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In an attempt to head off the sort of controversy that has followed other companies' unforced price raises, the company has also promised that Emflaza will be made free for poorer patients through its assistance program, and it's confident that insurance companies will cover the bulk of the drug's costs. "We hope that this treatment option will benefit many patients with DMD".

For the last two decades, patients have been buying deflazacort from other countries ever since clinical trials demonstrated its effectiveness for treating Duchenne muscular dystrophy.

DMD is the most common type of muscular dystrophy. The first symptoms are usually seen between 3 and 5 years of age and worsen over time.

People who have DMD eventually lose the ability to carry out activities on their own and are wheelchair bound by the time they reach their early teen years. As the disease progresses, life-threatening heart and respiratory conditions can occur. It is more common among boys than among girls. Marathon had to invest money in new trials before the FDA would approve US sales. 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.

Emflaza is marketed by Marathon Pharmaceuticals, headquartered in Northbrook, Illinois.

The drug wasn't previously sold in the USA because there was no company that would think it was profitable enough to warrant the effort of getting FDA approval.

The drug, Emflaza, known generically as deflazacort, belongs to a class of anti-inflammatory drugs known as corticosteroids that are frequently used to treat DMD and other conditions.

A physical therapist treats a patient with DMD.

Although the price has increased, Babar Ghias, Marathon's Chief Financial Officer, says that drug is still in the bottom 10 percentile of rare disease drug prices. With the orphan designation, the company is getting seven years of exclusive rights to sell the drug in the U.S.

The sponsor is receiving a rare pediatric disease priority review voucher under a program meant to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

With FDA approval, Marathon now has exclusive rights to sell the drug in the U.S. for the next seven years, even though it has been available as a generic in other countries. This is the ninth rare pediatric disease priority review voucher issued by the FDA since the program began.


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