For people, especially children, suffering from Duchenne muscular dystrophy, the news that BioMarin’s drisapersen commercialization gets denied by the FDA is extremely dire to say the least. Although several parents that have children which suffer from this illness have stated that after treatment with drisapersen during clinical trials, their offspring’s health improved, FDA’s decision is final
One of the factors which make parents criticize the Food and Drug Administration’s decision is how drisapersen treatments would have been marked at $300,000 per month, requiring them to be covered by insurance. It’s safe to say that FDA’s dismissal of the drug came as a devastating blow for both the 2,430 boys suffering from Duchene Muscular Dystrophy and their parents.
DMD is a genetic disease that consists of a massively reduced production of a protein used in the strengthening of muscles, named dystrophin. By lacking the required amounts of protein intake, muscles eventually degenerate. Diagnosis with DMD usually occurs in boys aged between 3 or 5 years old.
Most diagnosed patients will require wheelchairs when they reach their teens, with muscles no longer able to withstand the body’s weight. After that point, severe respiratory and heart problems will start to surface. The lifespan of a DMD diagnosed patient is of about 30 years.
Currently, there are no DMD treatments approved by the FDA, making parents heavily criticize the administration’s inflexibility towards allowing drisapersen to be made commercially available. The FDA has stated that the reason for their decision is based on the drug’s close to 0 benefit towards an increased production of dystrophin.
By selling this drug, BioMarin would have made an estimated $600 million in sales, boosting the company’s total market values and share prices by a rather significant amount. Unfortunately, once the FDA’s was made public, BioMarin’s shares dropped by 10%.
The drug drisapersen functions by altering the genetic code of exons, portions of the DNA that act as blueprints, in order to create a higher production of dystrophin. This technique, known as exon-skipping, has been approached by other pharmaceutical companies as well.
The drug eteplrisen, manufactured by Sarepta Therapeutics will be reviewed by the FDA advisory board on the 22nd of January. But even if this new drug gets FDA approval, it will still have to circumvent hindrances posed by BioMarin which patented the exon-skipping technique.
Although BioMarin’s drisapersen commercialization gets denied by the FDA, DMD diagnosed patients still have hope that eventually, the FDA will approve a treatment in order to help them live a normal life, regardless of the company which manufactures the drug.