329 participants with HeFH, who required additional lowering of LDL cholesterol despite statins with or without other lipid-lowering therapies, were randomized to receive Repatha or placebo for 12 weeks. Patients taking Repatha had an average reduction in LDL cholesterol of approximately 60 percent, compared to placebo. The most adverse effects of Repatha include nasopharyngitis, upper respiratory tract infection, flu, back pain, and reactions such as redness, pain, or bruising where the injection is given. Allergic reactions, such as rash and hives, have been reported with the use of Repatha. Patients should stop using Repatha and get medical help if they experience symptoms of a serious allergic reaction. Multiple clinical trials have demonstrated that statins lower the risk of having a heart attack or stroke. A trial evaluating the effect of adding Repatha to statins for reducing cardiovascular risk is ongoing.
Approved: August 27, 2015.
Addyi for treatment of sexual desire disorder.
Addyi (flibanserin) – Sprout - Addyi (flibanserin) has been approved to treat acquired, generalized hypoactive sexual desire disorder (HSDD) in premenopausal women. Prior to Addyi’s approval, there were no FDA-approved treatments for sexual desire disorders in men or women. HSDD is characterized by low sexual desire that causes marked distress or interpersonal difficulty and is not due to a co-existing medical or psychiatric condition, problems within the relationship, or the effects of a medication or other drug substance. HSDD is acquired when it develops in a patient who previously had no problems with sexual desire. HSDD is generalized when it occurs regardless of the type of sexual activity, the situation or the sexual partner. Addyi can cause severe hypotension and loss of consciousness. These risks are increased and more severe when patients drink alcohol or take Addyi with certain medicines (known as moderate or strong CYP3A4 inhibitors) that interfere with the breakdown of Addyi in the body. Because of the alcohol interaction, the use of alcohol is contraindicated while taking Addyi. Health care professionals must assess the likelihood of the patient reliably abstaining from alcohol before prescribing Addyi. Addyi is being approved with a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use (ETASU). The FDA is requiring this REMS because of the increased risk of severe hypotension and syncope due to the interaction between Addyi and alcohol. The REMS requires that prescribers be certified with the REMS program by enrolling and completing training. Certified prescribers must counsel patients using a Patient-Provider Agreement Form about the increased risk of severe hypotension and syncope and about the importance of not drinking alcohol during treatment with Addyi. Additionally, pharmacies must be certified with the REMS program by enrolling and completing training. Certified pharmacies must only dispense Addyi to patients with a prescription from a certified prescriber. Additionally, pharmacists must counsel patients prior to dispensing not to drink alcohol during treatment with Addyi. Addyi is also being approved with a Boxed Warning to highlight the risks of severe hypotension and syncope in patients who drink alcohol during treatment with Addyi, in those who also use moderate or strong CYP3A4 inhibitors, and in those who have liver impairment. Addyi is contraindicated in these patients. In addition, the FDA is requiring the company that owns Addyi to conduct 3 well-designed studies in women to better understand the known serious risks of the interaction between Addyi and alcohol. Addyi is a serotonin 1A receptor agonist and a serotonin 2A receptor antagonist, but the mechanism by which the drug improves sexual desire and related distress is not known. Addyi is taken once daily and is dosed at bedtime to help decrease the risk of adverse events occurring due to possible hypotension, syncope and central nervous system depression (such as sleepiness and sedation). Patients should discontinue treatment after 8 weeks if they do not report an improvement in sexual desire and associated distress. The effectiveness of the 100 mg bedtime dose of Addyi was evaluated in three 24-week randomized, double-blind, placebo-controlled trials in about 2,400 premenopausal women with acquired, generalized HSDD. The average age of the trial participants was 36 years, with an average duration of HSDD of approximately 5 years. In these trials, women counted the number of satisfying sexual events, reported sexual desire over the preceding 4 weeks and reported distress related to low sexual desire. On average, treatment with Addyi increased the number of satisfying sexual events and decreased the distress score related to sexual desire over placebo. Additional analyses explored whether the improvements with Addyi were meaningful to patients, taking into account the effects of treatment seen among those patients who reported feeling much improved or very much improved overall. Across the 3 trials, about 10 percent more Addyi-treated patients than placebo-treated patients reported meaningful improvements in satisfying sexual events, sexual desire or distress. Addyi has not been shown to enhance sexual performance. The most common adverse effects associated with the use of Addyi are dizziness, somnolence, nausea, fatigue, insomnia and dry mouth.
Approved: August 18, 2015.
Daklinza to treat chronic hepatitis C genotype 3 infections.
Daklinza (daclatasvir) – BMS - Daklinza has been approbed for use with sofosbuvir to treat hepatitis C virus (HCV) genotype 3 infections. Daklinza is the first drug that has demonstrated safety and efficacy to treat genotype 3 HCV infections without the need for co-administration of interferon or ribavirin, two FDA-approved drugs also used to treat HCV infection. The safety and efficacy of Daklinza in combination with sofosbuvir were evaluated in a clinical trial of 152 treatment-naive and treatment-experienced patients with chronic HCV genotype 3 infection. Patients received Daklinza 60 mg plus sofosbuvir 400 mg once daily for 12 weeks and were monitored for 24 weeks post treatment. The studies were designed to measure whether a patient’s hepatitis C virus was no longer detected in the blood 12 weeks after finishing treatment (sustained virologic response), suggesting a patient’s infection had been cured. Results showed that 98 percent of the treatment-naive patients with no cirrhosis of the liver and 58 percent of the treatment-naive patients with cirrhosis achieved sustained virologic response. Of the patients who were treatment-experienced, 92 percent with no cirrhosis of the liver and 69 percent with cirrhosis achieved sustained virologic response. Daklinza labeling carries a Limitations of Use statement to inform prescribers that sustained virologic response rates are reduced in HCV genotype 3 infected patients with cirrhosis. Safety information was available for approximately 1,900 patients with HCV treated with the recommended dose of Daklinza in combination with other anti-HCV drugs in clinical trials. The most common adverse effects of Daklinza with sofosbuvir were fatigue and headache. Daklinza carries a warning for patients and health care providers that serious symptomatic bradycardia and cases requiring pacemaker intervention have been reported when amiodarone is co-administered with sofosbuvir in combination with another HCV direct-acting antiviral, including Daklinza. Co-administration of amiodarone with Daklinza in combination with sofosbuvir is not recommended.
Approved: July 24, 2015.
Odomzo to treat advanced basal cell carcinoma.
Odomzo (sonidegib) – Novartis – Odomzo has been approved to treat patients with locally advanced basal cell carcinoma that has recurred following surgery or radiation therapy, or who are not candidates for surgery or radiation therapy. Odomzo is a pill taken once a day. It works by inhibiting a molecular pathway, called the Hedgehog pathway, which is active in basal cell cancers. By suppressing this pathway, Odomzo may stop or reduce the growth of cancerous lesions. Odomzo carries a Boxed Warning alerting healthcare professionals that Odomzo may cause death or severe birth defects in a developing fetus when given to a pregnant woman. Pregnancy status should be verified prior to the start of treatment with Odomzo, and both male and female patients should be warned about these risks and advised to use effective contraception. The efficacy of Odomzo was established in a multi-center, double-blind clinical trial, in which 66 patients with locally advanced basal cell carcinoma were randomly assigned to receive Odomzo 200 mg daily and 128 patients were assigned to receive Odomzo 800 mg daily. The study’s primary endpoint was objective response rate, which is the percentage of patients who experienced partial shrinkage or complete disappearance of their tumor(s). Results showed that 58 percent of patients treated with Odomzo 200 mg had their tumors shrink or disappear. This effect lasted at least 1.9 to 18.6 months, and approximately half of the responding patients’ tumor shrinkage lasted six months or longer. Response rates were similar in patients who received Odomzo 800 mg daily, however side effects were more common at this dose. At a dose of 200 mg daily, the most common adverse effects of Odomzo were muscle spasms, alopecia, dysgeusia (distortion in the sense of taste), fatigue, nausea, musculoskeletal pain, diarrhea, decreased weight, decreased appetite, myalgia, abdominal pain, headache, pain, vomiting and pruritus. Odomzo also has the potential to cause serious musculoskeletal-related side effects, including increased serum creatine kinase levels (with rare reports of rhabdomyolysis), muscle spasms, and myalgia.
Praluent to treat certain patients with high cholesterol.
Praluent (alirocumab) – Sanofi - The FDA has approved Praluent which is the first cholesterol-lowering treatment approved in a new class of drugs known as proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitors. Praluent is approved for use in addition to diet and maximally tolerated statin therapy in adult patients with heterozygous familial hypercholesterolemia (HeFH) or patients with clinical atherosclerotic cardiovascular disease such as heart attacks or strokes, who require additional lowering of LDL cholesterol. HeFH is an inherited condition that causes high levels of low-density lipoprotein (LDL) cholesterol. A high level of LDL cholesterol in the blood is linked to cardiovascular disease. Heart disease is the number one cause of death for Americans, both men and women. According to the Centers for Disease Control and Prevention, about 610,000 people die of heart disease in the U.S. every year. Praluent is an antibody that targets a specific protein, called PCSK9, which works by reducing the number of receptors on the liver that remove LDL cholesterol from the blood. By blocking PCSK9’s ability to work, more receptors are available to get rid of LDL cholesterol from the blood and, as a result, lower LDL cholesterol levels. The safety and efficacy of Praluent were evaluated in 5 placebo-controlled trials, involving 2,476 patients exposed to Praluent. All participants had HeFH or were otherwise at high risk for heart attack or stroke, and were taking maximally tolerated doses of a statin, with or without other lipid?modifying therapies. Patients taking Praluent had an average reduction in LDL cholesterol ranging from 36 to 59 percent, as compared to placebo. Multiple clinical trials have demonstrated that statins lower the risk of having a heart attack or stroke. A trial evaluating the effect of adding Praluent to statins on reducing cardiovascular risk is ongoing. The most common adverse effects of Praluent include itching, swelling, pain, or bruising where injection is given, nasopharyngitis, and flu. Allergic reactions, such as hypersensitivity vasculitis and hypersensitivity reactions requiring hospitalization, have been reported with the use of Praluent. Patients should stop using Praluent and get medical help if they experience symptoms of a serious allergic reaction.
Approved: July 24, 2015.
Rexulti to treat adults with schizophrenia.
Rexulti (brexpiprazole) – Otsuka – The FDA has approved Rexulti tablets to treat adults with schizophrenia and as an add-on treatment to an antidepressant medication to treat adults with major depressive disorder (MDD). The effectiveness of Rexulti in treating schizophrenia was evaluated in 1,310 patients in two 6-week clinical trials. Rexulti was shown to reduce the occurrence of symptoms of schizophrenia compared to placebo. The effectiveness of Rexulti as an add-on treatment for MDD was evaluated in two 6-week trials that compared Rexulti plus an antidepressant to placebo plus an antidepressant in 1,046 patients for whom an antidepressant alone did not effectively treat their symptoms. The patients taking Rexulti reported fewer symptoms of depression than those taking the placebo. Rexulti and other drugs used to treat schizophrenia have a Boxed Warning alerting health care professionals about an increased risk of death associated with the off-label use of these drugs to treat behavioral problems in older people with dementia-related psychosis. No drug in this class is approved to treat patients with dementia-related psychosis. The most common adverse effects reported by patients taking Rexulti in clinical trials included weight gain and an inner sense of restlessness, such as feeling the need to move.
Approved: July 13, 2015.
Entresto for the treatment of heart failure.
Entresto (sacubitril /valsartan) – Novartis - The FDA has approved Entresto (sacubitril/valsartan) tablets for the treatment of heart failure. The drug has been shown to reduce the rate of cardiovascular death and hospitalization related to heart failure. Entresto was studied in a clinical trial of more than 8,000 adults and was shown to reduce the rate of cardiovascular death and hospitalizations related to heart failure compared to another drug, enalapril. Most patients were also receiving currently approved heart failure treatments, including beta-blockers, diuretics, and mineralocorticoid antagonists. The most common adverse effects in clinical trial participants being treated with Entresto were hypotension, hyperkalemia, and renal impairment. Angioedema was also reported with Entresto; black patients and patients with a prior history of angioedema are at a higher risk. Patients should be advised to get emergency medical help right away if they have symptoms of angioedema or trouble breathing while on Entresto. Health care professionals should advise patients not to use Entresto with any drug from the angiotensin converting enzyme (ACE) inhibitor class because the risk of angioedema is increased. When switching between Entresto and an ACE inhibitor, use of the 2 drugs should be separated by 36 hours. Health care professionals should counsel patients about the risk of harm to an unborn baby. If pregnancy is detected, Entresto should be discontinued as soon as possible.
Approved: July 7, 2015.
Orkambi to treat cystic fibrosis.
Orkambi (lumacaftor /ivacaftor) – Vertex- Orkambi has been approved by the FDA to treat cystic fibrosis (CF) in patients 12 years and older, who have the F508del mutation, which causes the production of an abnormal protein that disrupts how water and chloride are transported in the body. Having two copies of this mutation (one inherited from each parent) is the leading cause of CF. Orkambi received FDA’s breakthrough therapy designation because the sponsor demonstrated through preliminary clinical evidence that the drug may offer a substantial improvement over available therapies. The FDA also reviewed Orkambi under the priority review program and was granted orphan drug designation. CF is a serious genetic disorder that results in the formation of thick mucus that builds up in the lungs, digestive tract and other parts of the body leading to severe respiratory and digestive problems, as well as other complications such as infections and diabetes. CF, which affects about 30,000 people in the United States, is the most common fatal genetic disease in Caucasians. The safety and efficacy of Orkambi was studied in two double-blind, placebo-controlled clinical trials of 1,108 patients with CF who were 12 years and older with the F508del mutation. In both studies, patients with CF who took Orkambi, two pills taken every 12 hours, demonstrated improved lung function compared to those who took placebo. The safety and efficacy of Orkambi have not been established in patients with CF other than those with the F508del mutation. If a patient’s genotype is unknown, an FDA cleared CF mutation test should be used to detect the presence of the F508del mutation on both alleles of the CFTR gene. The most common adverse effects of Orkambi include shortness of breath, upper respiratory tract infection, nausea, diarrhea, and rash. Women who took Orkambi also had increased menstrual abnormalities such as increased bleeding.
Approved: July 2, 2015.
Kengreal to prevent formation of blood clots.
Kengreal (cangrelor) – Medicines Company - The FDA has approved Kengreal, an IV antiplatelet drug that prevents formation of harmful blood clots in the coronary arteries. It is approved for adult patients undergoing percutaneous coronary intervention (PCI), a procedure used to open a blocked or narrowed coronary artery to improve blood flow to the heart muscle. By preventing platelets from accumulating, Kengreal reduces the risk of serious clotting complications related to the procedure, including heart attack and stent thrombosis. As with other FDA approved anti-platelet drugs, bleeding, including life-threatening bleeding, is the most serious risk of Kengreal. In a clinical trial that compared Kengreal to Plavix in more than 10,000 patients, Kengreal significantly reduced the occurrence of heart attack, the need for further procedures to open the artery and stent thrombosis. The overall occurrence of serious bleeding was low but more common with Kengreal than with Plavix. Approximately one in every 170 Kengreal patients had a serious bleed as compared with approximately one in every 275 Plavix patients.
Approved: June 22, 2015.
Viberzi to treat irritable bowel syndrome with diarrhea.
Viberzi (eluxadoline) – Patheon - The FDA has approved Viberzi for irritable bowel syndrome with diarrhea in adult men and women. Viberzi, which contains a new active ingredient (eluxadoline), is taken twice daily with food. Viberzi activates receptors in the nervous system that can reduce bowel contractions. VIBERZI has mixed opioid receptor activity; it is a mu receptor agonist, a delta receptor antagonist, and a kappa receptor agonist. Viberzi is intended to treat adults with IBS-D. The safety and effectiveness of Viberzi for treatment of irritable bowel syndrome were established in 2 double-blind, placebo-controlled clinical trials in which 2,425 patients were randomly assigned to receive Viberzi or placebo. Results showed Viberzi was more effective in simultaneously reducing abdominal pain and improving stool consistency than placebo over the 26 week period of treatment. The most common adverse effects in patients treated with Viberzi include constipation, nausea and abdominal pain. The most serious known risk associated with Viberzi is the risk of spasm in the sphincter of Oddi, the smooth muscle that surrounds the end portion of the common bile and pancreatic ducts, which can result in pancreatitis. Viberzi should not be used in patients with a history of bile duct obstruction, pancreatitis, severe liver impairment, or severe constipation, and in patients who drink more than three alcoholic beverages per day. The FDA has recommended that VIBERZI be classified as a controlled substance. Once VIBERZI receives final scheduling designation, the updated label will be available and pending final scheduling designation, product launch is anticipated in the first quarter of 2016.
Approved: May 27, 2015.
Kybella a treatment for adults with moderate-to-severe fat below the chin.
Kybella (deoxycholic acid) – Kythera - The FDA has approved Kybella, a treatment for adults with moderate-to-severe fat below the chin, known as submental fat. Using Kybella for the treatment of fat outside of the submental area is not approved and is not recommended. Kybella is identical to the deoxycholic acid that is produced in the body. Deoxycholic acid produced in the body helps the body absorb fats. Kybella is a cytolytic drug, which when injected into tissue physically destroys the cell membrane. When properly injected into submental fat, the drug destroys fat cells; however, it can also destroy other types of cells, such as skin cells, if it is inadvertently injected into the skin. Kybella is administered as an injection into the fat tissue in the submental area. Patients may receive up to 50 injections in a single treatment, with up to 6 single treatments administered no less than 1 month apart. Kybella is being provided in single patient use vials and should not be diluted or mixed with any other compounds. The safety and effectiveness of Kybella for treatment of submental fat were established in 2 clinical trials which enrolled 1,022 adult patients with moderate or severe submental fat. Patients were randomly assigned to receive Kybella or a placebo for up to 6 treatments. The results showed that reductions in submental fat were observed more frequently in participants who received Kybella versus placebo. Kybella can cause serious adverse effects, including nerve injury in the jaw that can cause an uneven smile or facial muscle weakness, and trouble swallowing. The most common adverse effects of Kybella include swelling, bruising, pain, numbness, redness and areas of hardness in the treatment area. Kybella should not be used outside of the submental area, and it should not be used if there is an infection at the injection site. Caution should also be used in patients who have had prior surgical or aesthetic treatment of the submental area. Kybella is being distributed in a dispensing pack that has a unique hologram on the vial label, therefore if no hologram is present, do not use the product.
Approved: April 29, 2015.
Corlanor to reduce hospitalization from worsening heart failure.
Corlanor (ivabradine) – Amgen – The FDA has approved Corlanor for use in certain people who have chronic heart failure caused by the lower-left part of their heart not contracting well. The drug is indicated for patients who have symptoms of heart failure that are stable, a normal heartbeat with a resting heart rate of at least 70 beats per minute and are also taking beta blockers at the highest dose they can tolerate. The safety and efficacy of Corlanor was studied in a clinical trial of 6,505 patients. Corlanor reduced hospitalization for worsening of heart failure when compared to placebo. The most common adverse effects experienced in clinical trial patients were bradycardia, hypertension, atrial fibrillation, and temporary vision disturbance (flashes of light). Patients should alert their physician if they experience symptoms of an irregular heartbeat, feel that the heart is pounding or racing, have chest pressure, or worsened shortness of breath. Low heart rate is a common adverse effect of Corlanor and can be serious. Patients should tell their physician if they have symptoms such as dizziness, weakness or fatigue.
Approved: April 15, 2015.
Cholbam to treat rare bile acid synthesis disorders
Cholbam (cholic acid) – Asklepion – The FDA approved Cholbam (cholic acid) capsules, the first FDA approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for patients with peroxisomal disorders (including Zellweger spectrum disorders). Patients with these rare, genetic, metabolic conditions exhibit manifestations of liver disease, steatorrhea (presence of fat in the stool) and complications from decreased fat-soluble vitamin absorption. Cholbam is approved as an oral treatment for children aged three weeks and older, and adults. The manufacturer of Cholbam was granted a rare pediatric disease priority review voucher–a provision that encourages development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. The efficacy of Cholbam for the treatment of patients with bile acid synthesis disorders due to single enzyme defects was assessed in a single arm trial involving 50 patients treated over an 18 year period. An extension trial followed 21 of these patients and enrolled an additional 12 patients with short-term efficacy data available for an additional 21 months. On average, patients were 4 years of age at the start of cholic acid treatment. Response to treatment was evaluated by improvements in baseline liver function tests and weight. Responses were noted in 64 percent of patients with evaluable data. Two-thirds of patients survived greater than three years. The efficacy of Cholbam for the treatment of peroxisomal disorders, including Zellweger spectrum disorders, was assessed in a single arm, treatment trial involving 29 patients treated over an 18 year period. An extension trial followed 10 of these patients and enrolled an additional 2 patients with interim efficacy data available for 21 additional months. The majority of patients were less than 2 years of age at the start of cholic acid treatment. Response to treatment was evaluated by improvements in baseline liver function tests and weight. Responses were noted in 46 percent of patients with evaluable data. Forty-two percent of patients survived greater than 3 years. Cholbam’s effects on other manifestations of bile acid disorders due to single enzyme defects or peroxisomal disorders, such as neurologic symptoms, have not been established. The most common adverse effect in patients treated with Cholbam was diarrhea. The use of Cholbam should be carefully monitored by an experienced hepatologist or pediatric gastroenterologist, and treatment discontinued in patients developing worsening liver function.
Approved: March 17, 2015.
Unituxin for high-risk neuroblastoma
Unituxin (dinutuximab) – United Therapeutics - The FDA has approved Unituxin as part of first-line therapy for pediatric patients with high-risk neuroblastoma, a type of cancer that most often occurs in young children. Unituxin is an antibody that binds to the surface of neuroblastoma cells. Unituxin is being approved for use as part of a multimodality treatment, including surgery, chemotherapy and radiation therapy for patients who achieved at least a partial response to prior first-line multi-agent, multimodality therapy. The safety and efficacy of Unituxin were evaluated in a clinical trial of 226 pediatric patients with high-risk neuroblastoma whose tumors shrunk or disappeared after treatment with multiple-drug chemotherapy and surgery followed by additional intensive chemotherapy and who subsequently received bone marrow transplantation support and radiation therapy. Patients were randomly assigned to receive either an oral retinoid drug, isotretinoin (RA), or Unituxin in combination with interleukin-2 and granulocyte-macrophage colony-stimulating factor, which are thought to enhance the activity of Unituxin by stimulating the immune system, and RA. Three years after treatment assignment, 63 percent of patients receiving the Unituxin combination were alive and free of tumor growth or recurrence, compared to 46 percent of patients treated with RA alone. In an updated analysis of survival, 73 percent of patients who received the Unituxin combination were alive compared with 58 percent of those receiving RA alone. Unituxin has a Boxed Warning alerting patients and health care professionals that Unituxin irritates nerve cells, causing severe pain that requires treatment with intravenous narcotics and can also cause nerve damage and life-threatening infusion reactions, including upper airway swelling, difficulty breathing, and low blood pressure, during or shortly following completion of the infusion. Unituxin may also cause other serious side effects including infections, eye problems, electrolyte abnormalities and bone marrow suppression. The most common adverse effects of Unituxin were severe pain, fever, low platelet counts, infusion reactions, low blood pressure, hyponatremia, elevated liver enzymes, anemia, vomiting, diarrhea, low potassium levels in the blood, capillary leak syndrome, neutropenia and lymphopenia, hives, and low blood calcium levels. Unituxin is supplied in a carton containing one 17.5 mg/5 mL (3.5 mg/mL) single-use vial. Store Unituxin vials under refrigeration at 2°C to 8°C until time of use.
Approved: March 10, 2015.
Cresemba a new antifungal drug
Cresemba (isavuconazonium sulfate) - Astellas Pharma - The FDA has approved Cresemba, a new antifungal drug product used to treat adults with invasive aspergillosis and invasive mucormycosis, rare but serious infections. Cresemba belongs to a class of drugs called azole antifungal agents, which target the cell membrane of a fungus. The approval of Cresemba to treat invasive aspergillosis was based on a clinical trial involving 516 patients randomly assigned to receive either Cresemba or voriconazole. Cresemba’s approval to treat invasive mucormycosis was based on a single-arm clinical trial involving 37 patients treated with Cresemba and compared with the natural disease progression associated with untreated mucormycosis. Both studies showed Cresemba was safe and effective in treating these serious fungal infections. The most common adverse effects associated with Cresemba include nausea, vomiting, diarrhea, headache, abnormal liver blood tests, low potassium levels in the blood (hypokalemia), constipation, shortness of breath (dyspnea), coughing and tissue swelling (peripheral edema). Cresemba may also cause serious side effects including liver problems, infusion reactions and severe allergic and skin reactions. Cresemba is available in oral and intravenous formulations.
Approved: March 6, 2015.
Avycaz new antibacterial drug product
Avycaz (ceftazidime-avibactam) – Forest Pharmaceuticals – The FDA has approved Avycaz, a new antibacterial drug, to treat patients with complicated intra-abdominal infections, including kidney infections, who have limited or no alternative treatment options. The efficacy of Avycaz was supported by the findings of the efficacy and safety of ceftazidime for the treatment of cIAI and cUTI. The contribution of avibactam to Avycaz was based on data from in vitro studies and animal models of infection. Avycaz was studied in 2 Phase 2 trials. Both were not designed with any recognized hypothesis for inferential testing against any active comparators. Avycav for injection is supplied in a single use, clear glass vial. Each vial contains 2 grams ceftazidime and 0.5 grams avibactam. Avycaz vials should be stored at 25°C (77°F); excursions permitted between 15°C and 30°C (59°F and 86°F). The most common adverse effects include vomiting, nausea, constipation, and anxiety. Patients should be informed of these risks and should also be advised that decreased efficacy, seizures and other neurologic events can be seen in patients with renal impairment. Serious skin reactions and anaphylaxis may occur in patients who are allergic to penicillin.
Approved: February 25, 2015.
Farydak for treatment of multiple myeloma
Farydak (panobinostat) – Novartis - The FDA approved Farydak for the treatment of patients with multiple myeloma. Multiple myeloma causes plasma cells to rapidly multiply and crowd out other healthy blood cells from the bone marrow. When the bone marrow has too many plasma cells, the cells may move to other parts of the body, which can weaken the body’s immune system, lead to anemia and cause other bone and kidney problems. Farydak works by inhibiting the activity of enzymes, known as histone deacetylases (HDACs). This process may hinder the over-development of plasma cells in multiple myeloma patients or cause these dangerous cells to die. Farydak is the first HDAC inhibitor approved to treat multiple myeloma. It is intended for patients who have received at least 2 prior standard therapies, including bortezomib and an immunomodulatory agent. Farydak is to be used in combination with bortezomib, a type of chemotherapy, and dexamethasone, an anti-inflammatory medication. The safety and efficacy of Farydak in combination with bortezomib and dexamethasone was demonstrated in 193 clinical trial patients with multiple myeloma who received at least 2 prior treatments that included bortezomib and an immunomodulatory agent. Patients were randomly assigned to receive a combination of Farydak, bortezomib and dexamethasone, or bortezomib and dexamethasone alone. Study results showed patients receiving the Farydak combination saw a delay in their disease progression for about 10.6 months, compared to 5.8 months in patients treated with bortezomib and dexamethasone alone. Additionally, 59 percent of Farydak-treated patients saw their cancer shrink or disappear after treatment, versus 41 percent in those receiving bortezomib and dexamethasone. Farydak has a Boxed Warning alerting patients and health care professionals that severe diarrhea and severe and fatal cardiac events, arrhythmias and electrocardiogram changes have happened in patients receiving Farydak. Because of these risks, Farydak is being approved with a REMS consisting of a communication plan to alert health care professionals of these risks and how to minimize them. The most common adverse effects of Farydak were diarrhea, tiredness, nausea, swelling in the arms or legs, decreased appetite, fever, vomiting and weakness. The most common laboratory abnormalities were hypophosphatemia, hypokalemia, hyponatremia, increased creatinine, thrombocytopenia, leukopenia and anemia. Healthcare professionals should also advise patients of the risk of bleeding in the GI tract and the lungs, and hepatotoxicity.
Approved: February 23, 2015.
Lenvima for thyroid cancer
Lenvima (lenvatinib) - Eisai - The FDA has granted approval to Lenvima to treat patients with progressive, differentiated thyroid cancer whose disease progressed despite receiving radioactive iodine therapy. The most common type of thyroid cancer, DTC is a cancerous growth of the thyroid gland which is located in the neck and helps regulate the body’s metabolism. The National Cancer Institute estimates that 62,980 Americans were diagnosed with thyroid cancer and 1,890 died from the disease in 2014. Lenvima is a kinase inhibitor, which works by blocking certain proteins from helping cancer cells grow and divide. Lenvima was approved approximately two months ahead of schedule. Lenvima’s efficacy was demonstrated in 392 patients with progressive, radioactive iodine-refractory DTC who were randomly assigned to receive either Lenvima or a placebo. Results showed Lenvima-treated patients lived a median of 18.3 months without their disease progressing, compared to a median of 3.6 months for patients who received a placebo. Additionally, 65 percent of patients treated with Lenvima saw a reduction in tumor size, compared to the 2 percent of patients who received a placebo. A majority of patients randomly assigned to receive the placebo were treated with Lenvima upon disease progression. The most common adverse effects of Lenvima were hypertension, fatigue, diarrhea, joint and muscle pain, decreased appetite, decreased weight, nausea, stomatitis, headache, vomiting, proteinuria, swelling and pain in the palms, hands and/or the soles of the feet, abdominal pain and changes in voice volume or quality. Lenvima may cause serious adverse effects, including cardiac failure, arterial thromboembolic events, hepatotoxicity, renal failure and impairment, gastrointestinal perforation or a fistula formation, QT Interval Prolongation, hypocalcaemia, the simultaneous occurrence of headache, confusion, seizures and visual changes, hemorrhage, risks to an unborn child if a patient becomes pregnant during treatment, and impairing suppression of the production of thyroid-stimulating hormone.
Approved: February 13, 2015.
Ibrance for postmenopausal women with advanced breast cancer
Ibrance (palbociclib) – Pfizer - The FDA granted accelerated approval to Ibrance to treat metastatic breast cancer. Ibrance works by inhibiting molecules, known as cyclin-dependent kinases (CDKs) 4 and 6, involved in promoting the growth of cancer cells. Ibrance is intended for postmenopausal women with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative metastatic breast cancer who have not yet received an endocrine-based therapy. It is to be used in combination with letrozole. The FDA granted Ibrance breakthrough therapy designation because the sponsor demonstrated that the drug may offer a considerable improvement over available therapies. It also received a priority review, which provides for an expedited review of drugs intended to provide a significant improvement in safety or effectiveness in the treatment of a serious condition or meet an unmet medical need. Ibrance was approved more than two months ahead of schedule. The drug’s efficacy was proven in 165 postmenopausal patients with ER-positive, HER2-negative advanced breast cancer who had not received previous treatment for advanced disease. Clinical study patients were randomly assigned to receive Ibrance in combination with letrozole or letrozole alone. Patients treated with Ibrance plus letrozole lived about 20.2 months without their disease progressing, compared to about 10.2 months seen in patients receiving only letrozole. Information on overall survival is not available at this time. The most common adverse effects were a decrease in neutrophils (neutropenia), low levels of white blood cells (leukopenia), fatigue, anemia, upper respiratory infection, nausea, stomatitis, alopecia, diarrhea, thrombocytopenia, decreased appetite, vomiting, asthenia, peripheral neuropathy and nosebleed. Healthcare professionals should inform patients of these risks. It is recommended that treatment begin with a 125 mg dose for 21 days, followed by 7 days without treatment. Healthcare professionals are advised to monitor complete blood count prior to start of therapy and at the beginning of each cycle, as well as on Day 14 of the first 2 cycles, and as clinically indicated.
Approved: February 3, 2015.
Natpara to control low blood calcium levels in patients with hypoparathyroidism
Natpara (parathyroid hormone) - NPS Pharmaceuticals - The FDA approved Natpara (parathyroid hormone) to control hypocalcaemia in patients with hypoparathyroidism, a rare disease that affects approximately 60,000 people in the United States. Hypoparathyroidism is caused by loss of function of the parathyroid glands and occurs most commonly as a result of surgical removal of the parathyroid glands and more rarely as a result of autoimmune or congenital diseases. Patients with hypoparathyroidism can experience numbness, tingling, muscle twitching, spasms or cramps, abnormal heart rhythm, and seizures as a consequence of low blood calcium levels. Hypoparathyroidism is also associated with long-term complications such as kidney damage, kidney stones, development of cataracts and calcification of soft tissues. Natpara, a hormonal injection administered once daily, helps to regulate the body’s calcium levels. The FDA granted Natpara orphan drug designation because it is intended to treat a rare disease. The safety and effectiveness of Natpara were evaluated in a clinical trial of 124 patients who were randomly assigned to receive Natpara or a placebo. The trial was designed to determine whether Natpara can be used as a substitute for, or be used to help reduce the amount of, vitamin D or oral calcium taken by patients. Results showed 42 percent of Natpara-treated patients achieved normal blood calcium levels on reduced doses of calcium supplements and vitamin D, compared to 3 percent of placebo-treated patients. Natpara has a boxed warning that bone cancer has been observed in rat studies with Natpara. It is unknown whether Natpara causes bone cancer in humans, but because of this risk, Natpara is only recommended for use in patients whose hypocalcaemia cannot be controlled on calcium supplementation and vitamin D, and for whom the benefits outweigh this risk. Natpara is only available through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). The most common adverse effects observed in Natpara-treated participants were sensations of tingling, tickling, pricking, or burning of the skin, low blood calcium, headache, high blood calcium, and nausea.
Approved: January 23, 2015.
Cosentyx to treat moderate to severe plaque psoriasis
Cosentyx (secukinumab) – Novartis – The FDA approved Cosentyx to treat adults with moderate-to-severe plaque psoriasis. Cosentyx is an antibody that binds to a protein (interleukin (IL)-17A) which is involved in inflammation. By binding to IL-17A, Cosentyx prevents it from binding to its receptor, and inhibits its ability to trigger the inflammatory response that plays a role in the development of plaque psoriasis. Cosentyx is administered as an injection under the skin and is intended for patients who are candidates for systemic therapy, phototherapy, or a combination of both. Safety and efficacy was established in 4 clinical trials with a total of 2,403 patients with plaque psoriasis who were candidates for phototherapy or systemic therapy. Patients were randomly assigned to receive Cosentyx or a placebo. The results showed that Cosentyx achieved greater clinical response than placebo, with skin that was clear or almost clear, as assessed by scoring of the extent, nature and severity of psoriatic changes of the skin. Cosentyx is being approved with a Medication Guide to inform patients that, because Cosentyx is a medicine that affects the immune system, patients may have a greater risk of getting an infection. Serious allergic reactions have been reported with the use of Cosentyx. Caution should be exercised when considering the use of Cosentyx in patients with a chronic infection or history of recurrent infection, and in patients with active Crohn’s Disease. The most common adverse reactions include diarrhea and upper respiratory infections.
Approved: January 21, 2015.
Savaysa to reduce risk of stroke and blood clots
Savaysa (edoxaban) - Daiichi Sankyo - The FDA has approved the anti-clotting drug Savaysa to reduce the risk of stroke and systemic embolism in patients with atrial fibrillation that is not caused by a heart valve problem. Savaysa also has been approved to treat deep vein thrombosis and pulmonary embolism in patients who have already been treated with an anti-clotting drug administered by injection or infusion, for 5 to 10 days. The safety and efficacy of Savaysa in treating patients with atrial fibrillation not caused by cardiac valve disease was studied in a clinical trial of 21,105 patients. The trial compared 2 dose levels of Savaysa with warfarin for their effects on rates of stroke and systemic emboli. The trial results showed the higher dose of Savaysa to be similar to warfarin for the reduction in the risk of stroke. Warfarin is highly effective in reducing the risk of stroke in patients with atrial fibrillation, but increases the risk of bleeding. Savaysa demonstrated significantly less major bleeding compared with warfarin. In a clinical trial with 8,292 patients, Savaysa was studied in the treatment of DVT and PE. The study compared the safety and efficacy of Savaysa to warfarin in treating patients with a DVT and/or PE to reduce the rate of recurrence of symptomatic venous thromboembolism (VTE) events. In the trial, 3.2 percent of patients taking Savaysa had a symptomatic recurrent VTE compared to 3.5 percent of those taking warfarin. The most common adverse effects were bleeding and anemia. As with other FDA-approved anti-clotting drugs, bleeding, including life-threatening bleeding, is the most serious risk with Savaysa. Currently there is no treatment that has been proven to reverse the anti-coagulant effect of Savaysa. Savaysa has a Boxed Warning that provides important dosing and safety information for health care professionals about specific patient groups, including a warning that Savaysa is less effective in atrial fibrillation patients with a creatinine clearance greater than 95 milliliters per minute. This should be assessed before initiating therapy with Savaysa. Patients with creatinine clearance greater than 95 milliliters per minute have an increased risk of stroke compared to similar patients given warfarin. Savaysa should not be used in nonvalvular atrial fibrillation patients with a higher creatinine clearance.
Approved: January 8, 2015.