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Parent Project Muscular DystrophyEarlier this week, PPMD, Best Day Ever Foundation, Inc., Kindness Over Muscular Dystrophy, Muscular Dystrophy Association, MDFF Indiana, and Team Joseph held a town hall webinar following the July 29, 2026, FDA Advisory Committee meeting for Deramiocel.
We reviewed the committee’s discussion and vote, offered insight into what the recommendation means (and what it doesn’t), discussed next steps as we approach the FDA’s August 22, 2026, PDUFA action date, and answered questions from the community.
Watch the recording now: www.parentprojectmd.org/aiovg_videos/town-hall-deramiocel-fda-advisory-committee-meeting-update/
#melanoma #DrugApproval AIM at MelanomaWe have good news to share!
Today, the U.S. Food & Drug Administration (FDA) approved RP1 (vusolimogene oderparepvec) in combination with nivolumab for advanced melanoma. This treatment is approved for patients whose melanoma has continued to grow despite previous treatment with immunotherapy—a patient population in need of more treatment options.
We've been updating you on RP1 for the last few years. Here's how we got here:
• 2024: FDA officials granted Breakthrough Therapy designation to RP1 + nivolumab, an FDA designation that is intended to expedite the development and review of drugs that treat serious conditions and show promising preliminary clinical evidence.
• January 2025: The FDA accepted the Biologics License Application (BLA) for RP1 and granted Priority Review, based on RP1's phase 1/2 clinical trial, IGNYTE, showing promising results that the combination of RP1 + nivolumab can be effective and safe for patients who have failed to benefit from immunotherapy.
• July 2025 and April 2026: The FDA issued Complete Response Letters (CRLs) and did not approve the application.
• July 2026: The FDA reviewed the treatment again. An FDA Advisory Committee met to review the data and voted in favor of RP1.
• Today: The FDA approved RP1 in combination with nivolumab for patients with advanced melanoma whose melanoma has continued to grow despite previous treatment with immunotherapy.
This approval represents the efforts of so many people working toward the same goal. AIM at Melanoma was honored to work alongside our fellow melanoma organizations to compile patient stories for the FDA after the first denial and again for the FDA Advisory Committee meeting. We also helped organize patient speakers for that meeting.
Our heartfelt thanks go to every patient who courageously shared their story, every patient who spoke before the Advisory Committee, every family member and caregiver who stood beside them, the physicians, nurses, researchers, and clinical trial teams whose dedication made this therapy possible, and our fellow melanoma organizations whose collaboration and advocacy helped ensure that the patient voice was heard.
We also extend our deepest gratitude to AIM at Melanoma President Sam Guild and Vice President Alicia Rowell, whose tireless, determined, and unwavering advocacy spearheaded these efforts. Their persistence in ensuring that patients' voices were heard throughout this process reflects AIM's steadfast commitment to expanding treatment options for the melanoma community.
Today is a meaningful step forward for patients with advanced melanoma who need more treatment options.
We'll share more about what this approval means for patients, how RP1 works, and who may be eligible in tomorrow's AIM at Melanoma newsletter. If you're not already subscribed, visit our website and sign up so you don't miss it.
FDA link in comments
Cure GM1 Foundation And the love goes on...Today, we hold Batul and her family in our hearts on what would have been her third birthday. It is for Batul, and for the many other children we have lost too soon, that we continue to fight to find a treatment for GM1.
#curegm1
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