RARE DISEASE WEEK EXTRAVAGANZA!
This Rare Disease Day, February 28, was historic––for the first time in history, FA has a treatment! The FDA notified Reata Pharmaceuticals that their New Drug Application for omaveloxolone, now SKYCLARYS, was approved.
It took many stakeholders to make this possible, including patients, FA families and friends, clinicians, researchers, industry partners, and regulators. Additionally, FA was the recipient of the incredible work by our Congressional Members and their staffers in bolstering this outcome.
The Orphan Drug Act, celebrating 40 years this year, began the legislative path only to be strengthened by PDUFA I-VII, 21st Century Cures Act, and Act for ALS, to name a few. The flexibility Congress granted to the FDA when reviewing a rare disease drug candidate permitted them to consider one clinical trial with confirmatory evidence, in this case, a comparison to years of natural history data, and enabled access to a treatment years earlier than would have been possible under the traditional model. These legislative efforts were instrumental in reaching this important milestone and would not have been possible without patient advocates working with Congressional Members.
As the FA community celebrated our first drug approval on Rare Disease Day, FARA also continued the important advocacy work toward more approved treatments. FARA, and many in the FA community, joined thousands of stakeholders throughout the world in raising awareness, developing Congressional relationships, and supporting legislation that will continue to expedite drug approvals.