Myalept gets FDA approval

SILVER SPRING, Md. — The Food and Drug Administration has approved Myalept (metreleptin for injection) to treat complications of leptin deficiency in patients with congenital generalized or acquired generalized lipodystrophy, which is a condition associated with a lack of fat tissue.

Patients with cogenital generalized lipodystrophy are born with little or no fat tissue, and those patients with acquired generalized lipodystrophy will generally lose fat tissue over time. Patients with both types are prone to develop severe insulin resistance at a young age, which may lead to diabetes that is difficult to manage or very high levels of triglycerides in the blood (hypertriglyceridemia) that may lead to inflammation of the pancreas.

“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,” said Mary Parks, M.D., deputy director of the Office of Drug Evaluation II in the FDA’s Center for Drug Evaluation and Research.

Myalept is available only through the Myalept Risk Evaluation and Mitigation Strategy program due to the risks associated with the development of neutralizing antibodies and lymphoma. Under the REMS program, prescribers must be certified with the program by enrolling in and completing training. Pharmacies must also be certified with REMS and only dispense the drug after receipt of the Myalept REMS Prescription Authorization Form for each new prescription.

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