Duchenne Muscular Dystrophy Treatment Emflaza Approved by FDA

The Muscular Dystrophy Association (MDA), which was involved in the trials of Emflaza, welcomed the approval, saying the drug has shown "meaningful benefits for patients living with DMD and has the potential to delay disease progression". In the U.S.it affects about 15,000 people.

Northbrook, Illinois-based Marathon Pharmaceuticals announced on Thursday that it had snagged an FDA green light for corticosteroid Emflaza, known generically as deflazacort.

The practical cost will be negligible if you have appropriate health insurance, and Marathon contends that this is a low price for a rare disease drug.

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.

Emflaza's approval also brings in a strong competitor for Sarepta, whose DMD drug Exondys 51 (eteplirsen) secured approval last September.

A physical therapist treats a patient with DMD.

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An email sent to a spokesperson for deflazacort drugmaker Marathon Pharmaceuticals regarding pricing for the drug was not returned as of press time. With the orphan designation, the company is getting seven years of exclusive rights to sell the drug in the U.S.

With its orphan designation, the pharmaceutical company is allowed seven years of exclusivity to sell the treatment in the US, even though the drug has been available for years in other countries as a less expensive generic.

In a separate trial of 29 male patients lasting 104 weeks, deflazacort showed a numeric advantage over placebo on an assessment of average muscle strength.

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. In addition, although the study was not statistically controlled for multiple comparisons, subjects treated with deflazacort appeared to lose the ability to walk later than did those given placebo. This is the 9th time that the FDA grants the pediatric disease priority voucher. Adverse events caused by deflazacort are similar to those experienced with other corticosteroids, with the most common including facial puffiness, weight gain, increased appetite, upper respiratory tract infection, and extraordinary daytime urinary frequency.

Other less-common AEs include problems with endocrine function, increased susceptibility to infection, hypertension, the risk of gastrointestinal perforation, serious skin rashes, behavioral and mood changes, decreased bone density, and vision problems, such as cataracts.

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