First Drug Approved for Rare Lipid Disorder
The FDA has approved Myalept (metreleptin; Bristol-Myers Squibb) 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. Myalept, a recombinant leptin analog, is the first drug approved for treating the complications associated with congenital or acquired lipodystrophy.
The safety and efficacy of Myalept were evaluated in an open-label, single-arm, study that enrolled 48 patients with congenital or acquired generalized lipodystrophy who also had diabetes, hypertriglyceridemia, and/or elevated fasting insulin levels. Study results showed reductions in HbA1c, fasting glucose, and triglycerides.
Due to the risks associated with neutralizing antibody and lymphoma development, Myalept is available only through the Myalept Risk Evaluation and Mitigation Strategy (REMS) Program. Prescribers and pharmacies must be certified with the program. The FDA is requiring seven studies as part of postmarketing requirements.
Myalept is available as a single vial containing 11.3mg of metreleptin.
For more information call (877) 417-1523 or visit BMS.com.