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Monthly News Roundup - April 2017

Takeda Announces FDA Accelerated Approval of Alunbrig

The US Food and Drug Administration (FDA) has approved once-daily oral Alunbrig (brigatinib) for the treatment of patients with anaplastic lymphoma kinase-positive (ALK+) metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib (Xalkori). In the pivotal Phase 2, open-label ALTA study which enrolled 222 patients, the overall response rate after a median follow up of 8 months at the recommended dosing was 53 to 54 percent, and the median duration of response ranged from 11.1 to 13.8 months. The most common adverse reactions (≥25%) with Alunbrig were nausea, diarrhea, fatigue, cough, and headache.

Tymlos Cleared by FDA for Treatment of High-Risk Osteoporosis

Roughly 2 million osteoporotic fractures occur annually in the U.S., and this number is expected to grow to 3 million by 2025. This month, the FDA approved Tymlos (abaloparatide) from Radius Health, a synthetic peptide analog of hPTHrP (human parathyroid hormone-related protein) for the treatment of postmenopausal women with osteoporosis at high risk for fracture. In the ACTIVE trial, Tymlos demonstrated significant reductions in the relative risk of new vertebral (86%) and nonvertebral fractures (43%) compared to placebo. Significant warnings include formation of osteosarcoma (a malignant bone tumor) in animals.

Rydapt Approved for Acute Myeloid Leukemia and Systemic Mastocytosis

Rydapt (midostaurin), a new targeted drug from Novartis, has been FDA-approved for 2 uses: acute myeloid leukemia (AML) in combination with chemotherapy for adults with an FMS-like tyrosine kinase-3 (FLT3) mutation as diagnosed by a special test, and for advanced systemic mastocytosis (SM). Rydapt is an oral kinase inhibitor that works by blocking several enzymes that promote cell growth. AML is an aggressive bone marrow cancer, while systemic mastocytosis (SM) includes several rare blood disorders. Side effects differ slightly between the 2 uses but can include nausea, vomiting, low levels of white blood cells with fever (febrile neutropenia), muscle pain, or upper respiratory tract infection, among others.

FDA Approves Brineura for CLN2 Disease, a Form of Batten Disease

Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease, is a rare inherited disorder that primarily affects the nervous system. CLN2 disease typically begins between the ages of 2 and 4 with symptoms such as language delay, recurrent seizures (epilepsy) and difficulty coordinating movements (ataxia). The FDA has now approved Brineura (cerliponase alfa) injection from BioMarin Pharmaceuticals for this specific form of Batten disease. Brineura is an enzyme replacement therapy for tripeptidyl peptidase-1 (TPP1), and is the first FDA-approved treatment to slow loss of walking ability (ambulation) in symptomatic children 3 years and older with CLN2.

Ingrezza First Approved Treatment for Adults with Tardive Dyskinesia

Tardive dyskinesia (TD) is characterized by uncontrollable, abnormal and repetitive movements of the trunk, extremities and/or face caused by dopamine blockade in the brain, such as occurs with antipsychotics. This month, the FDA approved Neurocrine’s Ingrezza (valbenazine) capsules for the treatment of adults with tardive dyskinesia. Ingrezza, a novel, highly selective vesicular monoamine transporter 2 (VMAT2) inhibitor, is the first product indicated for adults with TD. In studies, Ingrezza led to significant improvement in TD signs and symptoms compared to placebo through six weeks, with continued improvement through 48 weeks of treatment. Somnolence (drowsiness) was the most common side effect.

Renflexis: Second Remicade Biosimilar Gains FDA Approval

The FDA has approved Renflexis (infliximab-abda), a tumor necrosis factor (TNF) blocker from Samsung Bioepis that is biosimilar to Remicade (infliximab). Renflexis, given as an intravenous infusion, is indicated for the treatment of Crohn’s disease, ulcerative colitis, rheumatoid arthritis, ankylosing spondylitis, psoriatic arthritis and plaque psoriasis. The biosimilar must be shown to have no clinically meaningful differences in terms of safety and effectiveness from the reference product Remicade. Only minor differences in clinically inactive components are allowable in biosimilar products. The first biosimilar to Remicade, Pfizer’s Inflectra (infliximab-dyyb) was approved by the FDA in April, 2016.

Posted: April 2017


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