After weeks of anticipation, PTC Therapeutics has decided to charge $35,000 a year — depending upon patient weight — for a newly purchased drug for treating Duchenne muscular dystrophy, although the price may be a hard sell to some insurers and families.
The pricing is well below the $89,000 that Marathon Pharmaceuticals planned to charge before selling the decades-old steroid last month to PTC. But whether this lower price is enough to assuage parents or make it worth the $140 million purchase — plus royalties and other payments — is unclear.
At that price, “we expect payer pushback and continued headline noise,’’ wrote RBC Capital Markets analyst Matthew Eckler in an investor note. He also pointed out the price was higher than the $25,000 that had been expected for the drug, which alleviates symptoms of a rare disease that mostly affects young boys, causing muscles to deteriorate and leading to an early death.
After receiving regulatory approval in February, Marathon angered families because they had been able to buy the drug from online pharmacies in the UK and Canada for about $1,000 annually, or less than $2 a pill. And the approval precluded imports.
The move caused a commotion and prompted congressional lawmakers to investigate Marathon, which did little testing of its own and reportedly paid universities, researchers, and the Muscular Dystrophy Association to license data from studies of the drug in Duchenne patients conducted in the 1990s.
Marathon sought to avoid the political spotlight and found a willing buyer in PTC, which has been sparring with the Food and Drug Administration over a Duchenne drug that the agency rejected last year. The company bought Emflaza to build market share while pressing the agency to reconsider.
PTC’s drug could treat a broader set of patients than Exondys 51, developed by Cambridge’s Sarepta Therapeutics Inc., the first US-approved Duchenne treatment, which works on patients with a specific genetic mutation. The Sarepta drug costs $300,000 a year.
On a conference call with analysts, PTC executives forecast Emflaza sales to reach just $5 million to $10 million this year and acknowledged that insurers are likely to require prior authorization, which refers to receiving approval before insurance is provided for a prescription.
Even so, PTC executives were bullish and maintained the pricing is “sustainable’’ and a patient assistance program has been created for those without insurance.
“We think the patients are going to get coverage,’’ PTC chief operating officer Mark Rothera told analysts. “… Overall we’re seeing good progress and we’re looking forward to continuing sharing with the payers the high unmet needs.’’ There are about 9,000 eligible patients.
Some payers are already pushing back, though. Last month, the Washington State Drug Utilization Review Board rejected Emflaza and, instead, recommended prednisone, which is expected to cost the state Medicaid program just 5 cents per 20 mg tablet. This translates to an approximate cost of $55 per patient per year.
Some parents, meanwhile, are similarly put off.
“The cost is $35,000 for a child weighing 25 kilograms and my son is 100 kilograms, so it would cost four times that,’’ says Christine McSherry, who runs The Jett Foundation, a nonprofit group that advocates on behalf of children with DMD. “There are children in the 60 to 100 kilogram range.
“So obviously, I have concerns. Remember, this is not an innovative new drug and the investment wasn’t made [to win approval] as with other drugs. Ultimately, the burden falls on patients who will have to fight the payers …
“This was never about accessibility, because you could order it online and there are plenty of physicians who would write prescriptions. This was more about affordability, but now, it’s about both.’’
Robert Weisman of the Globe staff contributed to this report. Ed Silverman can be reached at ed.silverman@statnews.com. Follow Ed on Twitter @Pharmalot.
