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.

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