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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