CHMP Issues Negative Opinion on Translarna™ After EU Commission Review Request

PTC Therapeutics, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) has issued a negative opinion on renewing the conditional marketing authorization for Translarna™ (ataluren) for treating nonsense mutation Duchenne muscular dystrophy (nmDMD). This decision follows a previous negative opinion returned by the European Commission (EC) for re-evaluation.

Despite this setback, PTC plans to request a re-examination of the CHMP’s opinion. During this process and pending the re-examination and subsequent EC ratification, the marketing authorization for Translarna will remain effective. PTC expects Translarna to stay on the market through the end of 2024, even if the negative opinion is eventually upheld and ratified.

Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, expressed disappointment over the CHMP's decision. He highlighted that the European Commission had requested a review considering all evidence, including the real-world data from the STRIDE registry. Dr. Klein stressed that the Scientific Advisory Group, part of the recent review procedure, acknowledged the significant efficacy evidence from STRIDE, which should not be disregarded. This viewpoint aligns with those of key opinion leaders in Duchenne muscular dystrophy (DMD) worldwide. PTC intends to pursue a re-examination to ensure Translarna remains available for as long as possible for boys and young men with nmDMD.

Translarna (ataluren), developed by PTC Therapeutics, is a treatment that helps restore protein production in patients with genetic disorders caused by nonsense mutations. These mutations disrupt the genetic code and halt the synthesis of necessary proteins prematurely. The disorder's impact depends on which protein is affected, such as dystrophin in Duchenne muscular dystrophy. Translarna is approved in several countries for treating nmDMD in ambulatory patients aged two years and older. However, in the United States, ataluren is still classified as an investigational new drug.

Duchenne muscular dystrophy primarily affects males and is a severe genetic disorder characterized by progressive muscle weakness beginning in early childhood. This condition leads to premature death in the mid-20s due to heart and respiratory failure. Duchenne results from the lack of functional dystrophin protein, essential for the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients often lose their ability to walk by the age of 10 and subsequently lose the use of their arms. As the disease progresses, patients face life-threatening lung complications requiring ventilation support, along with severe heart issues in their late teens and 20s.

PTC Therapeutics, Inc. is a global biopharmaceutical company dedicated to discovering, developing, and commercializing innovative medicines that benefit patients with rare disorders. PTC's commitment to innovation and global commercialization is the driving force behind its investment in a diverse pipeline of transformative medicines. The company's mission is to provide access to top-notch treatments for patients with limited or no treatment options. PTC's strategy leverages its strong scientific and clinical expertise and global commercial infrastructure, aiming to deliver therapies to patients and maximize stakeholder value.