Uncertainty and Inaction in the Wake of a Court Ruling on Orphan Drug Exclusivity — The 2023 NEXT Report

A 2021 court ruling has left the rare drug development world uncertain about future orphan drugs.

Read an excerpt from the 2023 NEXT Report about how this court ruling has affected drug development for rare diseases and orphan drugs:

At the end of September 2021, the U.S. Court of Appeals for the 11th Circuit overturned a lower court ruling in the case of Catalyst Pharms., Inc. v. Becerra, a decision that the U.S. Food and Drug Administration expects to affect the incentives for research and development of medical products for rare diseases.

In June 2019, Catalyst filed a lawsuit against the FDA calling for it to withdraw its approval of the Jacobus drug Ruzurgi for the treatment of Lambert-Eaton Myasthenic syndrome, or LEMS, in pediatric patients. LEMS is a rare autoimmune condition in which the body attacks the junction between nerves and muscles.

Catalyst won FDA approval for its drug Firdapse in late 2018 to treat adults with LEMS and set the annual list price of the drug at $375,000. The FDA then approved Jacobus Pharmaceutical’s Ruzurgi for pediatric patients age 6 to 16. Both Firdapse and Ruzurgi are amifampridine and Catalyst charged that in approving.

The NEXT Report provides an overview of developments across the rare disease landscape and highlights trends in research, diagnosis, development, and treatment, as well as the changing regulatory and financial environment. Read about:

Read the Report