Axsome Plots New Fibromyalgia Trial After FDA Refuses Review of Former Pfizer Candidate

Axsome Therapeutics received a Refusal to File (RTF) letter from the FDA for its new drug application (NDA) for esreboxetine (AXS-14), a former Pfizer asset for fibromyalgia, due to inadequacies in one of the two clinical trials submitted145.

The FDA determined that while one 12-week, fixed-dose, placebo-controlled trial was adequate and well-controlled, the second 8-week, flexible-dose trial did not meet agency requirements, prompting the refusal15.

Both studies were originally conducted by Pfizer, and both met their primary endpoints, showing efficacy in improving pain, function, and fatigue in fibromyalgia patients15.

Axsome will initiate a new controlled clinical trial that aligns with the FDA’s feedback, aiming to start by the fourth quarter of 2025123.

Approximately 17 million people in the U.S. are affected by fibromyalgia, highlighting the ongoing unmet medical need for effective treatments15.

Sources:

1. https://www.fiercebiotech.com/biotech/axsome-plots-third-fibromyalgia-trial-after-fda-refuses-review-former-pfizer-candidate

2. https://axsometherapeuticsinc.gcs-web.com/node/12141/pdf

3. https://www.ainvest.com/news/axsome-therapeutics-regulatory-hurdle-term-pain-long-term-potential-2506/

4. https://www.pharmexec.com/view/fda-issues-refusal-file-letter-axsome-novel-fibromyalgia-candidate

5. https://trial.medpath.com/news/eccf204f400946db/fda-rejects-axsome-s-fibromyalgia-drug-application-due-to-trial-design-issues