FDA Approves Orphan Drug Myalept
AstraZeneca announced FDA approved orphan drug Myalept (metreleptin for injection), which is indicated as an adjunct to diet as replacement therapy for the treatment of complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy. Myalept, a recombinant analogue of human leptin, is the first and only treatment approved by the FDA for these patients.
AstraZeneca is working to complete the transfer of the Biologics License Application for Myalept from Bristol-Myers Squibb to AstraZeneca as part of the acquisition of the diabetes alliance assets, including Myalept and Amylin Pharmaceuticals, which was completed on February 1, 2014.
The safety and effectiveness of Myalept for the treatment of complications of partial lipodystrophy or for the treatment of liver disease, including non-alcoholic steatohepatitis, have not been established. Myalept is not indicated for use in patients with HIV-related lipodystrophy or for use in patients with metabolic disease, including diabetes mellitus and hypertriglyceridaemia, without concurrent evidence of congenital or acquired generalized lipodystrophy.
Myalept is for subcutaneous injection only and is available in an 11.3 mg vial that requires reconstitution.
The FDA is requiring seven studies (post-marketing requirements) for Myalept, including a long-term prospective observational study (product exposure registry) of patients treated with Myalept, a study to assess for the immunogenicity (antibody formation) of Myalept, and an assessment and analysis of spontaneous reports of potential serious risks related to the use of Myalept. Eight additional studies are being requested as post-marketing commitments.