Thursday, May 26, was the deadline for the Food and Drug Authority to approve Sarepta Drug- Eteplirsen as potential treatment for Duchenne Muscular Atrophy or DMA. A number of patients together with their families are all waiting for the results to come out. This is because; it is the only available drug that has the potential to cure this genetic disease.
The agency decided that they need to continue with the deliberation and no one actually knew why they had to extend the deadline. There are a lot of speculations going on but nobody knows what the real score is.
The Fight Against Duchenne Muscular Atrophy?
Duchenne Muscular Atrophy is a rare genetic disorder wherein a patient suffers progressive weakness and muscle degeneration. It is a type of muscular dystrophy. The muscle weakness begins at the age of 3, and then it affects the muscles of the shoulders, pelvic area, hips and thighs. Later on, it will progress to the skeletal muscles affecting the trunk, arms and legs.
The calf’s muscles are also enlarged, and as they reach the teenage years, the respiratory and heart muscles are already affected.
Since, the muscle degeneration is progressive; there is a need of an effective agent that will eventually stop the disease. DMD is caused by the absence of a protein substance known as dystrophin which helps keep the muscle cells intact.
Surprisingly, the stocks of Sarepta surged to 20.7% after the news came out. Analyst are not sure whether this is a good news for Sarepta considering that there was an advisory committee who voted against the approval of the drug during the last month’s deliberation.
Prolonging the Agony
Simos Simeonidis, an RBC Capital Market Analyst noted that there was a delay because of the pressure coming from the patients, their families and even the members of the Congress. The delay seems to prolong the agony of the patient but can also be a source of hope in case it will be approved. Simeonidis shares that he expects the FDA to reject the drug.
FDA has a lot of apprehensions in approving the drug considering that there are few patients who are enrolled in the clinical trial. The results of the data do not support a strong conclusion. It is a strong pressure for FDA to approve this drug, as it may set an example for other pharmaceutical companies to apply for approval even without statistically significant findings.
Delaying the approval could also give an opportunity for other competitors who are developing a similar drug. Early this year, BioMarin Pharmaceutical, a company that develops DMD drugs, was turned down by FDA.
The pressure is on for FDA, if they will approve this drug, it may serve as a precedent case for pharmaceutical companies who have inconclusive clinical finding to fight for their approval. The patient and their families are also desperate to find an immediate cure because the disease is progressive and life-threatening.
Patients with DMD are expected to live up until teenage years. But thanks to the advance cardiac and respiratory care, there are now patients who are able to survive adulthood. If Sarepta’s drug will be approved this is a breakthrough in treating DMD. If it is turned down, then the long wait for DMD patients is not yet over.