Pharmaceutical Business review

UK’s NICE recommends PTC Therapeutics’ Translarna for Duchenne muscular dystrophy

Under the recommendation, Translarna would be made available as part of a confidential financial agreement between NHS England and PTC.

The regulator noted that PTC should an acceptable price for the guidance to be put in place and the drug would be provided for five years under a managed access agreement permitting PTC to collect additional efficacy data.

In one clinical trial, none of the children in the most sensitive group taking Translarna lost the ability to walk over the 48 weeks of the trial compared with 8% on the placebo. The research predicts that it could delay loss of walking for about seven years.

PTC Therapeutics CEO Stuart Peltz said: "We are extremely pleased by the NICE recommendation, which recognizes Translarna as an innovative medicine with the potential to change the course of this devastating disease.

"This is a crucial step for the boys and young men in England with nonsense mutation DMD."

Translarna is a protein restoration therapy designed to allow the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.

The European Commission approved Translarna in August 2014 to treat nmDMD. The drug is presently available to patients in 23 countries via either expanded access programs or commercial sales.

The US Food and Drug Administration granted orphan drug designation to Translarna for the treatment of both nmDMD and cystic fibrosis caused by a nonsense mutation.