The company believes the Translarna data is compelling and pointed to the success the drug is having in Europe.
SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. is ready to fight for the Duchenne Muscular Dystrophy community and its drug Translarna (ataluren) that has been rejected by the U.S. Food and Drug Administration on more than one occasion.
Translarna, which has been approved for use in Europe, was rejected for the third time by the FDA in October 2017. The drug is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. In its Complete Response Letter, the FDA called for additional efficacy data from another trial. PTC objected saying at the time the FDA’s “decision fails to consider the benefit-risk of ataluren and the high unmet medical need.”
PTC Chief Executive Officer Stuart Peltz said the company has appealed the decision and expects to provide an update on the process during the first quarter.
In an exclusive interview with BioSpace, Peltz said the company believes the Translarna data is compelling and pointed to the success the drug is having in Europe. The FDA could change its mind. This past year the regulatory agency has had a change of heart on three drugs that cleared the way for commercialization. Peltz is hopeful Translarna will be the fourth drug in the past 12 months to get a second life.
“We’re pleased to be moving forward with this with the FDA,” Peltz said.
While PTC fights for the future of Translarna in the United States, the company does have an approved DMD product – Emflaza, which it acquired from Marathon Parma last year. Marathon won FDA approval for its Duchenne muscular dystrophy treatment Emflaza (deflazacort) but was raked over the coals of public opinion for its pricing. The same drug is sold in Europe by Sanofi for about $1,000, but Marathon offered the drug in the U.S. for $89,000. Following the public scolding and pressure, Marathon eventually sold the drug to PTC Therapeutics for $190 million.
When the opportunity presented itself to acquire the drug Peltz said it made sense to have a treatment that could accompany Translarna.
“We knew Emflaza was a superior product to prednisone…. The data was consistent. Our decision was based on all of that. We didn’t just buy another steroid, we bought a superior product,” Peltz said.
Duchenne muscular dystrophy is an X-linked degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. DMD is associated with specific errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function. The condition is universally fatal, and death usually occurs before the age of 30.
Once Emflaza came into the PTC pipeline, the company initiated multiple programs to get the drug into the hands of patients. As rare disease company with a focus on DMD, Peltz said they already knew the players and advocacy groups in the space and those relationships proved instrumental in providing a pathway for patients to receive the drug. Once Emflaza was in PTC’s hands, much of the criticisms aimed at Marathon went away.
“We believe the noise went away when we changed the conversation from pricing to access to patients. It was easy for us to look beyond that. The reality is that patients need this. We are a patient-focused company and the questions will always be ‘what is best for patients?’” Peltz said.
The company anticipates increased revenues over the next few years. In 2017, Emflaza generated about $29 million for the company and Translarna brought in $145 million from European sales. Over the course of the next fiscal year, PTC anticipates revenue of about $170 to $185 million from Translarna and $90 to $110 million from Emflaza. If Translarna is ultimately approved in the U.S., those figures would move higher.
In addition to the two DMD drugs, PTC is also moving forward with RG7916, a therapy for spinal muscular atrophy (SMA). Peltz said the company will present data from its FIREFISH study in Type 1 SMA patients at the upcoming SMA Europe International Scientific Congress. The company also initiated the SUNFISH trial for SMA patients with Type 2 and Type 3. In October, the company reported interim data from the SUNFISH trial that showed patients taking RG7916 for 28 days or longer demonstrated an exposure-dependent increase in SMN protein, a key protein affected by SMA patients.
“We’re really bullish on this program and believe it can be a best-in-class,” Peltz said.
Overall, Peltz said PTC is moving forward into the new year with a strong global structure that is “well-established and scalable with strong and experienced people involved in orphan diseases.”
