Time is Running Out: Help Secure Access to Elamipretide for Ultra-Rare and Rare Diseases
Elamipretide, a lifeline for patients with (ultra-)rare mitochondrial diseases like Barth syndrome and primary mitochondrial myopathy, faces a critical moment. Despite years of research, lived improvements, and community advocacy, the path to approval is uncertain. The FDA will make a pivotal decision in early August. I am one of the patients currently on Elamipretide, and I know firsthand the difference it makes. We need your voice.
- Why Elamipretide Matters (my article on MEDPAGETODAY KevinMD platform)
- Discover more about the company that developed Elamipretide
- Take a closer look at the New Drug Application Process (NDA)
- Learn more about FDA Expanded Access
- Explore the FDA Orphan Drug Act
- Barth Syndrome Foundation: Elamipretide Timeline
Please share this urgently. Post it, email it, talk about it. Please use the following hashtags BarthProgress #ApproveElamipretide #BarthSyndrome #RareDisease #FDAActNow #HopeforProgress
The window to act is closing—let’s make sure the FDA hears the real stories and sees the real lives at stake. This goes beyond Elamipretide—it’s about ensuring fair access and consideration for all treatments targeting ultra-rare and rare diseases.
We are asking the U.S. Food and Drug Administration to reconsider its denial of elamipretide and grant full, traditional approval—the first ever for primary mitochondrial disease. Doing so would honor the advisory committee’s recommendation, uphold the standard NDA pathway the FDA itself selected, and fulfill the intent of the Orphan Drug Act. We also urge the FDA to approve the drug with broad labeling, so that all appropriate patients—not just a narrow trial subset—can access a therapy that has demonstrated meaningful benefit. No more delays. No more deferrals. No more circular barriers. This is not just about one drug—it’s about whether we are willing to meaningfully advance treatment for (ultra) rare diseases at all. https://kevinmd.com/2025/07/fda-delays-could-end-vital-treatment-for-rare-disease-patients.html
