Episode 10 — When a rare disease treatment fails approval, what happens next?

Rare in Common: the podcast

This episode is the second in a special series recorded live at the 7th annual Global Genes RARE Patient Advocacy Summit—and an important follow-up to Episode 4, “Taking on Rare as a Family,” in which Justin Hastings spoke about living with familial chylomicronemia syndrome (FCS). In August 2018, Waylivra® (volanesorsen sodium) was slated to receive final approval from the Food and Drug Administration (FDA) and become the first-ever therapy specifically for the treatment of FCS. Instead, Akcea Therapeutics, the company that makes Waylivra, received a complete response letter (CRL) from the FDA, rejecting approval of the drug, causing a wave of surprise, shock, and upset for everyone affected. Hear how the FCS community is responding and how it’s working together—stronger than ever—to help Waylivra receive approval in the future.