FDA Approves Muscular Dystrophy Drug

Handsome, happy biracial eight year old boy smiling in wheelchair outdoors

The FDA has approved the first ever treatment for Duchenne muscular dystrophy, going against the recommendation of a panel of independent advisors who months earlier said eteplirsen did not demonstrate clear efficacy in treating the rare muscle-wasting disease.

Eteplirsen, which will be marketed under the brand name Exondys 51, won accelerated approval for muscular dystrophy despite the fact that the pivotal clinical trial used by the manufacturer enrolled just 12 boys with Duchenne and did not include a placebo arm. An unusually high number of patient advocates and boys with the disease attended and spoke in favor of the drug’s approval during an April FDA advisory committee meeting to discuss eteplirsen’s fate.

Eteplirsen is given as a once-weekly intravenous infusion. Sarepta Therapeutics, Exondys 51’s manufacturer, says the product will launch immediately. However, eteplirsen is only indicated for children with Duchenne that have a certain genetic mutation.

The FDA based its decision on data submitted by Sarepta that indicated the drug increases levels of dystrophin, a protein that keeps muscle intact. However, the agency conceded that in making the approval, the data did not demonstrate a clinical benefit, such as improved motor function, associated with eteplirsen.

Politics Over Science?

In addition, there have been no long-term studies on the benefit or side effects of the drug. The most common side effects seen in the pivotal trial were balance issues and vomiting.

What may have swung the pendulum in Sarepta’s favor was the immense amount of pressure from patient advocacy groups to have eteplirsen approved. A group of Republican senators even sent a letter to the FDA lobbying for eteplirsen’s approval.

While eteplirsen is on the market, Sarepta will have to conduct a randomized, controlled trial (meaning a placebo arm) lasting 2 years to confirm the drug’s clinical benefit through improvement in motor function. If the trial does not demonstrate this, the FDA could revoke eteplirsen’s approval, meaning it could become unavailable.

DISCLAIMER: MedShadow provides information and resources related to medications, their effects, and potential side effects. However, it is important to note that we are not a substitute for professional medical advice, diagnosis, or treatment. The content on our site is intended for educational and informational purposes only. Individuals dealing with medical conditions or symptoms should seek guidance from a licensed healthcare professional, such as a physician or pharmacist, who can provide personalized medical advice tailored to their specific circumstances.

While we strive to ensure the accuracy and reliability of the information presented on MedShadow, we cannot guarantee its completeness or suitability for any particular individual's medical needs. Therefore, we strongly encourage users to consult with qualified healthcare professionals regarding any health-related concerns or decisions. By accessing and using MedShadow, you acknowledge and agree that the information provided on the site is not a substitute for professional medical advice and that you should always consult with a qualified healthcare provider for any medical concerns.

Was This Article Helpful?

Show Comments (0)
0 0 votes
Article Rating
Notify of
Inline Feedbacks
View all comments
Would love your thoughts, please comment.x