The FDA has approved metreleptin (Myalept) to treat patients with congenital or acquired generalized lipodystrophy.
The leptin analog injection is indicated for use along with diet to relieve symptoms of this condition, in which patients are born with little to no fat tissue or generally lose fat tissue over time.
Fat tissue is necessary to produce leptin, so lipodystrophy leads to leptin deficiency and patients with the disorder may develop complications including insulin resistance, diabetes and high triglycerides, the FDA said in a press release.
The approval was based on results from an open-label, single-arm study involving 48 patients with congenital or acquired generalized lipodystrophy with complications of leptin deficiency. Patients treated with metreleptin experienced reductions in HbA1c, fasting glucose and triglycerides, according to the FDA.
Drug manufacturer Bristol-Myers Squibb was also pursuing an indication for partial lipodystrophy occurring with hypertriglyceridemia, uncontrolled diabetes and hepatic steatosis, but an FDA advisory committee on endocrinologic and metabolic drugs voted against the indication, stating lack of evidence.
Metreleptin is not approved for use in patients with HIV-related lipodystrophy or in patients with metabolic disease, including diabetes mellitus and hypertriglyceridemia, without concurrent evidence of generalized lipodystrophy, the agency clarified.
The two most prominent potential side effects linked to the metreleptin were T-cell lymphoma and immunogenicity.The FDA warned of the potential for patients to develop neutralizing antibodies that could result in severe infection or loss of treatment effectiveness.
The agency also noted T-cell lymphoma developed in both treated and placebo patients, so clinicians should “carefully consider the benefits and risks of treatment with Myalept in patients with significant hematologic abnormalities and/or acquired generalized lipodystrophy.”
Clinicians must complete a Risk Evaluation and Mitigation Strategy Program (REMS) to be able to prescribe metreleptin, and the FDA is requiring Bristol Myers Squibb to complete seven post-marketing studies to assess immunogenicity and track serious side effects.
Amylin Pharmaceuticals, a subsidiary of Bristol Myers Squibb, will market the drug.