News Feature | February 26, 2014

FDA Approves Myalept For Treatment Of Rare Metabolic Disease

Source: Clinical Leader

By Estel Grace Masangkay

The U.S. Food and Drug Administration (FDA) has announced its approval of Myalept (metreleptin for injection) as a replacement therapy for the treatment of complications of leptin deficiency. Myalept is commercialized by Amylin Pharmaceuticals, L.L.C. based in San Diego.  

The federal agency approved use of Myalept, in conjunction to proper diet, in patients with congenital generalized or acquired generalized lipodystrophy. Generalized lipodystrophy is a rare metabolic condition linked to a lack of fat tissue in patients. Congenital generalized lipodystrophy cause patients to have little or no fat tissue from birth. Those with acquired generalized lipodystrophy usually lose fat tissue as time progresses.

Leptin levels are observed to be very low in affected patients since the hormone leptin is produced by fat tissue. Leptin plays a role in regulating food intake and other hormones such as insulin. Myalept works as a replacement therapy for leptin deficiency in patients with lipodystrophy.

Mary Parks, M.D., deputy director of the Office of Drug Evaluation II in the FDA’s Center for Drug Evaluation and Research, said “Myalept is the first approved therapy indicated for treating the complications associated with congenital or acquired generalized lipodystrophy and provides a needed treatment option for patients with this orphan disease.”

The drug’s safety and effectiveness were assessed in an open-label, single arms study involving 48 patients with congenital or acquired generalized lipodystrophy who also had diabetes mellitus, hypertriglyceridemia, and/or elevated levels of fasting insulin. Results showed the drug induced reductions in HbA1c, fasting glucose, and triglycerides. As part of post marketing requirement, the FDA requested seven studies for Myalept. These include a long term prospective observational study of patients who have taken Myalept, an immunogenicity assessment study, and an evaluation and analysis of spontaneous reports of potential risks related to Myalept use. Additionally, eight more studies are being required as post-marketing commitments.

Due to risks associated with the development of neutralizing antibodies and lymphoma, the drug is available only through the Myalept REMS (Risk Evaluation and Mitigation Strategy) Program. Prescribers using Myalept are required to be certified through enrollment and complete training in the program. Likewise, pharmacies dispensing Myalept must be certified and must receive the Myalept REMS Prescription Authorization Form for each new prescription.