Orphan Drug Exclusivity Regulations Will Remain As Is, Says FDA

The FDA said it will keep in place its regulations on orphan drug exclusivity rather than altering them in response to a pivotal U.S. appeals court ruling.

The agency said it plans to continue to tie orphan drug exclusivity to the uses or indications for which the orphan drug was approved rather than considering allowing for more expansive marketing rights.

The seed of the controversy was the FDA’s approval of Catalyst Pharmaceuticals’ amifampridine product Firdapse in 2018 for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) in adults, giving Catalyst exclusive marketing rights for seven years.

In 2019, the agency approved Jacobus Pharmaceuticals’ amifampridine drug Ruzurgi for treatment of LEMS in children. Catalyst sued the FDA claiming orphan drug exclusivity. In 2021, a federal appeals court ruled in the company’s favor and the FDA withdrew its approval of Ruzurgi. The two companies settled their patent issues out of court in July 2022.

But in its notice this week, the agency said it believes that the existing regulations balance “the need to incentivize the development of drugs for rare diseases and conditions with the need to provide patient access to orphan drugs.”