#415: Achondroplasia Treatment Advances with Dr. Ravi Savarirayan

Please note this episode is only available on our website, DNAToday.com under Episode 415, and access is limited to those in the United States.

In just a few years, achondroplasia treatment has expanded from no approved precision medicines to multiple approaches targeting the condition’s underlying biology.

In this episode, we continue our conversation with Dr. Ravi Savarirayan, a global leader in skeletal dysplasia care and a lead investigator across multiple achondroplasia clinical development programs. Dr. Savarirayan previously joined us on Episode 401, where we explored the first approved precision treatment for achondroplasia and international guidance for its use.

This time, we take a broader look at the rapidly evolving achondroplasia treatment landscape, including how different therapeutic approaches target FGFR3 signaling, how researchers interpret clinical trial outcomes, and what results from the Phase 3 PROPEL 3 study of investigational infigratinib could mean for children and families.

We also discuss why treatments cannot be reliably compared using results from separate clinical trials, the importance of studying outcomes beyond growth, and the questions that remain about long-term safety, functional benefits, treatment selection, and switching therapies.

Thank you to BridgeBio for sponsoring this episode.

Episode Discussion Topics

How the achondroplasia treatment landscape has evolved in the last seven years

How achondroplasia affects more than height

How current and investigational therapies target FGFR3 signaling in different ways

What annualized growth velocity, height Z-scores, and body proportionality reveal in clinical trials

Key findings from the Phase 3 PROPEL 3 study of investigational infigratinib