Four-year-old boy with DMD. The company also gets a pediatric disease priority review voucher that can be redeemed at a later date for a different product; FDA says this is the ninth such voucher it has doled out.
"We now have a treatment option for kids and adults with Duchenne, which is a major advance for the community", said MDA chief medical and scientific officer Valerie Cwik.
In a separate 104-week trial of 29 male patients with Duchenne muscular dystrophy, those taking deflazacort again showed muscle improvement over those who were being given a placebo.
There's a new Duchenne muscular dystrophy (DMD) drug on the scene-at least, technically speaking. The incurable disease typically strikes teenage boys, most of whom lose their ability to walk and die before they reach age 40. The drugs also slow down the loss of arm function and slow down the onset and progression of respiratory and cardiac problems common to the disease. It primarily affects boys, occurring in about one out of every 3,600 male infants worldwide. In the U.S.it affects about 15,000 people. Although many questions, including pricing, still need to be answered, Parent Project Muscular Dystrophy said in a statement that the government's approval will increase access to more families that desperately need the treatment.
Over the past few years, companies that have gained approval for old or an existing drug to treat rare disease have received huge financial awards.
Emflaza is marketed by Marathon Pharmaceuticals, headquartered in Northbrook, Illinois. While, the price is significantly below the other DMD drug on the market, and for orphan drugs in general, the drug has been imported from overseas by patients for years at a fraction of the cost, an expert says.
The drug wasn't sold in the US mainly because no company thought it would be profitable enough to warrant the effort of seeking FDA approval.
However, it remains to be seen whether the drug will displace off-label and cheaper generic corticosteroids such as prednisone, which are already widely used to manage symptoms in DMD, a rare genetic disorder that causes progressive muscle deterioration and weakness.
A physical therapist treats a patient with DMD.
The big issue facing closely held Marathon will be how to price the drug. With the orphan designation, the company is getting seven years of exclusive rights to sell the drug in the U.S. It is third drug in 6 months approved by the FDA for diseases included in MDA's program.
With FDA approval, Marathon now has exclusive rights to sell the drug in the United States for the next seven years, even though it has been available as a generic in other countries.