Duchenne MD Drug Translarna Receives Managed Access Agreement for UK Patient Funding

PTC Therapeutics has successfully negotiated a Managed Access Agreement (MAA) with the National Health Service (NHS) in England for Translarna (ataluren) to treat ambulatory patients ages 5 and older with nonsense mutation Duchenne muscular dystrophy (nmDMD).

The decision provides reimbursed patient access to Translarna in England via a five-year MAA. The U.K.’s National Institute for Health and Care Excellence (NICE) had given a positive recommendation of Translarna in April 2016, subject to PTC and the NHS finalizing the terms of the Managed Access Agreement. Final guidance from NICE is expected later this month after the execution of the agreement, with implementation to come soon after.

In the United States, Translarna is an investigational new drug and has not yet been approved by the FDA for DMD. It has been granted FDA orphan drug designation for three diseases: DMD, cystic fibrosis, and mucopolysaccharidosis I, which is caused by a deficiency in an essential enzyme responsible for the breakdown of byproducts in cells.

Translarna is a protein restoration drug designed to enable the formation of a new, functional protein in patients with genetic conditions caused by nonsense mutations – a modification in the genetic code that stops the synthesis of an essential protein. The resulting genetic disorder is determined by which protein cannot be fully expressed and is no longer functional, such as dystrophin, in DMD.

“This is an important day in England for children and young adults suffering from DMD,” PTC Therapeutics CEO Stuart W. Peltz, Ph.D., said in a press release. “We are extremely pleased to have reached a successful outcome with NHS England, which will provide long-awaited access to Translarna for patients with nonsense mutation DMD.”

Peltz said PTC is grateful to the patients, families, advocacy groups, and physicians for their support of the company during “this important and rigorous access process.”

The development of Translarna has been supported by funding from organizations such as Cystic Fibrosis Foundation Therapeutics Inc., the Muscular Dystrophy Association, the FDA’s Office of Orphan Products Development, and Parent Project Muscular Dystrophy, among others.

The drug received marketing authorization from the European Commission to treat nmDMD in 2014, and is currently under an annual review by the European Medicines Agency (EMA) with a renewal decision expected this year. Patients in more than 20 countries have access to this drug either commercially or through expanded access programs.