The National Institute for Health and Care Excellence has recommended PTC Therapeutics’ Translarna for some patients with Duchenne muscular dystrophy, but only in connection with a managed access agreement (MAA) being drawn up with NHS England.

The cost watchdog is supporting the drug’s use for ambulatory patients aged five years and older with nonsense mutation Duchenne muscular dystrophy (nmDMD), a progressive muscle disorder caused by the lack of functional dystrophin protein.

The Institute noted that, in one clinical trial, none of the children in the most sensitive group taking the drug lost the ability to walk over the 48 weeks of the trial compared with 8 percent on the placebo, and that research predicts that it could delay loss of walking for up to seven years.

But the drug comes at a high price, at £222,000 per year (before discount). “Because of its very high cost, it is important that details of the financial and other arrangements to enable this new medicine to be offered to patients on the NHS are discussed and agreed between the company and NHS England, and set out in a managed access agreement,” said Sir Andrew Dillon, NICE’s chief executive.

PTC and NHS England are currently finalising an MAA outlining financial and clinical details surrounding the use of Translarna (ataluren), including a confidential financial arrangement. This is expected to allow PTC to collect further data on the drug’s efficacy over a five-year period, with NICE guidance to be reviewed again after this timeframe. 

“This decision is a hugely encouraging sign that both NICE and NHS England have listened to the patient community, bringing hope to each and every parent and patient fighting DMD,” said Paul Lenihan, Chief Executive of Action Duchenne. 

Removal from market?

The news is certainly a positive but analysts at RBC Capital Markets fear it could be short-lived, as the drug’s conditional approval in Europe is now looking shaky after on the failure of a confirmatory Phase III trial, which they believe means there is a 50% probability that it is removed from the market.

Translarna won conditional approval from the European Commission in August 2014 to treat nmDMD, and is currently available to patients in 23 countries through either expanded access programs or commercial sales. 

However, earlier this month the Scottish Medicines Consortium turned down the drug’s use by NHS Scotland, because of “too much uncertainty about the overall clinical benefits it might bring in relation to its cost”, again highlighting the postcode lottery of care for UK patients. 

Also, US regulators have refused to accept a marketing application for the drug, on grounds that it was “not sufficiently complete to permit a substantive review”.