EU regulators have given their approval for a drug which will treat a particular form of muscular dystrophy. The approval comes as a special measure which allows the drug to be given to patients who have no alternative in life threatening conditions.
European regulators have approved the first ever drug for treating a rare form of muscular dystrophy which affects children.
The ailment is named Duchenne muscular dystrophy and happens in children who suffer because they lack a specific protein named dystrophin. The new drug Translarna also known as ataluren can treat Duchenne muscular dystrophy by helping the human body create dystrophen to help prevent muscle impairment.
The approval of the new drug for Duchenne muscular dystrophy by the European Medicines agency has been hailed as historic. The approval was successful through a special exemption known as conditional marketing authorization, which enables sale of medicines in life threatening ailments and after all treatment options has been exhausted.
Earlier the agency had turned down the request for approval in January and has reviewed its decision after a reassessment of the evidence and new data from the U.S. maker of the drug, PTC Therapeutics of Plainfield, N.J.
At present there is no available treatment option for the ailment which mainly affects boys and is caused by an anomaly in genes which leads to a premature end in production of protein dystrophin. The new drug will give the ability to the protein producing part of the cells to ignore the genetic defects and resume producing the proteins.
Robert Meadowcroft of the Muscular Dystrophy Campaign in Britain hailed the decision and said, “This decision by the EMA is fantastic news. Most of those diagnosed with Duchenne muscular dystrophy, usually before the age of five, will use a powered wheelchair before they are 12, will not have the muscle strength to pick up a glass of water by the age of 20 and will not live to see their 30th birthday.”
EU’s decision will pave way to make the drug Translarna available across 28 nations within the European Union.