CHMP Upholds Negative Verdict on Translarna™ Review

PTC Therapeutics, Inc., a biopharmaceutical company listed on NASDAQ as PTCT, has reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has upheld its initial negative stance on renewing the conditional marketing authorization for Translarna™ (ataluren). This drug is intended for the treatment of nonsense mutation Duchenne Muscular Dystrophy (nmDMD). The European Commission (EC) will now review this opinion, with a decision expected in approximately 67 days.

Matthew B. Klein, M.D., the Chief Executive Officer of PTC Therapeutics, expressed disappointment, emphasizing that the CHMP’s decision was based on data from a specific subpopulation in Study 041 rather than considering the full spectrum of evidence. This broader evidence includes data from three placebo-controlled trials and the STRIDE registry, which collectively demonstrate consistent efficacy and safety of Translarna. Dr. Klein highlighted that the negative opinion goes against the wishes of physicians, patients, and their families across Europe. He assured that Translarna will remain available in Europe during the EC’s review period. Furthermore, PTC Therapeutics is committed to providing all possible evidence to the EC to support the drug’s continued authorization.

Translarna (ataluren) is a therapy developed to restore protein function in patients with genetic disorders caused by nonsense mutations. These mutations result in the premature termination of essential protein synthesis, leading to various disorders, such as Duchenne Muscular Dystrophy, where the dystrophin protein is not fully formed. Translarna is licensed in several countries for treating ambulatory patients aged two years and older with nonsense mutation Duchenne Muscular Dystrophy (nmDMD). In the United States, ataluren is still considered an investigational drug.

Duchenne Muscular Dystrophy is a rare, fatal genetic disorder predominantly affecting males, characterized by progressive muscle weakness beginning in early childhood. This condition typically results in loss of ambulation by age 10, followed by loss of arm function, and severe heart and respiratory issues leading to premature death in the mid-20s. The disease is caused by the absence of the functional dystrophin protein, which is crucial for muscle stability, including the skeletal, diaphragm, and heart muscles.

PTC Therapeutics, Inc. is a global biopharmaceutical company dedicated to developing and commercializing medicines for rare disorders. The company’s foundation is its ability to innovate, identify new therapies, and bring products to market globally. PTC’s mission is to provide access to top-tier treatments for patients with limited or no treatment options. The company leverages its scientific and clinical expertise and global commercial infrastructure to deliver therapies, aiming to maximize value for all stakeholders.

PTC Therapeutics continues to focus on providing best-in-class treatments and is committed to ensuring that patients with rare disorders have access to necessary medications. The company’s strategy involves robust investment in a diversified pipeline of transformative medicines to address unmet medical needs.