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.

Per NICE:

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.

Roche announced another leadership shuffle Thursday morning – the head of global product strategy, Teresa Graham, will take over as CEO of Roche Pharmaceuticals in March while the company’s corporate executive committee will make a spot for Levi Garraway, CMO and executive VP of global product development.

Thomas Schinecker will take over the top spot as Roche group CEO in March, leaving his spot as head of diagnostics.

Sanofi expects its RSV antibody jointly developed with AstraZeneca will be available next season, executive VP of vaccines Thomas Triomphe announced on the company’s quarterly call.

Beyfortus, also known as nirsevimab, was approved in the EU back in November and is currently under FDA review with an expected decision coming in the third quarter of this year. The news comes as the FDA plans to hold advisory committee meetings over the next couple months to review RSV vaccines from Pfizer and GSK.

After a whopping $4 billion asset buy from Nimbus Therapeutics, along with a $400 million deal with Hutchmed for a colorectal cancer drug, Takeda executives touted pipeline optimism on its latest earnings call this week.

That’s because the TYK2 inhibitor for psoriasis Takeda is getting from Nimbus, along with the Hutchmed fruquintinib commercialization outside of China, are just two of what it reports are 10 late-stage development programs of promising candidates.

Regeneron faced a substantial slump in overall revenue last year, but the focus still remains on some of its biggest blockbusters.

The pharma with several high-profile partnerships — Sanofi and Bayer among them — said Friday that Q4 revenue was down 31% for the quarter, and down 24% for the entire year. However, that won’t stop blockbuster expansion plans.

One of those is Eylea, the Bayer-partnered eye disease drug that has been in major competition with Roche’s Vabysmo. While Eylea is currently only approved in a 2 mg dose, the company recently filed for approval to give a 8 mg dose, in hopes of making a longer-lasting treatment.