Translarna vs. Duchenne myodystrophy
A drug for the treatment of muscular dystrophy has been approved in Europe
As reported in the press release of the agency, European Medicines Agency recommends first-in-class medicine for treatment of Duchenne muscular dystrophy, the drug will be recommended to patients five years and older who are able to move independently.
Duchenne muscular dystrophy (MDD) is a hereditary disease in which muscle weakness gradually develops, leading to a deterioration of motor abilities, a progressive decrease in limb strength and the development of heart and lung failure. This disease affects only boys and is the most common type of muscular dystrophy. As a rule, MDD occurs in about one child out of three thousand. Patients have a deficiency of the protein dystrophin, which protects the muscles from damage, as a result of which the muscles cease to function normally over time. To date, there has been no adequate therapy aimed at preventing and treating complications.
Ataluren is the first drug developed to restore impaired functional protein synthesis in patients with a gene mutation in the sex X chromosome that causes impaired dystrophin synthesis: PTC Therapeutics Receives Positive Opinion From CHMP For Translarna™ (Ataluren).
Structural formula of ataluren – VM
The decision of the expert commission was made after studying the data of clinical trials involving 174 patients. According to the results obtained, patients receiving ataluren at a dosage of 40 mg / kg of body weight daily for 48 weeks, during the test with a six-minute walk, overcame 31.3 meters more compared to patients taking a placebo. According to the researchers, no serious side effects of the drug were noted during clinical trials.
After obtaining permission to register and sell the drug ataluren, the share price of the American biotech company PTC Therapeutics, which developed the drug, doubled. It is expected that the final decision on the investigated drug will be made by the European Commission in the next three months.
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