European Union regulators say they have given approval for the first-ever drug for treating a particular form of muscular dystrophy that affects children.
Translarna, also identified under the name ataluren, treats Duchenne muscular dystrophy by helping the human body create a protein known as dystrophin to protect against muscle impairment experienced by children who suffer from the disease because they lack that protein.
The approval by the European Medicines Agency, which medical experts have deemed “historic,” was granted under a special exemption known as a conditional marketing authorization, intended to allow for medicines that may combat life-threatening illnesses when there are no other drug options available.
The agency, which had denied approval in January, said it was issuing the new decision after a re-examination of the evidence and new data from the U.S. manufacturer of the drug, PTC Therapeutics of Plainfield, N.J.
There are currently no other treatments for the disease, which primarily effects boys and is the result of a mutational defect in genes that prematurely ends production of dystrophin, experts say.
The newly approved drug is believed to give protein-making parts of cells the ability to “skip over” such genetic defects so the cells can resume producing the protein.
“This decision by the EMA is fantastic news,” says Robert Meadowcroft of the Muscular Dystrophy Campaign in Britain.
“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.”
The European Union approval will allow Translarna to be made available in the 28 countries that are Member States of the European Union.
The Muscular Dystrophy Campaign said the drug needed to reach those children with the particular form of Duchenne muscular dystrophy that could respond to it as quickly as possible, and said the European Union approval means Translarna could be available in the United Kingdom within six months.
The drug would be of most benefit to children five years of age and above whose disease has not progressed past the point where they are unable to walk, the E.U. regulators said.