Sarepta muscle wasting drug, eteplirsen, is subject for approval by an advisory panel after the Food and Drug Administration (FDA) has reviewed the drug's effectiveness.
Sarepta Therapeutics manufactures the eteplirsen drug, which has yet to be approved by the Peripheral and Central Nervous System Drugs Advisory Committee. After its review on April 21, the FDA asked the panelists to answer a series of voting questions which are mainly focused on the drug's effectivity.
The voting questions aim to guide the panel in giving its recommendations based on significant evidence of eteplirsen's effectivity on trial data.
On April 22, Sarepta's shares went down to 44 percent just when the FDA released the voting questions for the advisory panel's meeting on April 25.
The advisory panel will meet to include the FDA's review on Sarepta's eteplirsen drug. The meeting will also take into consideration public commentary and Sarepta's drug analysis. The public commentary will allot two hours and 30 minutes for doctors, patients and drug advocates.
The FDA's head of pharmaceutical division will be attending the meeting on Monday and is expected to thoroughly scrutinize Sarepta's drug vis-à-vis the disease.
Sarepta is pushing for the approval of eteplirsen in treating DMD or Duchenne muscular dystrophy. Patients of this disease typically die at the age of 30.
DMD is a disease caused by defective dystrophin, or the protein present in the muscles. The genetically passed disease commonly occurs in 1 out of 3,600 male infants.
There is much pressure on the FDA to approve the drug because there is no current FDA-approved treatment for DMD, and steroidal drugs could slow the loss of muscle strength among DMD patients.
The effects of steroidal medicines such as albuterol, amino acids, creatine, fish oil, and others that are said to help in muscle treatment have not yet been proven.
A drug previously developed by BioMarin Pharmaceutical Inc. for the disease was dismissed last January.
Photo : The U.S. Army | Flickr
