Orphan Product Extensions Now–Accelerating Cures and Treatment

The OPEN Act (S. 1509) establishes an exclusivity extension, which would provide an additional six months of market exclusivity for a drug or biological product approved by the FDA when the product is additionally approved to treat a new indication that is an orphan disease.

Per the EveryLife Foundation for Rare Disease, scientific literature shows that a single targeted drug is likely to have multiple therapeutic uses and that biopharmaceutical companies can repurpose drugs for the treatment of different diseases. Doing so is faster, cheaper, and presents fewer risks than traditional drug development.

The legislation has bipartisan support, sponsored by Sens. Orrin Hatch and Robert Menendez and Reps. Gus Bilirakis and G.K. Butterfield, with 26 total sponsors in the House (9 Democrats, 17 Republicans). It is also supported by 268 patient organizations, including Genetic Alliance, Global Genes, National MPS Society, the National Organization for Rare Disorders, and the Pediatric Cancer Foundation. ASGCT has joined the EveryLife Foundation’s list of supporting organizations.

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