PTC Therapeutics announce the launch of Emflaza for the treatment of Duchenne muscular dystrophy (DMD) within the coming weeks, with an expected net price of $35,000.

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The Approval

Earlier this year, the FDA approved Emflaza, a drug used to treat Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness.The DMD treatment, commonly referred to by its generic name deflazacort has shown to prolong movement, and preserve cardiac and respiratory function.Unfortunately due to recent pricing controversies as previously discussed by TheSocialMedwork, the drug used to treat children over the age of five with DMD has not been made available to the public yet.

The Pricing

Emflaza, since its approval has never been shy of controversy. The approval was granted to Marathon Pharmaceuticals who slapped a hugely inflated price tag on a drug that has been available as a generic outside the US at a cost of approximately $1000 a year. This ignited an intense pricing kickback involving a bombardment of complaints from patients, families and lawmakers, over its wholesale planned price of $89,000. Disputes followed when Marathon then sold the drug to PTC Therapeutics3, leading to an additional US congressional inquiry by US Senator Bernie Sanders and Representative Elijah Cummings.

The acquisition by PTC Therapeutics renewed some hope amongst those affiliated with DMD, with the anticipation that the drug would be made available sooner, and at a lower price. Last  week PTC announced that Emflaza will be made available within a number of weeks4, but the news failed to alleviate cost concerns as the price remains high at an annual net price of $35,000, a cost 35 times higher than its price overseas. 

The Outcome

According to a recent article by The Wall Street Journal, the vague pricing statement was announced on a conference call with analysts, causing some controversy regarding patients’ weight. The FDA’s prescribing instructions illustrates a direct correlation between patients’ weight and dosage, indicating that the cost of treatment may be higher for those that weigh more.5 However, according to the article a PTC spokesperson believes that pricing will not be affected by the patients’ weight.

In a letter to Duchenne families, PTC acknowledged the anxiety families are facing about the affordability and access to Emflaza. In response to this, they have stated a goal “to provide Emflaza to all eligible Duchenne patients, regardless of insurance status or type, and have developed a process so that patients have seamless access to the appropriate service.”

Most pharmaceutical companies will argue that the high price of new drugs particularly for rare diseases, are necessary to make up for development costs and market size. But PTC will find it hard to justify this price as the drug was fully developed and had US approval when it was acquired from Marathon. PTC have not announced how it determined the net price, a term used to describe the revenue they receive after payment assistance for those without insurance.

It is not clear whether Emflaza will be widely covered by US insurers.

The Future

In recent months, we have witnessed similar pricing disputes such as the recent FDA approval of edaravone for the treatment of ALS - at a cost four times higher than outside the US.

Affordability and access to the latest medicines is now a global concern, and these issues are directly affecting our patients, friends and family. Sjaak Vink CEO of TheSocialMedwork, has stated that “while the goal is clear, the path is not. One step in the right direction however, is to voice our views on pricing on behalf of our patients as it is only through a shared understanding of fair pricing that we can reach our goal of the affordable access to medicines for everyone, including those in the US.”



2. McAdam et al. Acta Myol. May, 2012;