PTC Therapeutics announced that Emflaza (deflazacort), an approved treatment for all Duchenne muscular dystrophy (DMD) patients regardless of mutation, will be available in a matter of weeks in the U.S., and this time around with a starting list price of $35,000 a year — for a child weighing 25 kilograms, or about 55 lbs.
In other words, the heavier the patient, the heftier the treatment’s likely price tag.
The company, which acquired Emflaza from Marathon Pharmaceuticals in March, set a weight-based price scale for the treatment, calling this scale “more relevant” to Duchenne community in a May 8 conference call announcing the therapy’s release, Parent Project Muscular Dystrophy (PPMD) reported.
It also promised to assist patients and their families in affording the treatment — whose market arrival was halted in February, just weeks after its FDA approval, because of outrage over Marathon’s proposed $89,000 per year list price.
“We have set 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,” PTC said in a letter to the Duchenne community.
PPMD responded with a statement that calmly outlined the call’s main points, then gave voice to the community’s unease.
“While the price is lower than the proposed Marathon price point there remains confusion about the weight based dosing and how that is tied to pricing,” the statement reads. It concludes in noting that — despite company and Duchenne community meetings regarding Emflaza’s reintroduction to the market — “The price announced today does not reflect our conversations with PTC, nor the concerns we expressed.”
PPMD also wondered if the new price will “lead health insurance providers to first recommend the less expensive alternative, prednisone.” And it noted PTC’s warning that the “point of start form initiation” process — necessary for assistance with access to treatment, including from families wanting to enter the EMFLAZACares program — “could take many weeks and likely require prior authorization.”
Marathon’s list price was swiftly and roundly criticized — leading the company to postpone market introduction, and later to sell the treatment to PTC for $140 million.
Largely, outrage focused on the fact that the key ingredient in Emflaza, deflazacort, is a corticosteroid — and these steroids, including deflazacort, have long been used in other countries to slow the decline in muscle strength in boys with Duchenne MD. Patients in the U.S. have been able to purchase deflazacort from abroad at a cost estimated to range from $1,000 to $1,200 a year. That access, however, will close once Emflaza officially arrives on the American market.
PTC’s weight-based annual price — which starts at $35,000 for a child of 25 kg to seem much lower than Marathon’s $89,000 — may actually make Emflaza a more expensive treatment for some DMD patients, others noted.
Christine McSherry, co-founder and executive director of the DMD nonprofit group, the Jett Foundation, was among the new price’s harshest critics. Patients range from “young boys, as little as 30 kilograms, to young men who are 100-plus kilograms,” she said in an interview with CNBC. “That price is up there over $100,000 annually.”
Her 21-year-old son, Jett, has been taking deflazacort since he was 8, a treatment he switched to after prednisone, another corticosteroid, failed to help. She said her out-of-pocket costs were about $1,200 a year for Jett’s treatment.
“Where does this fall — on drug companies or patients? The burden is really with the patients and families,” McSherry said, noting that pharmacies and insurance companies often try to push less-expensive therapies first, and Emflaza’s scaled price might limit its attractiveness from a payer’s point of view. Families are “the ones who are going to have to write all these appeals to get this drug covered, or find alternative ways to get ahold of the drug.”
Stuart Peltz, PTC’s chief executive officer, called the company’s list price is “sustainable,” a goal sought by PPMD and other groups in their pre-announcement meetings with company officials. “The factors in our decision include a consideration of the resources required to provide access to Emflaza for a small patient population to improve the standard of care and further the Duchenne community’s educational need while enabling continual investment in therapies for rare diseases and providing financial return for our shareholders,” Peltz said, according to Marketwatch.
“We know some families are anxious about the affordability and access to EMFLAZA … We have a trained team of case managers at EMFLAZACares who will work with Duchenne patients every step of the way to help them get EMFLAZA with minimal difficulty and expense,” PTC also stated in its letter.
Patients or families wanting information or interested in starting with Emflaza are advised to call 1‑844‑EMFLAZA (5292) to speak with a nurse case manager.