MDA welcomes FDA approval for muscular dystrophy drug

Modesto Morganelli
Febbraio 11, 2017

Corticosteroids are a staple for boys with Duchenne and until the approval of deflazacort, families would you use off label corticosteroids or buy deflazacort from overseas pharmacies. But deflazacort is a generic steroid, and should not be treated as a "new" drug.

Centers for Disease Control and Prevention researchers analyzed data from 41 states and found that 457 people were diagnosed with high lead levels in their blood from bullets between 2003 and 2012, NBC News reported. Importantly, the unwanted side effects often experienced with corticosteroids, such as weight gain, loss of bone mass, glucose intolerance (diabetes) and behavioral issues, may be less severe with Emflaza as compared to other steroids.

While this is the first time the FDA has approved deflazacort, the steroid has been available outside the USA for decades.

FDA approval of Emflaza marks a third major proof point of MDA's research program in recent months.

That will now change. Marathon Pharmaceutical, the Illinois-based company that shepherded the drug through its clinical trials, will sell their version, called Emflaza, for an estimated list price of $89,000 a year, a sharp hike up from the brands of deflazacort available elsewhere in the world, some of which can run as low as $30 for a 100-pill bottle.

The move is reminiscent of a plan former pharma CEO Martin Shkreli had with a treatment for another rare disease. The decades-old drug hasn't been approved in the USA, but it's used around the world and provided in specific instances to the U.S. for free. Drugs like Emflaza aren't developed in silos, but rather through the combined efforts of biotechs and drug companies, patient organizations like MDA, corporate partners and donors, and the families who are there every step of the way to see it through. These vouchers have been sold for up to $350 million.

The tablet and liquid forms of Emflaza (deflazacort) were approved to treat patients age 5 years and older.

Marathon will also get a priority review voucher, which are granted to companies who develop treatments for neglected diseases as a way to add incentives on drugs that otherwise wouldn't have much commercial value.

Marlin, who has treated his children with imported deflazacort from the United Kingdom, is hopeful though that the FDA's decision will spark future drug approvals.

Altre relazioniGrafFiotech

Discuti questo articolo

Segui i nostri GIORNALE