Published: Mon, February 13, 2017
Medicine | By Daryl Nelson

FDA approves drug to treat DMD

FDA approves drug to treat DMD

A steroid treatment that has been available for years outside the US was given approval by the FDA this week to be used to treat a rare disease afflicting approximately 15,000 people in the United States.

There's a new Duchenne muscular dystrophy (DMD) drug on the scene-at least, technically speaking. The Wall Street Journal article quoted Christine McSherry, founder of the Jett Foundation, who said she pays $1600 a year for deflazacort from a British online pharmacy but now that the FDA has approved deflazacort for the United States, the British pharmacy will no longer ship it to America. It is third drug in 6 months approved by the FDA for diseases included in MDA's program.

Until now, the drug was not sold in America because no company thought it was worth the effort. The drug, which Ghias tells the Journal will be sold under the brand Emflaza starting in March, is a steroid that helps mitigate muscle weakness and cut inflammation, per an FDA release.

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.

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Even at a cost of $89,000, patient advocacy groups are enthusiastic about the FDA approval of deflazacort. It has been available for years in other countries, and the family of the individuals with Duchenne Muscular Dystrophy. The drug was never sold in the USA because companies didn't think it would be profitable enough, The Wall Street Journal reported, but some families have obtained the drug from online pharmacies overseas to help improve muscle strength for boys at least for a while.

Deflazacort is the usual treatment for DMD across the world, but it is not a cure.

In a year-long phase III trial, deflazacort was able to achieve a significant improvement over placebo on a lung function measure and muscle strength, with a non-significant trend towards an improvement on functional measures, such as time to stand from supine, time to climb four stairs and time to walk or run 30 feet. As the disease progresses, patients lose muscle strength, and usually end up confined to a wheelchair by the time they reach their early teens. 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. It can also lead to a reduction in the density of the bones.

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