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.
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