FDA Update: May 2017

May 9, 2017
Express Scripts Office of Clinical Evaluation and Policy tracks some recent updates to the drug pipeline.

New Pill Bottle

During April, the U.S. Food and Drug Administration (FDA) took several important actions. Key updates as monitored by Express Scripts’ Emerging Therapeutics team include:

Austedo Approved for Chorea Associated with Huntington’s Disease

On April 3, 2017, Teva received approval from the FDA for Austedo™ (deutetrabenazine) to treat chorea (involuntary movements) associated with Huntington’s disease (HD). A progressive, neurodegenerative condition, HD affects about 35,000 Americans. Symptoms generally begin after age 30 and the life expectancy is 10 years to 20 years after diagnosis. The recommended starting dose for Austedo is 6mg once daily – increased after one week to 12mg per day; after another week to 18mg per day; and then by weekly 6mg/day increments to a maximum dose of 48mg per day divided into two 24mg doses. Teva plans on launching Austedo within the by mid-May. Full prescribing information can be found here.

Sovaldi and Harvoni Approved for Pediatric Hepatitis C

On April 7, 2017, FDA approved the use of Gilead’s Sovaldi® (sofosbuvir) and Harvoni® (ledipasvir/sofosbuvir) for treating hepatitis C virus (HCV) infections in patients as young as 12 years of age, or at least 77 pounds (35kg). Both are oral direct-acting antiviral drug regimens for treating HCV infections. Harvoni can be used in children who have genotype 1, 4, 5 or 6 chronic HCV infections. Sovaldi is indicated, in combination with ribavirin, for pediatric patients with genotype 2 or 3 chronic HCV infection. According to the Centers for Disease Control and Prevention (CDC), an estimated 23,000 to 46,000 children in the U.S. are infected with HCV, usually at birth.

Ingrezza Approved for Tardive Dyskinesia

On April 11, 2017, Neurocrine Biosciences received approval from FDA for Ingrezza™ (valbenazine) capsules for the treatment of adults with tardive dyskinesia (TD). Characterized by abnormal, involuntary movements, TD affects as many as 500,000 patients in the U.S. The initial dose for Ingrezza is 40mg once daily. After a week, it can be increased to the recommended dose of 80mg once daily. Complete prescribing information is available here.

Competitors to Advair Launched

On April 20, 2017, Teva launched its AirDuo™ RespiClick®, a dry-powder inhaler that combines fluticasone propionate and salmeterol. Teva also released its authorized generic (AG) to AirDuo RespiClick at the same time. Both products are indicated for the maintenance treatment of asthma in patients who are 12 years of age or older. Recommended dosing is one inhalation twice daily. While they contain the same active components as Advair Diskus® (fluticasone propionate/salmeterol – GlaxoSmithKline), neither is A-rated, or directly substitutable, with Advair. Prescribing information for Teva’s brand product can be found here.

In March 2017, Mylan received a complete response letter, meaning that it must provide additional information before the FDA will review its proposed Advair Diskus® generic. Although another generic – from Hikma/Vectura – is under review with an action date of May 10, 2017, FDA is not expected to approve it at that time. True generics to Advair Diskus may not reach the market until sometime in 2018.

Renflexis, a Biosimilar to Remicade, Approved

On April 21, 2017, the FDA approved Merck and Samsung Bioepis’ Renflexis™ (infliximab-abda), a biosimilar to Janssen’s Remicade®. Renflexis was approved for all Remicade-approved indications, except pediatric ulcerative colitis. It will be used for treating patients with rheumatoid arthritis, adult ulcerative colitis, plaque psoriasis, psoriatic arthritis, ankylosing spondylitis, adult Crohn’s disease and pediatric Crohn’s disease. The dose of Renflexis varies depending on the indication. Launch information is not available at this time. Full prescribing information is available here.

Generics to Vytorin Launched

The FDA has approved multiple AB-rated generics to Merck’s Vytorin® (ezetimibe/simvastatin), a fixed-dose combination product approved to lower high blood cholesterol. Ezetimibe (Zetia®, generics) is a unique drug that inhibits the absorption of cholesterol by the small intestine. Simvastatin (Zocor®, generics) belongs to a class of cholesterol-lowering medications known as HMG-CoA reductase inhibitors, or “statins”. Generic exclusivity was not granted and shipments of the generics began on April 26, 2017, from at least three companies, Dr. Reddy’s Laboratories, Impax and Teva.

Brineura Approved for Late Infantile Neuronal Ceroid Lipofuscinosis type 2

The FDA approved Brineura™ (cerliponase alfa) on April 27, 2017. It is an enzyme replacement therapy for children at least three years old who have a rare form of Batten disease known as late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Only about 20 infants per year are born with CLN2 in the United States. Only about 20 infants per year are born with CLN2 in the United States. Symptoms typically begin between the ages of two and four years, patients gradually lose function, including speech and most cases are fatal before the age of 12 years. As the first drug FDA approved to treat CNL2, Brineura helps affected children maintain the ability to crawl or walk. It is administered under sterile conditions directly into cerebrospinal fluid of the brain using an implanted reservoir and catheter. The recommended dose is 300mg infused once every two weeks. BioMarin Pharmaceuticals plans to begin sales within six weeks. Distribution will be through hospitals only. Prescribing information is available here.

Rydapt Approved for Acute Myeloid Leukemia and Systemic Mastocytosis

On Apr. 28, 2017, Novartis received approval from the FDA for Rydapt® (midostaurin), in combination with chemotherapy, for the first-line treatment of adults with FMS-like tyrosine kinase 3 mutation-positive (FLT3+) acute myeloid leukemia (AML) as detected by an FDA-approved test. LeukoStrat® CDx FLT3 Mutation Assay was approved as a companion diagnostic to detect the FLT3 mutation in patients with AML. Rydapt is the only targeted therapy approved to treat patients with newly diagnosed FLT3+ AML – about 7,000 new cases per year. It also is approved to treat adults with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN) or mast cell leukemia (MCL). The recommended dose for AML is 50mg twice daily with food. For ASM, SM-AHN and MCL, the recommended dose is 100mg twice daily with food. Novartis plans on launching Rydapt in the near future at an estimated annual WAC of $180,000. It will be available through open distribution. Full prescribing information can be found at here.

Tymlos Approved to Treat Osteoporosis

Tymlos™ (abaloparatide) was approved by the FDA on April 28, 2017, to treat postmenopausal women who have osteoporosis and who also have a high risk of fracture. A human parathyroid hormone related peptide (PTHrP [1-34]) analog, its recommended dose is one 80mcg subcutaneous injection into the abdomen each day. In studies, using Tymlos decreased the incidence of new fractures in the spine and other bones. Additionally, it increased bone mineral density (BMD) and a specific blood marker indicating new bone formation. A boxed warning on its labeling warns that using Tymlos may increase the risk of osteosarcoma (bone cancer). Using it for longer than a total of two years is not recommended and it is not yet approved for treating osteoporosis in men. The manufacturer, Radius Health, plans to launch Tymlos in late-May 2017, through open distribution. Forteo® (teriparitide – Eli Lilly), the only other parathyroid hormone on the U.S. market, has similar dosing recommendations and warnings for postmenopausal women. Available since 2002, Forteo does have additional indications to treat men with osteoporosis. Tymlos’ complete prescribing information is available here.

Alunbrig Approved for Non-Small Cell Lung Cancer

Takeda Oncology’s Alunbrig™ (brigatinib) received accelerated FDA approval on April 28, 2017. An oral tyrosine kinase inhibitor (TKI), Alunbrig is indicated for treating patients who have metastatic anaplastic lymphoma kinase-positive (ALK+) non-small cell lung cancer (NSCLC) and who have progressed on or are intolerant to Xalkori® (crizotinib – Pfizer/EMD Serono). NSCLC, the most common form of lung cancer, affects around 190,000 new patients in the United States each year. However, only about 2% to 8% of NSCLC patients also have ALK mutations. For ALK+ patients whose cancer returns after initial treatment, approximately 70% have metastases in their brains. Recommended dosing for Alunbrig is 90mg per day for one week, followed by 180mg per day, if the patient can tolerate the higher dose. Takeda Oncology plans to launch Alunbrig in mid-May. It will be available through a limited network of specialty pharmacies that includes Accredo. Complete prescribing information is available here.

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