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Flashback: FDA Drug Approvals 2013

Diabetes Compound Alogliptin Approved in Three Formulations

On January 25, 2013, Takeda Pharmaceutical received approval for alogliptin, a type 2 diabetes drug. Alogliptin is a dipeptidyl peptidase IV (DPP-4) inhibitor that stimulates the release of insulin. Alogliptin was approved in the stand-alone form (as Nesina), and in combination with metformin (as Kazano), and pioglitazone (as Oseni). Pioglitazone (Actos), a thiazolidinedione, is also made by Takeda, but the Actos patent expired in 3Q 2012, opening the door for generics and competition. Nesina gives the company a new exclusive medicine in the diabetes market. Oseni’s label contains a boxed warning for congestive heart failure.

Genzyme’s Kynamro Approved to Treat Inherited Cholesterol Disorder

On January 29, 2013, FDA approved Kynamro (mipomersen) injection, an orphan drug used to treat patients with a rare type of high cholesterol called homozygous familial hypercholesterolemia (HoFH). HoFH affects about one out of every one million Americans. Kynamro is used with other lipid-lowering drugs and diet to reduce low-density lipoprotein-cholesterol (LDL-C) and other lipids. In a trial of 51 patients, levels of LDL-C fell by 25 percent during the first 26 weeks. Juxtapid (lomitapide) was also approved for HoFH in December 2012. Kynamro has a Boxed Warning on the serious risk of liver toxicity due to elevated liver enzymes and fat accumulation.

Celegene’s Pomalyst Approved for Advanced Multiple Myeloma

On February 8, 2013, FDA approved Pomalyst (pomalidomide) to treat patients with multiple myeloma who have not responded to treatment and have progressed after being treated with at least two prior cancer therapies. Multiple myeloma is a form of blood cancer that primarily affects older adults. Pomalyst, an analogue of thalidomide, is contraindicated in pregnancy and is only available through a restricted distribution program. In trials, over 29 percent of patients treated with Pomalyst plus low-dose dexamethasone had a complete or partial response with a 7.4-month median duration of response. In July 2012, FDA also approved Kyprolis (carfilzomib) to treat multiple myeloma.

Kadcyla Approved Under FDA Priority Review Program

On February 22, 2013, FDA approved Kadcyla (ado-trastuzumab emtansine) as a treatment for HER2-positive, metastatic (late-stage) breast cancer. Kadcyla is intended for patients who were previously treated with trastuzumab, an anti-HER2 therapy, and taxanes, another class of chemotherapy drugs. In a clinical study of 991 patients randomly assigned to receive Kadcyla or lapatinib plus capecitabine, 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.

Osphena Approved to Treat Painful Sex in Women

On February 26, 2013 FDA approved Osphena (ospemifene), an oral estrogen agonist/antagonist with selective tissue effects, to treat women with moderate to severe dyspareunia (pain during sexual intercourse). Dyspareunia can result in thinner and drier vaginal tissues due to reduced levels of estrogen that occur during menopause. Osphena has estrogenic activity to build vaginal tissues and reduce pain during sex. Generally, when estrogenic products are prescribed in postmenopausal women with an intact uterus, the combined use of a progestin should be considered to reduce the risk of endometrial cancer. Women with unexplained vaginal bleeding should see a doctor.

FDA Approves Lymphoseek to Map Lymph Nodes in Cancer

On March 13, 2013, FDA approved Lymphoseek (technetium Tc 99m tilmanocept) Injection, a radioactive diagnostic imaging agent that helps doctors locate lymph nodes in patients with breast cancer or melanoma who are undergoing surgery to remove tumor-draining lymph nodes. Lymphoseek is an imaging drug that helps locate lymph nodes; it is not a cancer imaging drug.

Lymphoseek was the first new agent for lymph node mapping to be approved in more than 30 years. Other FDA-approved drugs used for lymph node mapping include sulfur colloid (1974) and isosulfan blue (1981).

Dotarem Okayed for Central Nervous System Imaging

On March 20, 2013 FDA approved Dotarem (gadoterate meglumine) for use in magnetic resonance imaging (MRI) of the brain, spine and associated tissues in patients 2 years and older. Dotarem is a gadolinium-based contrast agent that helps radiologists see abnormalities on images of the central nervous system (CNS). Dotarem carries a boxed warning about the risk of rare nephrogenic systemic fibrosis (NSF) in patients with kidney disease, characterized by pain, skin thickening, and internal organ fibrosis. There is no known treatment for NSF; approved labeling describes ways to minimize the NSF risk. Dotarem is the seventh gadolinium-based CNS MRI contrast agent approved by the FDA.

Tecfidera FDA-Approved for Multiple Sclerosis

On March 27, 2013, the FDA approved Tecfidera (dimethyl fumarate), also known as BG-12, an oral, twice daily Nrf2 pathway activator for the treatment of relapsing-remitting multiple sclerosis. Multiple sclerosis (MS) is a central nervous system disease that disrupts communication between the brain and other parts of the body. MS, which results in muscle weakness and difficulty with coordination and balance, affects close to 400,000 people in the U.S. Other oral competitors include Aubagio (fingolimod) and Gilenya (teriflunomide). Tecfidera may decrease white blood cell (WBC) counts; baseline and annual WBC counts are recommended.

Invokana: New First-in-Class Type 2 Diabetes Treatment Approved

The first in a new class of type 2 diabetes drugs was FDA-approved on March 29, 2013 - Invokana (canagliflozin) is an oral tablet indicated to improve blood sugar control in conjunction with diet and exercise in patients with type 2 diabetes. Invokana is a sodium-glucose co-transporter 2 (SGLT2) inhibitor that blocks the reabsorption of glucose (sugar) by the kidneys and increases glucose excretion in the urine. An improvement in hemoglobin A1c and fasting blood sugar levels in trials of more than 10,000 patients secured FDA approval. However, the FDA is requiring five post-approval drug studies from Janssen to assess safety outcomes.

Breo Ellipta: Approved for Chronic Obstructive Pulmonary Disease

On May 10, 2013, FDA approved Breo Ellipta (fluticasone furoate and vilanterol inhalation powder) for the once-daily, maintenance treatment of chronic obstructive pulmonary disease (COPD), including chronic bronchitis and/or emphysema. It is also approved to reduce exacerbations of COPD. Breo Ellipta is a combination of fluticasone furoate, an inhaled corticosteroid, and vilanterol, a long-acting beta2-adrenergic agonist (LABA). Breo Ellipta decreases inflammation and relaxes airway muscles to improve breathing in COPD, a chronic lung disease that worsens over time. Cigarette smoking is the leading cause of COPD and is the third leading cause of death in the U.S.

FDA Approves Xofigo for Advanced Prostate Cancer

In May 2013, the FDA approved Xofigo (radium Ra 223 dichloride) to treat men with late-stage, castration-resistant prostate cancer that has spread only to the bones. Xofigo was evaluated in a single trial of 809 men with castration-resistant prostate cancer. The study was designed to measure overall survival. Men receiving Xofigo lived a median of 14 months compared to a median of 11.2 months for men receiving placebo. Xtandi (enzalutamide), approved in August 2012, has also been shown to extend the survival of men with metastatic prostate cancer. In 2013, it was estimated that 239,000 men were diagnosed with prostate cancer and roughly 30,000 would die.

FDA Approves Two Skin Cancer Drugs with Companion Genetic Test

On May 29, 2013 FDA approved GlaxoSmithKline’s Tafinlar (dabrafenib), an oral BRAF inhibitor, and Mekinist (trametinib), an oral MEK inhibitor, for patients with metastatic (advanced) or unresectable (cannot be removed by surgery) melanoma. Tafinlar and Mekinist have been approved as single agents. In clinical trials, patients who took Tafinlar or Mekinist had a delay in tumor growth that was either 2.4 months or 3.3 months later, respectively, than those on other chemotherapy. FDA also approved the companion THxID BRAF genetic test to determine the mutation type. The National Cancer Institute estimates about 9,500 Americans died from melanoma in 2013.

Boehringer Ingelheim’s Gilotrif Approved for Lung Cancer

Gilotrif (afatinib), a kinase inhibitor indicated for patients with late-stage (metastatic) non-small cell lung cancer (NSCLC), was FDA-approved after a priority review on July 12, 2013. Gilotrif is used in patients whose tumors express epidermal growth factor receptor (EGFR) gene mutations, as detected by an FDA-approved test. In clinical trials, patients receiving Gilotrif had a delay in tumor growth (progression-free survival) that was 4.2 months longer than those receiving the chemotherapy drugs pemetrexed and cisplatin -- there was no difference in overall survival time. In May 2013, the FDA also approved Tarceva (erlotinib) for first-line treatment of patients with NSCLC.

FDA Approves GSK's Tivicay to Treat HIV Infection

On August 12, 2013, the FDA approved GlaxoSmithkline’s Tivicay (dolutegravir), an oral treatment for HIV-1 infection. Tivicay, an integrase inhibitor, interferes with an enzyme necessary for HIV to multiply. Tivicay was evaluated in four trials with over 2,500 participants who received 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. Tivicay is also approved for children ages 12 years and older weighing at least 88 pounds who have not previously taken other integrase strand transfer inhibitors.

Brintellix Approved for Major Depression

On September 30, 2013 FDA approved Brintellix (vortioxetine) to treat adults with major depressive disorder (MDD). Brintellix’s proposed mechanism of action is inhibition of serotonin (5-HT) reuptake. Brintellix is also an agonist at 5-HT1A receptors, a partial agonist at 5-HT1B receptors and an antagonist at 5-HT3, 5-HT1D and 5-HT7 receptors. Brintellix was shown effective compared to placebo in six adult MDD clinical trials, and an additional study that showed Brintellix decreased the likelihood of depression recurrence. More than 7,500 total patients aged 18 to 88 years old were enrolled in clinical trials. The most common side effects included nausea, constipation and vomiting.

Pfizer’s Duavee Okayed for Hot Flashes and Osteoporosis in Menopause

Duavee (conjugated estrogens/bazedoxifene) was approved by the FDA on October 3, 2103 to treat menopause-related hot flashes and for osteoporosis prevention in women with a uterus. Duavee is an estrogen agonist/antagonist and conjugated estrogen combination. Duavee uses bazedoxifene, instead of a progestin, to help protect against uterine hyperplasia that may result from estrogen-alone treatment. Women taking the drug had 74 percent fewer moderate-to-severe hot flashes after 12 weeks of treatment, compared with a 47 percent reduction among women who took a placebo. Blood clots and uterine cancer are a risk with estrogen use.

Bayer’s Adempas Approved for Pulmonary Hypertension

Pulmonary hypertension is a devastating, life-threatening condition caused by excessively high blood pressure in the lung arteries that puts stress on the heart. On October 8, 2013 Adempas (riociguat) was FDA-approved for patients with chronic thromboembolic pulmonary hypertension (CTEPH) and for patients with pulmonary arterial hypertension (PAH). Adempas is an oral guanylate cyclase stimulator that helps to relax arteries, increase blood flow, and decrease blood pressure, allowing greater capacity for exercise and walking. Female patients can receive the drug only through the Adempas REMS program due to possible fetal risk.

Actelion’s Opsumit Now Approved for Pulmonary Arterial Hypertension

On October 18, the FDA approved Opsumit (macitentan) for the treatment of pulmonary arterial hypertension (PAH), a chronic and serious lung disease that can lead to a required lung transplantation or death. Opsumit is classified as a dual endothelin receptor antagonist, and is in a similar drug class as Tracleer (bosentan) and Letairis (ambrisentan). Clinical trials spanned two years with over 740 patients receiving active drug or placebo. Opsumit was found to be effective in delaying disease progression including a decline in the ability to exercise, worsened PAH symptoms or added medication. Opsumit is in a restricted access program due to a risk for birth defects in this class.

Vizamyl Approved to Detect Beta Amyloid in Dementia

Dementia is associated with diminishing brain functions such as memory, judgment, language and complex motor skills. By 2025, close to 7 million people over the age of 65 years are predicted to have Alzheimer’s disease (AD) -- a 40 percent increase from the 5 million seniors currently affected. On October 25, 2013 the FDA approved Vizamyl (flutemetamol F 18 injection), a radioactive diagnostic drug for use with positron emission tomography (PET) imaging of the brain in adults being evaluated for AD and dementia. Vizamyl is the second diagnostic drug approved for this use; in 2012, FDA also approved Amyvid (florbetapir F 18 injection) .

Gazyva: First Designated Breakthrough Drug to Receive FDA Approval

On November 1, 2013, FDA approved Gazyva (obinutuzumab) for use in combination with chlorambucil to treat patients with previously untreated chronic lymphocytic leukemia (CLL). CLL is a slow-growing blood and bone marrow disease. Gazyva is a monoclonal antibody that attacks blood cells called B-cells, the cause of common blood cancers. In clinical studies that compared the use of Gazyva with chlorambucil to chlorambucil alone in participants with previously untreated CLL, the combined treatment lead to a significant improvement in progression free survival: an average of 23 months compared with 11.1 months with chlorambucil alone.

FDA Approves Sunovion’s Aptiom for Seizure Control

Epilepsy is a brain disorder caused by abnormal or excessive activity in the brain’s nerve cells. On November 8, 2013 the FDA approved Aptiom (eslicarbazepine acetate) as an add-on medication to treat partial seizures associated with epilepsy. In clinical trials in over 1,400 patients with partial-onset seizures uncontrolled by one to three other antiepileptic drugs (including carbamazepine, lamotrigine, valproic acid and levetiracetam), Aptiom led to significant reductions in standardized seizure frequency versus placebo. Common side effects with Aptiom include dizziness, drowsiness, nausea, headache, double-vision, vomiting, fatigue and loss of coordination.

Imbruvica: Breakthrough Therapy FDA-Approved for Mantle Cell Lymphoma

Mantle cell lymphoma (MCL) is a rare and aggressive type of blood cancer - a form of non-Hodgkin lymphoma - and represents about 6 percent of all non-Hodgkin lymphoma cases in the U.S. MCL spreads rapidly and by the time it is diagnosed it has usually spread to the lymph nodes, bone marrow and other organs. On November 13, 2013 FDA approved Imbruvica (ibrutinib), a new treatment for patients with MCL who have received at least one prior therapy. Imbruvica was evaluated in a study of 111 participants. Of those who took the drug daily, 66 percent had their cancer shrink or disappear. Velcade (bortezomib) and Revlimid (lenalidomide) are also approved for MCL treatment.

Valeant’s Luzu Cream Approved for Skin Fungal Infections

On November 14, 2013, FDA approved Luzu (luliconazole) Cream, 1%, for the adult treatment of skin fungal infections, including athlete's foot in between toes (interdigital tinea pedis), jock itch (tinea cruris), and ringworm (tinea corporis), caused by the organisms Trichophyton rubrum and Epidermophyton floccosum. Luzu is applied in a one-week, once-daily treatment regimen for tinea cruris and tinea corporis. Other topical antifungal products, such as econazole, clotrimazole or ketoconazole require a 2-4 week treatment period. However, treatment with Luzu for interdigital tinea pedis is only approved with a two-week, once-daily treatment.

Janssen’s Olysio Approved to Treat Hepatitis C Virus

On November 22, 2013, the FDA approved Olysio (simeprevir) for hepatitis C virus (HCV) genotype 1 infected adults with compensated liver disease, including cirrhosis. Olysio is a once-a-day, 150-milligram oral dose, given for 12 weeks, used in combination with peginterferon and ribavirin (PR). Peginterferon and ribavirin are dosed for 24 or 48 weeks based on prior treatment and history. Olysio plus PR effectiveness is reduced in patients with genotype 1a Q80K - testing is available for this genotype and alternate therapies may be needed. Olysio works by blocking the protease enzyme that enables the hepatitis C virus to replicate in host cells.

Sovaldi: Breakthrough Therapy Approved for Hepatitis C Virus

On December 6, 2013 the FDA approved Gilead’s Sovaldi (sofosbuvir) to treat chronic hepatitis C virus (HCV) infection. Sovaldi is the first oral drug of a new paradigm of hepatitis C treatment that has demonstrated safety and efficacy, in combination with ribavirin, to treat certain genotypes of HCV infection without the need for co-administration of interferon alfa. The use of interferon is often associated with intolerable side effects such as flu-like symptoms, anemia, and nausea. However, for genotype 4, interferon plus ribavirin will still be needed with Sovaldi. In November, the FDA also approved Olysio (simeprevir), a once-daily protease inhibitor for HCV.

FDA Approves Anoro Ellipta for Chronic Obstructive Pulmonary Disease

Cigarette smoking is the leading cause of COPD and is the third leading cause of death in the United States. On December 18, 2013, FDA approved Anoro Ellipta for the once-daily, long-term maintenance treatment of chronic obstructive pulmonary disease (COPD). Anoro combines two inhaled COPD bronchodilator agents, umeclidinium bromide (UMEC), a long-acting muscarinic antagonist and vilanterol (VI), a long-acting beta2 agonist. VI is also found in a once-a-day COPD combination with fluticasone furoate (Breo). Anoro is administered using the Ellipta inhaler (also used for Breo) and is developed by GlaxoSmithkline and Theravance.

Finished: Flashback: FDA Drug Approvals 2013

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