UK's NICE stamps approval on Alnylam, PTC rare disease drugs
The UK’s National Institute for Health and Care Excellence (NICE) is playing nice with Alnylam and PTC Therapeutics, endorsing rare disease treatments from both companies.
The public health body has recommended coverage of Alnylam’s Amvuttra for hereditary transthyretin-related amyloidosis and PTC’s Translarna for Duchenne muscular dystrophy Thursday.
Vutrisiran, also known as Amvuttra, was FDA-approved just last year for the indication, four years after Alnylam’s other hATTR amyloidosis drug Onpattro. Amvuttra is administered once every three months under the skin, while Onpattro is given via IV infusion once every three weeks.
A media advisory from NICE pointed out that hATTR is estimated to affect only around 150 people in the UK. And while vutrisiran currently costs $118,000 per injection or $472,000 per year at the recommended dose, Alnylam has a “confidential commercial” agreement that makes the drug available to the NHS at a discount. That exact discount is still undisclosed.
If there are no objections to NICE’s guidance, it will be finalized in February, the agency said.
NICE also issued a positive recommendation for PTC Therapeutics’ therapy ataluren, branded Translarna. Though the DMD therapy has been approved outside the US, it has struggled in its attempt to win an FDA OK.
PTC Therapeutics tells Endpoints News that the company, beyond the obvious pleasure with the recommendation, follows NICE’s commitment to re-evaluate the therapy. NICE said in a statement that the drug has been evaluated under a managed access agreement for the last six years, ending Friday, allowing around 60 children to have taken ataluren while more efficacy data were collected. The agency said it has reviewed that data, along with real-world evidence and feedback from clinicians and patients.
It suggests that ataluren is likely to slow down disease progression and delay the time at which the ability to walk is lost. Evidence for improvements in later stages of the disease and improved survival with ataluren is limited and highly uncertain. However, the committee agreed that ataluren may also improve outcomes, such as preventing scoliosis and maintaining upper body strength, once the ability to walk has been lost.