US FDA program favorable for rare disease drug developers, says Jefferies

Dyne Therapeutics

04/09/2025 12:39

** Brokerage Jefferies says a new program that aims to expedite approvals for rare disease treatments should bode well for drug developers in this space

** Earlier this week, the U.S. FDA proposed a new process to streamline the approval of drugs targeting rare diseases with very small patient populations, including cases supported by data from single-arm clinical trials

** The brokerage says the decision could provide some upside if the FDA shows flexibility in its eligibility threshold of fewer than 1,000 patients affected by the disease

** Jefferies says companies like BridgeBio Pharma BBIO.O, Sarepta Therapeutics SRPT.O, Dyne Therapeutics DYN.O and Rocket Pharmaceuticals RCKT.O could be "more emboldened" to pursue treatments for diseases with smaller patient population that show a valid mechanism of action

** Up to last close, RCKT down 73.6%, DYN down 42.7% and SRPT down 84.4% YTD

 (Reporting by Christy Santhosh)

 ((Christy.Santhosh@thomsonreuters.com))