Malvern-based Ocugen has been granted rare pediatric disease designation by the Food and Drug Administration for its experimental gene therapy targeting Stargardt disease, writes John George for the Philadelphia Business Journal.
The move “reaffirms the urgency of providing a therapeutic option to Stargardt patients, who have no FDA-approved treatment available,” said Ocugen CEO Dr. Shankar Musunuri.
However, the primary benefit of the designation remains in limbo. The voucher program expired last year, and Congress has yet to reauthorize the initiative.
It is designed to encourage biotech and pharmaceutical companies to invest their research and development funds in diseases affecting small populations of children.
As part of the program, a company with an approved rare disease product was awarded a voucher for priority review of its subsequent new drug application.
What made these vouchers especially attractive to smaller life science companies strapped for cash is that they can be sold to any drug developer and can fetch prices exceeding $100 million.
Lobbying efforts to reauthorize the voucher program have been underway by several industry trade groups and rare disease patient advocacy organizations, including EveryLife Foundation for Rare Diseases, Global Genes, and the Alpha-1 Foundation.
Read more about Ocugen working on a rare pediatric disease, Stargardt disease, in the Philadelphia Business Journal.
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