PTC Therapeutics secures Russian approval for Translarna to treat Duchenne muscular dystrophy

PTC Therapeutics, a leading US-based biotechnology company, has received marketing approval in Russia for its innovative drug, Translarna (ataluren), designed to treat patients with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation. The approval, granted by the Ministry of Health of the Russian Federation, is a significant milestone for PTC Therapeutics and marks the first time a therapy targeting the underlying cause of nonsense mutation Duchenne muscular dystrophy has been made available in Russia.

This approval paves the way for the introduction of Translarna to patients aged two years and older who suffer from the devastating effects of nonsense mutation Duchenne muscular dystrophy. The condition, characterized by the absence of a critical protein—dystrophin—results from a genetic mutation that prematurely halts protein production. Translarna works by facilitating the production of a functional protein, thereby helping to restore some normal function for affected patients.

Expanding access to treatment for Duchenne muscular dystrophy

Stuart W. Peltz, CEO of PTC Therapeutics, expressed his excitement about the approval, stating that Translarna is “the first therapeutic ever approved for nonsense mutation Duchenne muscular dystrophy patients.” He further emphasized the importance of expanding the treatment’s availability into Russia, where there has been considerable interest from healthcare professionals eager to offer a solution to patients with this rare genetic condition.

Peltz noted that numerous patients in Russia have already been identified as potential candidates for the treatment, offering new hope to those affected by this life-limiting condition. “Our goal is to bring this product to nmDMD patients globally, and this approval marks another important milestone,” Peltz added. This statement underscores PTC Therapeutics’ ongoing commitment to addressing unmet medical needs in the field of genetic disorders, particularly those as devastating as Duchenne muscular dystrophy.

How Translarna helps combat genetic disorders

Translarna is a protein restoration therapy specifically designed to treat Duchenne muscular dystrophy in patients with a nonsense mutation. A nonsense mutation in the genetic code leads to the early termination of protein synthesis, which results in a dysfunctional or missing protein. In the case of Duchenne muscular dystrophy, the missing protein is dystrophin, which plays a crucial role in muscle function. By enabling the production of functional dystrophin, Translarna helps mitigate the progression of the disease, offering affected individuals a better quality of life.

Duchenne muscular dystrophy, a rare and severe condition, leads to progressive muscle weakness, loss of mobility, and eventually, respiratory and heart failure. The approval of Translarna in Russia is an important step toward ensuring that patients with this genetic disorder have access to effective treatments, potentially improving their long-term health outcomes.

Key takeaways

PTC Therapeutics’ recent approval for Translarna in Russia provides a glimmer of hope for Duchenne muscular dystrophy patients suffering from a rare genetic mutation. The treatment offers a novel solution for managing the underlying cause of the disease, helping patients restore some degree of normal protein function. With global expansion plans in place, Translarna is positioning itself as a key therapeutic option in the fight against genetic disorders, bringing hope to families affected by Duchenne muscular dystrophy around the world.