FDA approves Myalept, the first drug to treat a rare metabolic disease

The US Food and Drug Administration has approved Myalept (metreleptin for injection) as replacement therapy to treat the complications of leptin deficiency, in addition to diet, in patients with congenital generalized or acquired generalized lipodystrophy.

The drug,, the first to be approved by the FDA for this rare disorder, was developed by Amylin Pharmaceuticals, which was acquired by US drug major Bristol-Myers Squibb (NYSE: BMY) in 2012 for $5.3 billion.

However, as a result of Anglo-Swedish drug major AstraZeneca’s (LSE: AZN) recent acquisition of B-MS’s franchised diabetes business, which it completed earlier this month, Myalept is now the property of the UK-based firm AstraZeneca, which said it is working to complete the transfer of the Biologics License Application (BLA) for Myalept from B-MS, adding that it is working to make the drug available to patients as soon as possible in the US