FDA Approves Amylin’s Rare-Disease Drug

Amylin Pharmaceuticals’ Myalept has been approved by the FDA as replacement therapy to treat the complications of leptin deficiency, in addition to diet, in patients with congenital generalized or acquired generalized lipodystrophy.

Myalept (metreleptin for injection) is the first approved therapy indicated for treating the complications associated with congenital or acquired generalized lipodystrophy, a condition associated with a lack of fat tissue, CDER’s Mary Parks said Tuesday.

Patients with both types of generalized lipodystrophy often develop severe insulin resistance at a young age and may have diabetes mellitus that is difficult to control or hypertriglyceridemia, which can lead to inflammation of the pancreas, according to the FDA.

The safety and effectiveness of Myalept, an analog of leptin made through recombinant DNA technology, were evaluated in an open-label, single-arm study that included 48 participants with congenital or acquired generalized lipodystrophy who also had developed serious complications.

The trial showed lowered HbA1c, fasting glucose and triglycerides, according to the agency.

Myalept comes with a restrictive risk evaluation and mitigation strategy (REMS) requiring prescribers to be certified in the REMS program. The FDA is also requiring Amylin to conduct seven postmarket studies of the drug, including a long-term prospective observational study of patients treated with Myalept, a study analyzing the drug’s immunogenicity, and an assessment and analysis of spontaneous reports of potential serious risks associated with the use of Myalept.

Eight additional studies are being requested as post-marketing commitments, the FDA said. — Johnathan Rickman

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