January 9, 2023 / Clinical Trials

Update from Santhera & ReveraGen: FDA Acceptance of New Drug Application for Vamorolone in Duchenne

PPMD is excited to learn from Santhera and ReveraGen that the FDA has accepted the New Drug Application (NDA) for vamorolone, an investigational dissociative steroid for individuals with Duchenne. Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of October 26, 2023 to complete its review and announce its decision. Subject to approval, Santhera plans to launch vamorolone in the U.S. in Q4-2023.

We look forward to additional updates from Santhera and ReveraGen as the review process continues, and we are so grateful to all of the families who have participated in this clinical trial program. We are hopeful that vamorolone will be another approved therapeutic option for the Duchenne community.

Read the community letter from Santhera

Dear Duchenne community,

We are writing to share the news that the U.S. Food and Drug Administration has accepted the new drug application for vamorolone for the treatment of Duchenne muscular dystrophy. The FDA has set the PDUFA date for vamorolone as Oct. 26, 2023.

We will continue to keep you informed of key developments in the regulatory process. As stated in the attached press release, the FDA has indicated that it does not intend to hold an advisory committee meeting to discuss the vamorolone application. Contingent on the approval of the NDA, Santhera is on track to commercially launch vamorolone in the U.S. in the fourth quarter of this year.

We encourage you to read the attached press release for more details about the filing, commercialization plans outside the U.S., and comments from the top leadership of Santhera and ReveraGen.

Santhera and its partner ReveraGen would once again like to express deep gratitude to all of the global patient advocacy groups that have helped to make this milestone possible. Your patience and cooperation throughout the development of vamorolone have demonstrated again and again the collaboration that is at the core of this exceptional community.

If you have any questions, please do not hesitate to contact Santhera’s Patient Advocacy Consultant Mindy Cameron at mindy.cameron@santhera.com.

Click here to read the full press release.

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