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Monthly News Roundup - February 2014

FDA Approves Amylin’s Myalept for Rare Metabolic Disease

The U.S. Food and Drug Administration (FDA) has approved the orphan drug Myalept (metreleptin for injection) as replacement therapy to treat the complications of leptin deficiency, in addition to diet, in patients with congenital or acquired generalized lipodystrophy. Patients with generalized lipodystrophy often develop severe insulin resistance, diabetes, or high levels of triglycerides (hypertriglyceridemia) that can lead to pancreas inflammation. In clinical trials, Myalept led to reductions in HbA1c (a measure of blood sugar control), fasting glucose, and triglycerides. Due to the risk of neutralizing antibodies and lymphoma, Myalept is only available through the Myalept Risk Evaluation and Mitigation Strategy (REMS) Program.

Northera FDA-Approved for Neurogenic Orthostatic Hypotension

The U.S. Food and Drug Administration (FDA) has approved Chelsea Therapeutic’s Northera (droxidopa) capsules for the treatment of neurogenic orthostatic hypotension (NOH). NOH is a rare and often disabling drop in blood pressure upon standing that is associated with Parkinson's disease, multiple-system atrophy, and pure autonomic failure. This severe drop in blood pressure can often limit the ability to complete daily activities that require walking or standing. Northera has a boxed warning about the risk of increased blood pressure while lying down. Patients must keep their head and upper body elevated when sleeping and lying down, and blood pressure should be monitored.

Vimizim Approved for Rare Morquio A Syndrome

The U.S. Food and Drug Administration has approved BioMarin Pharmaceutical’s Vimizim (elosulfase alfa), the first FDA-approved treatment for Mucopolysaccharidosis Type IVA (Morquio A syndrome). Mucopolysaccharidosis Type IVA is a rare lysosomal storage disease that can lead to problems with bone development, growth and mobility. Vimizin replaces a necessary enzyme called N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Clinical trials with Vimizim involved 176 participants ranging in age from 5 to 57 years. On average, patients treated with Vimizim walked 22.5 meters farther in 6 minutes compared to the patients who received placebo.

Hetlioz Approved For Non-24 Hour Sleep-Wake Disorder in the Blind

The U.S. Food and Drug Administration (FDA) has approved Vanda Pharmaceutical’s Hetlioz (tasimelteon), a melatonin receptor agonist, to treat non-24-hour sleep-wake disorder (non-24) in totally blind individuals. Non-24 is a chronic, circadian rhythm disorder resulting from the misalignment of the internal body clock to the 24-hour day, disrupting the sleep-wake cycle. This is the first FDA approval of a treatment for the disorder. Hetlioz was evaluated in a clinical study of 104 completely blind people. Reported side effects included headache, elevated liver enzymes, unusual dreams and infections of the upper respiratory or urinary tract.

FDA Approves Another Low Dose NSAID: Iroko’s Tivorbex

The U.S. Food and Drug Administration (FDA) has approved the 20 mg and 40 mg strengths of Tivorbex (indomethacin), a nonsteroidal anti-inflammatory drug (NSAID) for the treatment of mild to moderate acute pain in adults. Tivorbex capsules were approved at dosage strengths that are 20 percent lower than currently marketed indomethacin and contain submicron particles that provide an increased surface area, leading to faster dissolution. Lower doses may help to prevent serious NSAID side effects such as heart attacks, strokes, stomach bleeds, and kidney damage. Iroko Pharmaceutials' Zorvolex (diclofenac), also a lower dose NSAID, was approved in October 2013.

Posted: February 2014