Question · Q4 2025
Akash Kashi asked if Palynziq's upcoming PDUFA for adolescents with PKU is something to pay attention to. He also inquired about the gross-to-net for Sephience, specifically if the 15%-25% range previously mentioned should be expected this year.
Answer
Matthew Klein, CEO, stated that even with a potential label expansion for Palynziq, Sephience is expected to become the first-line treatment due to its oral convenience, strong safety, and efficacy profile, contrasting with Palynziq's administration challenges and titration. Pierre Gravier, CFO, clarified that Sephience's gross-to-net is currently at the lower end of the 15%-25% range and is expected to increase slowly and steadily over multiple quarters, not reaching the top end this year.
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