A major question arising from the US Food and Drug Administration’s rejection of Intercept Pharmaceuticals Inc.’s new drug application for obeticholic acid (OCA) in non-alcoholic steatohepatitis (NASH) on 29 June was whether the goalposts for approval in the disease, which has yet to see a therapy approved, were being shifted for all companies in the space. However, the early sense from analysts is that the agency was expressing concerns specific to OCA’s risk-benefit profile, rather than about the currently identified surrogate endpoints for NASH.
Intercept CRL Likely About OCA-Specific Concerns, Not NASH Endpoints
The US FDA complete response letter for Intercept’s obeticholic acid raised speculation that the agency might be rethinking approvable endpoints for NASH. But analysts generally think OCA’s unspectacular efficacy data is behind the decision.
More from Complete Response Letters
• By
While CMC glitches linger over a US NDA for Elevar/Hengrui’s novel liver cancer combination following a second complete response letter, the separate issue of underrepresentation of US patients in multiregional trials is looming large after new FDA draft guidance last year.
• By
Acting Commissioner Sara Brenner rejected the company’s request for a hearing, saying the lack of an adequate and well-controlled trial showing tasimelteon's effectiveness in treating sleep-onset insomnia is “fatal to the application.”
• By
Prospects are promising for many of the 16 novel candidates that received complete response letters in 2024 after half of the 2023 CRL class was approved the following year.
• By
Vanda’s fight against a complete response letter for its gastroparesis drug results in a notice of opportunity for hearing on a formal FDA proposal to refuse to approve the NDA.
More from Product Reviews
• By
The European Medicines Agency has recommended five drugs for EU-wide approval , including Averoa’s Xoanacyl for concomitant hyperphosphatemia. Two companies have withdrawn their marketing authorization applications.
• By
Eli Lilly’ will request a re-examination after the European Medicines Agency declined to recommend its Alzheimer’s disease drug Kisunla for EU approval.
• By
The European Medicines Agency’s qualification of the AIM-NASH tool is said to signify a major advancement for clinical trials for metabolic dysfunction-associated steatohepatitis. The market size for MASH treatments is expected to grow substantially in the coming years.