A drug for treating a particular form of Duchenne muscular dystrophy has surprisingly got approval from the European Medicines Agency.
The Muscular-dystrophy drug results in rise of stock up to 50% in early afternoon. Earlier the stock rose more than 70%.
The drug is presumed to be in stock in UK in six months.
On the basis of phase-two data the Committee for Medicinal Products for Human Use (CHMP), the advisory panel of the European Medicines Agency, commended conditional approval i.e. it still requires confirmation for the previous studies of PTC’s drug Translarna.
“This is a historic day for the DMD community,” Craig MacDonald, a professor of medicine at the University of California-Davis, said.
The patients who will benefit the most are those aged five years and over who are still able to walk.
The reason behind the change of mind of the agency is not disclosed as it rejected the application earlier in January this year only; however on Friday morning on a conference call with analysts, PTC CEO Stuart Peltz mentioned about “educating” the agency about this rare disease and the nature of the clinical trial.
“Our discussions during the review process highlighted the urgent need for treatment options, the expectation of a disciplined restoration therapy and the importance of the natural-history data, the safety profile of the drug and the meaningfulness of the results that we obtained from our trial,” Peltz said.
Duchenne muscular dystrophy is an inherited disorder that causes muscle weakness, which quickly worsen. DMD occurs because the mutated gene fails to produce virtually any functional dystrophin(a protein in the muscles). About 1 in 3,500 boys in the UK are born with DMD.The drug is advised for only those whose condition is caused by a particular ‘nonsense mutation’ – namely 200 children.
“This decision by the EMA is fantastic news, said Robert Meadowcroft, chief executive of the campaign.
“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.
“This step forward for Translarna offers much needed hope.”