- Emflaza (deflazacort), the first Food and Drug Administration-approved corticosteroid for Duchenne muscular dystrophy (DMD), will launch "in the next few weeks," according to PTC Therapeutics, with an annual net price of $35,000 for a typical 25 kg Duchenne patient after rebates and discounts.
- The price appears lower than previous owner Marathon Pharmaceuticals' planned wholesale acquisition cost (WAC) of $89,000 per year. Yet, PTC did not announce its own WAC for the drug.
- Emflaza brings controversy with it, as deflazacort is decades old and has been used off-label for DMD for many years. The drug costs about $1,000 in some other countries.
Most companies argue that high prices for new drugs, particularly those for smaller indications diseases, are necessary because of the balance between development costs and market size. The glucocorticoid Emflaza, however, was fully developed and had U.S. approval for Duchenne muscular dystrophy when it was acquired by PTC Therapeutics in March 2017 from Marathon Pharmaceuticals. It arrived with its own pricing baggage, after Marathon Pharmaceuticals had faced a barrage of complaints over its planned price tag of $89,000 per year before rebates and discounts, driving it to offload the drug.
CEO Stuart Peltz said during a first quarter call this week that the price, which does look more modest at least in comparison with Marathon's much bigger tag, as being a result of having consulted with several stakeholders and having evaluated the current and future dynamics of the market.
"We believe this represents sustainable pricing which balances providing access to all eligible patients in the United States in an ultra-orphan population while maintaining sufficient infrastructure and program including continued investment in Duchenne," he said.
This includes market access programs to help eligible patients access Emflaza regardless of their financial or insurance status and as well as minimize or eliminate their out-of-pocket expenses. Further explanations for the price tag included the need to improve the standards of care, to provide education for the Duchenne community, and to provide financial returns for shareholders.
It’s worth noting, however, that this is a net price, so reduced by rebates and discounts, and that it only applies to a 25 kg patient, the weight of an average seven or eight year old. As care advances, boys with DMD are living slightly longer, often into their teens.
"Given our commitments to work with the community to help with earlier diagnosis and access to treatment even earlier we think that in the long run that represents a good weight to anchor this price on and yes some patients will be heavier and some will be lighter, but that I think is a good guide post," said Mark Rothera, CCO of PTC.
While PTC Therapeutics, in its earnings call, claimed "over 900 prescriptions and start forms submitted by physicians" it remains to be seen whether the promise will match the reality once the launch gets under way.
Pricing isn't going to be the only challenge faced by PTC. Deflazacort has been used for many years off label to treat DMD, so there are many, U.S. senators included, who believe that PTC, and Marathon before it, should not have exclusivity for the drug under orphan drug regulations and should be setting the price at comparable levels to other countries.