The FDA has tentatively approved a new drug for muscular dystrophy, despite an apparent lack of evidence that it works. Pressure from family and industry lobbyists helped the FDA come to this landmark decision, which has been seen as a victory for the families of sufferers.
Duchennes Muscular Dystrophy
Duchennes Muscular Dystrophy is a rare muscle-wasting disease that affects boys. One in 3600 boys across the world will develop Duchennes, the most common form of Muscular Dystrophy. It causes weakness, a loss of movement and eventually an early death, with sufferers rarely getting past the age of 25. An actual cure has never been found. Until now all medication for muscular dystrophy has been in the form of steroids, which help muscles grow, in an attempt to slow the disease.
The drug, Sarepta Therapeutics’ Exondys 5, has only been tested on 12 boys, and its effects are more ‘expected’ rather than proven. Exondys 5 acts on an important protein known as dystrophin. Dystrophin plays a key role in the growth of muscle fibres.
This is a rare approval from the FDA, which normally require more rigorous testing. However, the internal dispute became so heated that the Chief Advisor had to be involved in the final decision. Desperation for a treatment has led families to lobby the FDA for approval. The FDA is the US Federal Food and Drug Association, and all new drugs and medicine need to be approved by them. Many find out the answer to the question of how long does it take to get an FDA 510K approved drug on the market the hard way, although going through a third-party review company such as http://www.fdathirdpartyreview.com/ can shave up to three months from the review and approval process.
The drug was approved under an accelerated approval system which allows drugs that haven’t been fully proven but so show promising early signs. If the drug fails to work, the FDA has said they may remove their approval. The drug company has promised to help families fund payment for the drug, which will cost around $300,000 dollars a year for one patient.
Hopefully, the early indicators of the drug prove right and this will lead to more hope for those with Muscular Dystrophy.