FDA reversals on UniQure, Moderna approvals worry investors
CNBC • • business
Key Points:
- The FDA has recently rejected or discouraged approval for at least eight experimental drugs targeting hard-to-treat diseases, citing concerns over the evidence supporting their applications, including lack of placebo-controlled studies and reliance on biomarkers instead of direct efficacy measures.
- Companies like UniQure and Regenxbio have accused the FDA of reversing previous guidance, creating regulatory uncertainty that is causing investor wariness about the future prospects of treatments for rare diseases.
- Some analysts and investors are closely watching other gene therapy developers such as Dyne Therapeutics, Taysha Gene Therapies, Wave Life Sciences, and Lexeo Therapeutics, whose stocks have declined amid concerns about FDA approval standards.
- The FDA maintains that biomarkers reasonably likely to predict efficacy and non-randomized data can
