Sarepta Therapeutics Inc. shares rise after announcing that the US Food and Drug Authority requested for additional report on the company’s experimental trial for the treatment of muscular dystrophy. The company shares rise to 31 percent after hours of the announcement to $21.05 per share.
Sarepta is planning to submit the said report in the coming weeks to assists FDA in their approval decision. The report will consist of the results of the Week 48 of the experimental trial.
The company is known to develop innovative treatments for RNA targeted therapeutics for treating rare conditions, infectious, and other diseases. FDA asked if they could provide the dystrophin data which is a western blot confirmatory study for Eteplirsen. The company had thirteen patients who participated in the study.
Last month, FDA delayed its decision in approving Sarepta’s candidate drug. The news stirs the expectation of the public that FDA may approve Eteplirsen despite a recommendation from an advisory panel to reject the treatment.
FDA is in-charge of approving new therapeutic medicines. Although, FDA is not required to follow the Advisory Panel’s recommendation, but generally they do so. The latest request for additional information gives the company hope that FDA may be considering to approve the medication.
If granted an approval, eteplirsen will be used for the treatment of Duchenne Muscular Dystrophy, a crippling and disabling condition. The disease has no known treatment yet, if approved eteplirsen will be the first-line treatment. This condition destroys the muscles and it can kill the patient when they reach the age of 30s.
A New Hope for the Patient
Eteplirsen’s approval has been subjected to a myriad of challenges. Regulators debated whether the drug has provided an adequate evidence to treat Duchenne Muscular Dystrophy. It was a difficult challenge for Sarepta as the approving body continues to change its safety guidelines for the compassionate use of the drug.
Sarepta has been focusing on finding a treatment for DMD candidates. Eteplirsen is designed to target the cause of Duchenne Muscular Dystrophy by restoring the dystrophin messenger. By restoring the RNA reading frame, this will enable the cell to produce dystrophin protein.
Eteplirsen uses phosphorodiamidate morpholino oligomer (PMO), Sarepta’s proprietary chemistry that uses an exon-skipping technology. It skips exon 51 of dystrophin gene. Approximately, 13 percent of DMD patients had a skipping pattern of exon 51.
Sarepta’s clinical study proves that Eteplirsen has demonstrated tolerability and safety profile. The drug was also able to express dystrophin gene. Eteplirsen aims to promote the synthesis of a shorter sequence of dystrophin gene to prevent the onset of DMD.
As of now, there is no current treatment available to treat Duchenne Muscular Dystrophy. Eteplirsen has not yet been approved by FDA or any other regulatory body.
DMD is a fatal disease. A patient suffers respiratory paralysis because of the dysfunction of respiratory muscle. Patient may also have cardiac dysfunction. At the late stages of the disease, patient requires respiratory ventilation. Some patients end up in cardio-respiratory arrest due to heart failure.
More Pressure on FDA
Eteplirsen may be the only hope of DMD patients fighting this debilitating disease. Patients and their families are also hoping that a treatment can be found to stop the progression of the condition. FDA is indeed pressured to approve the drug considering that it is the only hope of the patient right now. But the regulatory body is also trying to perform their duty of weighing the safety and tolerability profile of the drug. As the FDA’s main goal is to come up with a decision that is best for the patient.