NICE re­jects No­var­tis' mi­graine drug Aimovig, months af­ter de­clin­ing to en­dorse pricey Kym­ri­ah for adult use

No­var­tis $NVS is will­ing to play ball with UK’s cost-ef­fec­tive­ness watch­dog NICE, who re­ject­ed their mi­graine pre­ven­tion drug in a draft guid­ance on Thurs­day, months af­ter de­clin­ing to en­dorse the Swiss drug­mak­er’s cell ther­a­py Kym­ri­ah in adult lym­phoma pa­tients.

The drug, erenum­ab, is sold as Aimovig in the Unit­ed States by No­var­tis and part­ner Am­gen $AMGN. It forms part of a new crop of bi­o­log­ics tar­get­ing the CGRP pro­tein that trans­mits pain sig­nals in­to the brain, and is con­sid­ered in­stru­men­tal in gen­er­at­ing and main­tain­ing headaches as­so­ci­at­ed with mi­graine. Erenum­ab costs around £5,000 (about $6368) per year, ex­clud­ing a con­fi­den­tial dis­count that would have been grant­ed to Eng­land’s na­tion­al health ser­vice (NHS) had NICE rec­om­mend­ed the drug.

In its re­port, NICE ac­knowl­edged that al­though the drug is a clin­i­cal­ly ef­fec­tive treat­ment, the to­tal­i­ty of the da­ta sup­port­ing the med­i­cine did not in­clude all “rel­e­vant” com­par­isons against ex­ist­ing drugs and out­comes, which prompt­ed its re­jec­tion for NHS use.

Mein­dert Boy­sen

“There was not enough ev­i­dence to sug­gest that it (erenum­ab) is more ef­fec­tive than bot­u­linum tox­in type A (Al­ler­gan’s Botox) for peo­ple with chron­ic mi­graine, which NICE al­ready rec­om­mends. And for both the chron­ic and episod­ic mi­graine pop­u­la­tions there was no ev­i­dence to show that erenum­ab is ef­fec­tive in the long-term in peo­ple for whom 3 pre­vi­ous pre­ven­tive treat­ments had failed,” said NICE’s di­rec­tor of the cen­tre for health tech­nol­o­gy eval­u­a­tion Mein­dert Boy­sen in a state­ment.

No­var­tis pledged to work with NICE to ad­dress the agency’s ques­tions, but sug­gest­ed the watch­dog had not cap­tured the broad­er so­ci­etal im­pact of its med­i­cine.

“NICE does not con­sid­er the im­pact of dis­eases on broad­er so­ci­ety when eval­u­at­ing the cost-ef­fec­tive­ness of med­i­cines. We be­lieve this fails to cap­ture the true val­ue of med­i­cines like Aimovig. As mi­graine pre­dom­i­nant­ly af­fects peo­ple of work­ing age, it costs the UK econ­o­my £8.8 bil­lion per year in lost pro­duc­tiv­i­ty alone,” the drug­mak­er said in a state­ment, un­der­scor­ing that the UK al­ready “lags sig­nif­i­cant­ly be­hind” its oth­er Eu­ro­pean coun­ter­parts in en­sur­ing ac­cess to new med­i­cines.

It is es­ti­mat­ed there are 190,000 mi­graine at­tacks ex­pe­ri­enced every day in Eng­land, ac­cord­ing to NICE. The agency con­ced­ed that ex­ist­ing mi­graine treat­ments in­clud­ing be­ta-block­ers, an­ti­de­pres­sants and epilep­sy med­i­cines can have sig­nif­i­cant side-ef­fects and don’t work for some pa­tients, but for those with chron­ic mi­graine for whom at least 3 pre­vi­ous pre­ven­tive oral treat­ments have failed, Botox re­mains an op­tion.

The pro­vi­sion­al de­ci­sion on erenum­ab comes af­ter the agency re­fused to en­dorse No­var­tis’ CAR-T drug Kym­ri­ah in adults, say­ing it was too ex­pen­sive, in Sep­tem­ber, al­though it did rec­om­mend the treat­ment’s use in pe­di­atric pa­tients in a pre­vi­ous rul­ing.

UP­DAT­ED: Roche bags 'break­through' an­ti-fi­bro­sis drug in $1.4B biotech buy­out deal

Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

The pharma giant announced Friday that it is acquiring Promedior, primarily to get its hands on PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that has nailed down mid-stage clinical data on idiopathic pulmonary fibrosis and demonstrating its potential for a range of fibrotic conditions.

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Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

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No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.

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No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

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As­traZeneca gains EU nod for di­a­betes triple; Am­gen and Duke launch re­al-world PC­SK9 ob­ser­va­tion­al study

→ Weeks after winning EU approval to start marketing dapagliflozin as Forxiga, AstraZeneca has racked up another OK for a triplet combo involving the SGLT2 diabetes drug. Named Qtrilmet, the pill combines Forxiga with the DPP-4 inhibitor Onglyza (saxagliptin) and the bedrock drug metformin in a modified-release format. That 3-in-1 approach proved superior in reducing average blood glucose levels to a number of other dual combinations across 5 Phase III trials, including Forxiga plus metformin, Onglyza with metformin, or glimepiride with metformin.

Five drugs, in­clud­ing two No­var­tis ther­a­pies, win EMA en­dorse­ment

As is custom, an EMA panel on Friday issued its weekly recommendations on marketing applications submitted by drug developers. This week, the agency backed the use of five new therapies — including two Novartis drugs — but issued no negative reviews.

Novartis’ S1P drug for relapsing forms of multiple sclerosis (MS) drug, Mayzent (known chemically as siponimod), which was approved by the FDA in March — has been given the nod by the EMA. The Swiss drugmaker already sells its other MS drug, Gilenya, in both regions.

Atom­wise's X-37 spin­out gets $14.5 mil­lion to launch AI dis­cov­ery ef­forts

The folks behind Atomwise’s spinout X-37 like to think in cosmological metaphors, and you can think of their AI drug development model as probes sent into space from a central station. That station just got $14.5 million in Series A funding from DCVC Bio, Alpha Intelligence Capital and Hemi Ventures to back those missions.

X-37 uses Atomwise’s AI platform to identify drug targets and – unlike the parent company, which largely sticks to computers  – bring those into a wet lab and preclinical testing.  In addition to AI professionals, it’s led in by part by drug developers from Velocity Pharmaceutical Development.

Ab­bott Lab­o­ra­to­ries CEO Miles White pass­es ba­ton down to suc­ces­sor; Lon­za CEO Marc Funk hits the ex­it

→ Abbott Laboratories has named a successor to CEO Miles White after he announced that he was stepping down in March after 21 years of service. Robert Ford, the company’s COO and president, will take the helm. Ford is known for his work in the $25 billion merger between St. Jude Medical into Abbott in January 2017. White will remain with the company as executive chairman of the board. 

→ After snapping up Novartis’ Swiss facility, Novartis Center of Excellence, in July, Lonza has announced that their CEO, Marc Funk, is hitting the exit for “personal reasons.” Funk has been the CEO of the company for less than a year — brought onto the company back in March. In the meantime, chairman Albert Baehny will serve as interim CEO. 

UCB adds on more pos­i­tive PhI­II da­ta for IL-17A/17F in­hibitor bimek­izum­ab, clear­ing a path to the FDA

A month after posting positive top-line data from their first Phase III trial of the IL-17A/17F inhibitor bimekizumab, Belgium’s UCB says they’ve added more upbeat results from their second late-stage test in moderate-to-severe plaque psoriasis.

That leaves the company on track for regulatory submissions in the middle of next year, says CMO Iris Loew-Friedrich.
Their drug beat out a placebo on the co-primaries — a 90% improvement in PASI 90 (the Psoriasis Area and Severity Index) and Investigator Global Assessment (IGA) response of clear or almost clear (IGA 0/1) at week 16, compared to placebo. Investigators also boasted of hitting some key secondaries.
UCB is angling to enter an increasingly crowded market space.
In their first of 3 Phase III studies for bimekizumab, researchers touted top-line wins on statistically significant results on clearing plaque psoriasis, including a victory over J&J’s IL-23 contender Stelara on key endpoints. The drug targets both IL-17A and IL-17F, a modification on the IL-17A strategy laid out for Taltz (Eli Lilly) and Cosentyx (Novartis). And the new group also includes J&J’s Tremfya and AbbVie’s Skyrizi.

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