Background on Duchenne Muscle Dystrophy
Duchenne Muscle Dystrophy (DMD) is a rare, severe genetic disorder characterized by progressive muscle degeneration, including the heart. This debilitating condition primarily affects boys, with an average life expectancy of around 28 years.
Who is Affected?
DMD predominantly impacts young boys, causing them to lose muscle strength and mobility over time. The disease affects approximately 1 in every 3,500 to 5,000 live male births worldwide.
FDA’s Decision and Its Implications
The U.S. Food and Drug Administration (FDA) announced on Friday that it will restrict access to an innovative treatment for Duchenne Muscle Dystrophy (Elevidys) following the deaths of two teenage patients.
Reasons for Restriction
The FDA decided to limit the use of Elevidys, a treatment aimed at slowing DMD progression, after two patients suffered acute liver failure and passed away following the therapy. Both teenagers were no longer able to walk when they began the treatment.
New Prescription Criteria
As a result of these tragic events, the FDA will now only allow Elevidys prescriptions for ambulatory patients over four years old. This restriction aims to minimize potential risks associated with the treatment.
Sarepta’s Role and Response
Sarepta, a leading American biopharmaceutical company responsible for Elevidys, halted distribution to non-ambulatory patients. The FDA suspended trials and initiated a safety review in July, subsequently requesting Sarepta to cease all Elevidys shipments.
Despite the FDA’s actions, Sarepta stated its commitment to continuing Elevidys provision for ambulatory patients.
Key Questions and Answers
- What is Duchenne Muscle Dystrophy? DMD is a rare genetic disorder causing progressive muscle degeneration, including the heart. It primarily affects young boys with an average life expectancy of around 28 years.
- Why did the FDA restrict Elevidys? The FDA restricted Elevidys following the deaths of two teenage patients who experienced acute liver failure after receiving the treatment.
- Who is eligible for Elevidys treatment now? The FDA will only allow Elevidys prescriptions for ambulatory patients over four years old to minimize potential risks.
- What is Sarepta’s role in this situation? Sarepta, the biopharmaceutical company behind Elevidys, halted distribution to non-ambulatory patients and continues providing the treatment for ambulatory ones.
