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

Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; and more

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.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

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The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.

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Massachusetts-based iVexSol has secured funding to the tune of $23.8 million in its latest Series A round. The new investors include Bristol Myers Squibb, manufacturer Charles River Laboratories and Asahi Kasei Medical.

iVexSol is a manufacturer of lentiviral vectors (LVV), used in making gene therapies, and this latest round of fundraising brings its total Series A total over $39 million, which will be used to recruit more employees and bolster its technology.

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John Rim, Samsung Biologics CEO (Samsung/PR Newswire)

Sam­sung Bi­o­log­ics spells out ex­pan­sion plans in South Ko­rea and US

The CDMO arm of one of South Korea’s largest conglomerates has posted its year-end results and plans for 2023, which include new construction.

Samsung Biologics netted north of KRW 3 trillion ($2.4 billion) in 2022 revenue and an operating profit of KRW 983.6 billion ($799 million), which the company touted on Friday as “record-high earnings.” The revenue boost was 55% compared to 2021.

No­var­tis' ap­proved sick­le cell dis­ease drug fails to beat place­bo in PhI­II

Novartis’ sickle cell drug, approved in 2019 and branded as Adakveo, has failed an ongoing Phase III, according to preliminary results.

The Swiss pharma giant unveiled early data from the ongoing STAND Phase III study on Friday, saying that crizanlizumab showed no statistically significant difference between the drug at two different dose levels compared to placebo in annualized rates of vaso-occlusive crises that lead to a healthcare visit over the first year since being randomized into the trial.

Sen. Elizabeth Warren (D-MA) (Michael Brochstein/Sipa USA/Sipa via AP Images)

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Sen. Elizabeth Warren (D-MA) is calling on senior Federal Trade Commission officials to “closely scrutinize” two proposed pharma mergers.

Warren expressed concern over “rampant consolidation in the pharmaceutical industry,” in particular Amgen’s $28 billion plans to take over Horizon Therapeutics, and Indivior’s proposed acquisition of Opiant for $145 million upfront, in a letter to FTC Chair Lina Khan and Commissioners Alvaro Bedoya and Rebecca Kelly Slaughter earlier this week.