Dive Brief:
- PTC Therapeutics and England's National Health Service (NHS) reached a managed access agreement deal to cover Translarna (ataluren), a treatment for certain patients with Duchenne muscular dystrophy (DMD), the company announced Thursday.
- The National Institute for Health and Care Excellence (NICE) recommended in April that NHS England make the drug available to patients if the agency could reach a cost deal with PTC. The deal reached includes a confidential financial arrangement and a five-year review on the efficacy of Translarna for DMD patients.
- Translarna is a protein restoration therapy designed to treat patients with a subset of DMD. The drug was conditionally approved by the European Commission in August 2014 and is subject to EMA review and renewal this year.
Dive Insight:
The market agreement is a big win for PTC, but it could be short-lived: 2016 has seen Translarna spurned in the U.S. and Scotland, and the EMA is set to decide on whether to renew the drug's conditional approval this year.
The regulatory consensus seems to be that the drug is promising, but severely lacking in data.
The FDA informed PTC that it could not submit Translarna since "both the Phase 2b and ACT DMD trials were negative and do not provide substantial evidence of effectiveness," the company announced in February. Meanwhile, in April the Scottish Medicines Consortium refused to license the drug for NHS Scotland's use in April.
Yet it was that same month that NICE recommended the drug for coverage by the NHS England, citing a clinical trial in which, over the course of 48 weeks, 0 of 47 children lost the ability to walk when treated with the drug, compared to 4 of 52 on the placebo. Researchers suspect the drug could delay loss of walking, although the evidence supporting that conclusion appears thin.
Translarna is conditionally licensed in Europe to treat nonsense mutation DMD in children over five who can walk. According to NICE's April recommendation, an access agreement would help around 50 children with DMD over the five-year term.
PTC has been hit hard since the FDA in February said its application for U.S. approval of Translarna was incomplete. The company laid off roughly 18% of its staff, mostly in the U.S., one month later and its stock is currently trading around 25% of its value prior to the FDA's decision.