Duchenne, the rare and fatal form of muscular dystrophy will not remain untreatable for long and there is finally hope for patients of the disease. A new drug which is in its trial stages has been found to be effective in treating the rare form of disease which mostly affects boys.
Bill and Terri Ellsworth of Coraopolis suffer from the rare disease and are being treated along with 11 other boys. The drug is free from any major side effects. The experimental drug is yet to receive approval from the FDA. The disease affects more than 20000 boys who will have to wait before the drug gets approval from the FDA.
Meanwhile drug developers and parents of the Duchenne patients had expressed frustration about the stop and go approach of the FDA. However on Monday, Duchene patients had something to cheer with the news that exceptional results have been obtained in the trial of the drug. It is still not clear if the new drug eteplirsen will obtain FDA approval but there is a potential path forward for the drug.
The drug developers Sarepta Therapeutics of Massachusetts is scheduled to conduct two additional studies. The FDA has also hinted that it will consider the application for accelerated approval of the drug. If everything goes as planned the drug could hit the stores by summer 2015. Additionally Sarapta has agreed to use the latest and more precise method of measuring dystrophin. Dystrophin is the muscle protein which is lacking in patients of Duchenne.
The Drug manufacturers believe that the current data are adequate to merit an approval but FDA feels that it needs additional information to ensure the safety profile of the drug. The delay in approval must be seen in the context of additional safety parameters of the drug being confirmed.