Rare Disease Report

The High Cost of Corticosteroids to Treat Duchenne

MAY 08, 2017
James Radke
Emflaza (deflazacort) has an interesting history.
Earlier this year, the FDA approved the corticosteroid for treating Duchenne muscular dystrophy and people in the Duchenne community were furious. 
Families could previously get a year’s supply of drug for about $1,600 from England or Mexico prior to Marathon Pharmaceuticals receiving approval of the drug.

Shortly thereafter, those same families could no longer legally obtain the drug overseas, and its price tag was set at $89,000 a year
Following that announcement, the backlash from the community eventually led Marathon to halt the drug’s launch and finally sell the drug to PTC Therapeutics.
Those within the Duchenne community who impatiently waited to see what price PTC Therapeutics would place on the drug got their answer earlier today – the price is not that much different from Marathon’s original number.
The good news is that PTC said the average 25kg boy with Duchenne will cost about $35,000 per year to get treatment with Emflaza. The bad news, however, is that most Duchenne boys weigh more than 25 kg (55 lb). As these boys grow into teenagers, because they are taking corticosteroids, they typically weigh more than 75 kg, meaning the price for those boys could be more than $100,000. 
$100,000 for a drug that, until last year, could be purchased for $1,600.
During their webcast, PTC Therapeutics stressed that they will have a patient support network set up to help both families with proper medical insurance and those without to be able to access the drug.

During a call to the Duchenne community, PTC said their ‘emflaza-care’ program will provide a specific case manager to help patients have access to the drug with the goal of keeping the price of the drug for the patient to as low as zero as possible. The case manager will work with the patient and the insurance companies to review the options available for the patient. 

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a progressive muscle disorder caused by the lack of functional dystrophin protein. Patients with Duchenne muscular dystrophy lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties.
Corticosteroids are a staple for boys with Duchenne and deflazacort is the only corticosteroid specifically approved for Duchenne muscular dystrophy.
Corticosteroids do not cure Duchenne muscular dystrophy but can extend life. Drugs like PTC Therapeutics' Translarna (ataluren) and Sarepta’s Exondys 51 (eteplirsen) are drugs that change the protein synthetic process of these patients.  

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