Episode 10 — When a rare disease treatment fails approval, what happens next? by Rare in Common published on 2018-11-26T20:43:11Z 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. Genre Storytelling