In blow to Shire, Roche bags an FDA OK for its block­buster he­mo­phil­ia A prospect

Guy Young, USC Keck School of Med­i­cine. Im­age: USC

De­spite be­ing dogged by ques­tions about safe­ty and an un­usu­al le­gal coun­ter­at­tack from ri­val Shire, Roche to­day scored an FDA ap­proval for emi­cizum­ab (ACE910), its he­mo­phil­ia A ther­a­py which will now hit the mar­ket as Hem­li­bra.

The drug is de­signed to bring to­geth­er fac­tor IXa and fac­tor X pro­teins to re­store the blood clot­ting process for he­mo­phil­ia A pa­tients, re­duc­ing the threat of bleeds. And an­a­lysts have pegged po­ten­tial sales at up to $5 bil­lion, though Reuters‘ most re­cent con­sen­sus was a con­sid­er­ably less frothy $1.6 bil­lion.

Sig­nif­i­cant­ly, the ap­proval comes more than three months ahead of its late-Feb­ru­ary PDU­FA date, reg­is­ter­ing as the 40th new mol­e­c­u­lar en­ti­ty of the year as the in­dus­try looks ready to cruise past the cur­rent-era record 44 new ap­provals achieved at CDER in 2015.

The ap­proval was based on some im­pres­sive da­ta on a drug that Roche has stead­fast­ly main­tained is a near cer­tain block­buster-to-be. But it al­so comes with a boxed warn­ing on a threat of blood clots and dam­age to blood ves­sels, which could al­so crimp sales.

Last sum­mer the phar­ma gi­ant laid out da­ta de­tail­ing how the drug re­duced the rate of treat­ed bleeds by 87%, hit­ting the pri­ma­ry end­point, the drug al­so hand­i­ly outscored by­pass­ing agents like Shire’s. Their ther­a­py slashed “on-de­mand BPAs across all bleed mea­sure­ments, in­clud­ing ze­ro treat­ed bleeds (62.9 per­cent vs. 5.6 per­cent), ze­ro treat­ed spon­ta­neous bleeds (68.6 per­cent vs. 11.1 per­cent), ze­ro treat­ed joint bleeds (85.7 per­cent vs. 50.0 per­cent), ze­ro treat­ed tar­get joint bleeds (94.3 per­cent vs. 50.0 per­cent) and ze­ro bleeds over­all, which in­cludes all treat­ed and non-treat­ed bleeds (37.1 per­cent vs. 5.6 per­cent).”

Along the way, Roche blamed Shire’s by­pass­ing agents for throm­boem­bol­ic events that they saw in their study, ad­vis­ing doc­tors to stop us­ing FEI­BA.

Shire, to put it mild­ly, flipped.

“To im­ply a cause-and-ef­fect of FEI­BA hav­ing caused the se­vere ad­verse events is mis­lead­ing,” Shire hema­tol­ogy chief Ju­liana Dierks told Reuters. “We are look­ing for­ward to trans­paren­cy. Give us the da­ta, give us the facts.”

Shire al­so claimed that Roche has been mis­lead­ing­ly of­fer­ing a look at “treat­ed bleeds” — a sec­ondary end­point — in­stead of the “num­ber of bleeds over time,” orig­i­nal­ly laid out as the pri­ma­ry end­point in HAVEN 1. And by blam­ing throm­boem­bol­ic in­ci­dents in the study — with one pa­tient dy­ing of a rec­tal he­m­or­rhage — on its by­pass­ing agent, Shire al­so main­tained that it had been dam­aged. Shire sought an in­junc­tion against Roche in Ger­many, but the ac­tion was re­ject­ed by the court and the com­plaint hasn’t slowed its Big Phar­ma ri­val at all.

How bad could this get for Shire? Bern­stein an­a­lysts say Hem­li­bra along with new ther­a­pies from No­vo Nordisk and Bay­er could shrink its share of the he­mo­phil­ia mar­ket from 49% to 29%

Roche has nev­er done any­thing but shrug the as­sault off. The phar­ma gi­ant is los­ing patent pro­tec­tion on three fran­chise drugs, and it needs to field ma­jor new drugs to fill the gap.

Hem­li­bra is priced to do just that. Roche, like a grow­ing num­ber of com­pa­nies, is us­ing an av­er­age whole­sale price based on av­er­age weight. At an av­er­age weight of about 127 pounds, Roche says, the cost “is ap­prox­i­mate­ly $482,000 for the first year of treat­ment and then ap­prox­i­mate­ly $448,000 per year, which is less than half of the WAC of the on­ly oth­er ap­proved pro­phy­lac­tic treat­ment for these pa­tients.”

The price fits in at the low­er rung of the list of the 10 most ex­pen­sive drugs in the world, all among the rare dis­ease crowd.

“To­day’s ap­proval pro­vides a new pre­ven­ta­tive treat­ment that has been shown to sig­nif­i­cant­ly re­duce the num­ber of bleed­ing episodes in pa­tients with he­mo­phil­ia A with Fac­tor VI­II in­hibitors,” said Richard Paz­dur, whose ti­tles in­clude act­ing di­rec­tor of the Of­fice of Hema­tol­ogy and On­col­o­gy Prod­ucts. “In ad­di­tion, pa­tients treat­ed with Hem­li­bra re­port­ed an im­prove­ment in their phys­i­cal func­tion­ing.”

“Peo­ple with he­mo­phil­ia A who de­vel­op in­hibitors face sig­nif­i­cant chal­lenges pre­vent­ing bleeds and typ­i­cal­ly re­quire in­fu­sions of med­i­cine mul­ti­ple times a week, which can be es­pe­cial­ly dif­fi­cult for young chil­dren and their fam­i­lies,” said Guy Young, di­rec­tor of he­mo­sta­sis and throm­bo­sis pro­gram, Chil­dren’s Hos­pi­tal Los An­ge­les, and pro­fes­sor of pe­di­atrics, Uni­ver­si­ty of South­ern Cal­i­for­nia Keck School of Med­i­cine. “This new med­i­cine has been shown to re­duce the fre­quen­cy of bleeds com­pared to the cur­rent­ly avail­able med­i­cines and on­ly needs to be in­ject­ed once a week. This could make a mean­ing­ful dif­fer­ence for these chil­dren.”

Im­age: Roche, John Mc­Carter

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Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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Seagen interim CEO Roger Dansey and Daiichi Sankyo CEO Sunao Manabe

Paving the way for Mer­ck­'s buy­out, Seagen los­es ar­bi­tra­tion dis­pute with Dai­ichi over ADC tech

As Seagen awaits a final buyout offer from Merck that could be in the territory of $40 billion, Seagen revealed Friday afternoon that it lost an arbitration dispute with Daiichi Sankyo relating to the companies’ 2008 collaboration around the use of antibody-drug conjugate (ADC) technology.

But that loss likely won’t matter much when it comes to Merck’s deal.

After breaking off its pact with Daiichi in mid-2015, the two companies battled over “linker” tech — a chemical bridge between an ADC’s antibody component and the cytotoxic payload — that Seagen claims Daiichi would improve upon and implement in its current generation of ADCs.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

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CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

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No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

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