The first few months of 2025 brought hope to the rare disease community. Food and Drug Administration leadership promised transparency and increased use of the accelerated approval pathway to deliver timely treatments to those in urgent need.

However, those hopes have been dimmed this summer as the agency has issued Complete Response Letters blocking approval of drugs for patients with terminal diseases, even when good safety data exists and patients in trials are showing meaningful benefit. This was the case with Ultragenyx’s gene therapy (UX111), which has nine years of robust data showing safety and benefit for those suffering from the fatal neurodegenerative disease Sanfilippo syndrome Type A.

It’s left those suffering from diseases like Duchenne muscular dystrophy, advanced cancers, and Sanfilippo syndrome wondering what’s next.

That’s why, in a letter delivered earlier this month, a coalition of patient advocacy organizations supported by more than 43,000 signatories called on FDA Commissioner Dr. Martin Makary and agency leaders to prioritize the advancement of promising therapies for Sanfilippo syndrome, Barth, and Hunter syndrome.

In the world of rare illnesses and late-stage cancers, any unnecessary delay can harm patients and cast aside future development of transformative therapies because the company may no longer have enough financing to wade through protracted regulatory timelines. And even when drug companies are able to weather the delays, many patients will have irreversibly deteriorated or died because an approved treatment was not available in time.

Drug development delays affect real families. Our daughter, Eliza, was diagnosed with

© The Hill