FDA unveils new pathway for rare disease treatments
statnews.com • • business
Key Points:
- The Trump administration unveiled detailed FDA guidance for approving personalized medicines designed to target patients' unique genetic mutations.
- This new regulatory approach, called the plausible mechanism pathway, was initially outlined by FDA leaders in a November New England Journal of Medicine article.
- The guidance marks the first formal step toward implementing this pathway, offering essential details for developers of individualized therapies.
- The pathway has received strong support from academics, pharmaceutical companies, and patient advocacy groups as a vital means to advance gene editing treatments and drugs for rare or unique mutations.
