Articles with #HopeforProgress

One Minute, Real Impact: Help Advocate for Elamipretide Approval

One Minute, Real Impact: Help Advocate for Elamipretide Approval

I’m writing today with an urgent request that could change lives—including mine.

The FDA is currently considering approval of a treatment called Elamipretide, which has helped stabilize key functions for people living with primary mitochondrial disease—a rare, progressive, and often devastating condition. I’ve personally benefited from this treatment, which has helped me remain more independent, contribute to my family, and avoid costlier, more intensive care.

But despite a positive recommendation from the FDA’s own advisory committee, the agency has not yet approved Elamipretide. This delay puts vital progress—and people—at risk.

If you want to understand the stakes, I’ve written more here:
👉 Read my KevinMD article

If you’re unfamiliar with terms like mitochondrial disease, orphan drug, or FDA advisory committee, I explain them briefly here:
👉 What These Terms Mean

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Here’s how you can help:

➡️ Visit the FDA’s feedback page:
https://www.fda.gov/news-events/interactive-and-social-media/fda-direct-ask-question

Then follow these simple steps:
1. Under “Topic,” select “Drugs and Biologics”
2. Under “I am a,” choose the category that best describes you (e.g., Patient, Caregiver, Advocate, Concerned Citizen)
3. In the message box (250 characters), paste this message:

Please approve Elamipretide for primary mitochondrial disease. The FDA advisory committee recommended it. As an orphan drug, this treatment deserves wise and timely action. Patients can’t afford more delays. #ApproveElamipretide

Your message goes straight to the FDA. It takes less than a minute—and it matters more than you know.

Thank you from the bottom of my heart.

Time is Running Out: Help Secure Access to Elamipretide for Ultra-Rare and Rare Diseases

Time is Running Out: Help Secure Access to Elamipretide for Ultra-Rare and Rare Diseases

A group of four individuals, including a patient in a wheelchair, smile together in a brightly colored medical office. They are discussing Elamipretide, a treatment for rare mitochondrial diseases.
Together with my amazing health care team https://www.chp.edu/our-services/rare-disease-therapy

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.

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