The European Commission (EC) has officially adopted the Committee for Medicinal Products for Human Use (CHMP) recommendation to not renew the authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). This decision effectively removes the drug's conditional marketing authorization throughout the European Economic Area after a prolonged review period.
Despite this setback, the EC has indicated that individual European Union member states can leverage specific regulatory provisions—Articles 117(3) and 5(1) of EU Directive 2001/83—to potentially allow continued use of Translarna for patients who need it.
"We are of course disappointed that after this prolonged period of review the European Commission has decided to adopt the CHMP negative opinion on Translarna," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "The EC's indication that European Union member states have a mechanism to maintain treatment speaks to the safety, benefit and lack of alternative therapies for boys and young men with nonsense mutation Duchenne muscular dystrophy."
Klein added that the company looks forward to "working on a country-by-country basis to provide commercial drug where possible."
Regulatory Background and Commercial Impact
This decision follows a complex regulatory journey for Translarna in Europe. In May 2024, the EC had initially decided not to adopt the CHMP's negative opinion on the annual renewal of Translarna's conditional marketing authorization and returned the opinion to the CHMP for re-evaluation. However, the final decision has now aligned with the CHMP's recommendation against renewal.
Translarna has been a significant revenue generator for PTC Therapeutics, with net product revenues of $339.9 million for the full year 2024, though this represented a slight decrease from $355.8 million in 2023. The European market has been crucial for the drug, as it has not received approval in the United States.
Ongoing Regulatory Efforts
While facing this European setback, PTC Therapeutics continues to pursue approval in other markets. In 2024, the company submitted four regulatory approval applications to the U.S. Food and Drug Administration (FDA), all of which have been accepted for review, including Translarna for nmDMD.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a rare and fatal genetic disorder that primarily affects males, resulting in progressive muscle weakness from early childhood and leading to premature death in the mid-20s due to heart and respiratory failure. The condition is caused by the lack of functional dystrophin protein, which is critical to the structural stability of all muscles.
Patients with Duchenne can lose the ability to walk as early as 10 years old, followed by loss of arm function. Life-threatening lung complications requiring ventilation support and heart complications typically develop in their late teens and 20s.
Translarna's Mechanism of Action
Translarna, discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of functioning protein in patients with genetic disorders caused by a nonsense mutation. In Duchenne, a nonsense mutation prematurely halts the synthesis of dystrophin, an essential protein for muscle function.
The drug has been licensed in multiple countries for the treatment of nmDMD in ambulatory patients aged 2 years and older. In the United States, ataluren remains an investigational new drug.
As PTC Therapeutics navigates this regulatory challenge, the company's focus will shift to working with individual European countries to maintain access to Translarna for patients with nmDMD, while continuing to pursue approval in the United States and other global markets.
