FDA grants approval for Metreleptin

The US Food and Drug Administration (FDA) has approved a priority review for the Biologics License Application for metreleptin.

It is an investigational agent for the treatment of metabolic disorders linked with inherited or acquired lipodystrophy, which is believed to impact a few thousand people throughout the world.

The FDA grants Priority Review to medicines that may offer a treatment option where little or no adequate therapy is available. As part of the Prescription Drug User Fee Act, the FDA aims to carry out the work within an eight month review cycle.

Fred Fiedorek, MD, senior vice president, Head of Development, CV and Metabolics at Bristol-Myers Squibb, said: “The FDA’s granting of Priority Review for the metreleptin BLA highlights the significant unmet medical need for patients affected by lipodystrophy and brings us an important step closer to potentially bringing a treatment option to these patients.

“Developing metreleptin for the treatment of lipodystrophy demonstrates Bristol-Myers Squibb and AstraZeneca’s continued dedication to scientific innovation and global patient care for people impacted by diabetes and related metabolic disorders.”

The primary data supporting the BLA filing are from the National Institutes of Health’s (NIH) ongoing open-label trial in adult and pediatric patients with inherited or acquired LD.

Metreleptin is an investigational recombinant analogue of the human hormone leptin and has obtained orphan designation from the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

It is being reviewed for the treatment of metabolic disorders linked with inherited or acquired lipodystrophy.

Patients with lipodystrophy suffer a loss of fat tissue, including fat under their skin. Due to this, there is a deficit in the hormone leptin and, without enough fat tissue, the body’s system, for regulating energy use falls out of balance.

Bristol-Myers Squibb is aiming to discover, develop and deliver innovative medicines that assist patients beat serious diseases.

Posted by Ben Evans