Repurposed Drugs Offer Hope and Momentum for Rare Disease Treatments

While at the 2025 World Orphan Drug Congress (WODC) in Boston, CTF CEO Annette Bakker, PhD, spoke with CheckRareabout the urgent need and growing opportunity to repurpose shelved drugs for rare diseases like NF.

When small biotech companies are acquired, promising early-stage drugs are often sidelined due to shifting business priorities, not because they lack safety or efficacy. "These drugs don't just disappear," Dr. Bakker explains. "They sit on shelves. And with the right systems in place, we can take them into clinical trials for rare diseases like NF."

The path isn't easy-accessing rights, finding usable data, and convincing leadership to revisit old assets are all major hurdles. But progress is happening. Two repurposed drugs received FDA approval.

With CTF's leadership, the field is gaining momentum. A preclinical hub now helps de-risk promising drugs, platform trials are streamlining testing, and nearly 70 clinical trials are currently underway, many sparked by this strategy.

Repurposed drugs aren't just full of promise. They're fueling real progress.

Watch the full interview with Dr. Annette Bakker on CheckRare.