FDA reviewers say metreleptin effective for subgroup

FDA reviewers said Myalept metreleptin from the Amylin Pharmaceuticals Inc. subsidiary of Bristol-Myers Squibb Co. (NYSE:BMY) is effective in a subgroup of lipodystrophy patients and backed the company's proposed REMS to educate prescribers about the potential risk of developing neutralizing antibodies and lymphoma. The reviewers said metreleptin led to a benefit "unlikely to have been achieved spontaneously" in patients with generalized lipodystrophy with severe insulin resistance resulting in diabetes and/or severe hypertriglyceridemia that is not adequately controlled with other therapies. The BLA is based on 72 patients in two open-label trials sponsored by NIH, as well as 28 patients enrolled in the Bristol-Myers-sponsored FHA101 trial. The reviewers did note that it is "unclear if a subgroup of patients with partial lipodystrophy can be clearly identified" that would benefit from the recombinant form of human leptin.

The reviewers also said there are serious risks with metreleptin, including the risk of T cell lymphoma and neutralizing antibodies. The reviewers backed implementation of a REMS to monitor for development of adverse events. Bristol-Myers has proposed a REMS that would include a physician and pharmacy certification component, as well as a prescriber education program and a metreleptin safety registry. ...