Bio­Marin nets con­di­tion­al ap­proval for val­rox in Eu­rope, mark­ing the first he­mo­phil­ia gene ther­a­py

As Bio­Marin pre­pares to head back to the FDA with its he­mo­phil­ia A gene ther­a­py, the EU is sail­ing for­ward at full speed ahead.

The Eu­ro­pean Com­mis­sion grant­ed con­di­tion­al ap­proval to the com­pa­ny’s val­oc­toco­gene rox­a­parvovec — more col­lo­qui­al­ly known as val­rox — Wednes­day af­ter­noon, mak­ing it the first he­mo­phil­ia A gene ther­a­py ap­proved any­where. Val­rox, to be brand­ed on the con­ti­nent as Roc­ta­vian, will treat adults with se­vere he­mo­phil­ia A who need reg­u­lar treat­ments to pre­vent bleed­ing episodes.

There’s lim­it­ed in­fo so far about the time­line for con­fir­ma­to­ry da­ta. Bio­Marin said Wednes­day that it will pro­vide fol­low-up da­ta from ear­li­er stud­ies, as well as new re­sults from a con­fir­ma­to­ry tri­al, for which en­roll­ment has com­plet­ed. But the com­pa­ny did not say when it ex­pects to re­port such da­ta in a press re­lease.

In an email to End­points News, a Bio­Marin spokesper­son did not di­rect­ly an­swer ques­tions about when the com­pa­ny would re­port con­fir­ma­to­ry da­ta. But they of­fered clues hint­ing at when the re­sults might come. Of note, the spokesper­son says the con­fir­ma­to­ry da­ta re­quired in­clude:

  • 5 years of da­ta from the Phase 3 study, and Bio­Marin will reach year three this No­vem­ber 2022.
  • 2 years of da­ta from the steroid study, and year one da­ta is ex­pect­ed in 1Q23.

Wednes­day’s ap­proval fol­lows a pos­i­tive CHMP opin­ion in June, with the EMA say­ing that the ther­a­py pro­vid­ed some ben­e­fit for pa­tients more than two years down the road, as well as for a few pa­tients at least five years post-treat­ment. But reg­u­la­tors hedged at the time, not­ing they don’t know for sure how long the ef­fects will last.

The con­fir­ma­to­ry da­ta are os­ten­si­bly ex­pect­ed to help an­swer that key ques­tion. For now, though, Bio­Marin is tout­ing tri­al re­sults show­ing the ther­a­py re­duced bleed­ing episodes by 95% over four years.

Val­rox was ini­tial­ly hand­ed down a CRL back in Au­gust 2020 af­ter the FDA re­quest­ed more fol­low-up da­ta. The move came as a sur­prise both to the com­pa­ny and to an­a­lysts, as reg­u­la­tors sought an ad­di­tion­al two years of re­sults show­ing whether or not the ther­a­py was durable.

With the Eu­ro­pean ap­proval now in hand, Bio­Marin says it ex­pects to re-file its BLA by the end of next month. That time­line was pushed back three months in May af­ter the com­pa­ny said the FDA want­ed more in­for­ma­tion, though cru­cial­ly did not ask for any more clin­i­cal tri­als.

Though sub­mis­sions fol­low­ing a CRL typ­i­cal­ly take six months to re-re­view, the com­pa­ny al­so said Wednes­day it would not be sur­prised if the FDA takes longer than the stan­dard half-year pe­ri­od.

Dura­bil­i­ty has proven a key stick­ing point at the agency in re­cent months, with seem­ing­ly every gene ther­a­py de­vel­op­er po­si­tion­ing such da­ta more promi­nent­ly to bet­ter their ap­proval chances. And as safe­ty is­sues con­tin­ue to loom over the field, reg­u­la­tors ap­pear to be scru­ti­niz­ing such ap­pli­ca­tions clos­er and clos­er.

In Eu­rope, reg­u­la­tors plan to mon­i­tor pa­tients who take val­rox for 15 years to en­sure every­thing re­mains hunky-do­ry. The ther­a­py us­es an AAV vec­tor to de­liv­er the gene for fac­tor VI­II and is ex­pect­ed to car­ry the gene in­to the liv­er cells, help­ing the blood to clot more eas­i­ly to pre­vent or re­duce bleed­ing episodes.

Ed­i­tor’s note: This ar­ti­cle has been up­dat­ed to in­clude com­ment from a Bio­Marin spokesper­son. 

Illustration: Assistant Editor Kathy Wong for Endpoints News

How Pur­due's $272M ad­dic­tion pay­out fund­ed a new home for its dis­card­ed non-opi­oid re­search

Don Kyle spent more than 20 years working for Purdue Pharma, right through the US opioid epidemic that led to the company’s rise and eventual infamy. But contrary to Purdue’s focus on OxyContin, Kyle was researching non-opioid painkillers — that is, until the company shelved his research.

As the company’s legal troubles mounted, Kyle found an unlikely way to reboot the project. In 2019, he took his work to an Oklahoma State University center that’s slated to receive more than two-thirds of the state’s $272 million settlement with Purdue over claims that the drugmaker’s behavior ignited the epidemic of opioid use and abuse.

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President Joe Biden at the State of the Union address with Vice President Kamala Harris and House Speaker Kevin McCarthy (Patrick Semansky/AP Images)

The drug pric­ing pres­i­dent: Biden warns of ve­to for any IRA re­peal at­tempts

President Joe Biden made clear in his “finish the job” State of the Union address last night that one of those jobs to be finished is insulin prices.

Biden’s push again to tackle insulin prices, after Republicans rebuffed the idea last summer and just after Biden won Medicare drug price negotiations/caps via the Inflation Reduction Act, shows how heavily he’s leaning into this work.

Rupert Vessey, Bristol Myers Squibb head of research and early development

Up­dat­ed: R&D tur­bu­lence at Bris­tol My­ers now in­cludes the end of a $650M al­liance and the de­par­ture of a top re­search cham­pi­on

This morning biotech Dragonfly put out word that Bristol Myers Squibb has handed back all rights to its IL-12 clinical-stage drug after spending $650 million to advance it into the clinic.

The news arrives amid a turbulent R&D stage for the pharma giant, which late last week highlighted Rupert Vessey’s decision to depart this summer as head of early-stage R&D following a crucial three-year stretch after he jumped to Bristol Myers in the big Celgene buyout. During that time he struck a series of deals for Bristol Myers, and also shepherded a number of Celgene programs down the pipeline, playing a major role for a lineup of biotechs which depended on him to champion their drugs.

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FDA Commissioner Robert Califf on Capitol Hill, Feb. 8, 2023 (Drew Angerer/Getty Images)

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Califf, who stressed to the House Energy and Commerce’s Subcommittee on Health that the CDC also needs better data, made clear that the FDA’s ability to monitor the safety of vaccines “would also benefit greatly by a coordinated federal public health data reporting authority.”

Bill Haney, Dragonfly CEO (Dave Pedley/Getty Images for SXSW)

Drag­on­fly chief: Bris­tol My­ers shouldn’t blame IL-12’s clin­i­cal per­for­mance for de­ci­sion to scrap the deal — eco­nom­ics played a key role

Bristol Myers Squibb says the IL-12 drug they were developing out of Dragonfly Therapeutics was scrubbed from the pipeline for a simple reason: It didn’t measure up on clinical performance.

But Bill Haney, the CEO of Dragonfly, is taking issue with that.

The early-stage drug, still in Phase I development, has passed muster with Bristol Myers’ general clinical expectations, advancing successfully while still in Phase I, he says.

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Utpal Koppikar, new Verily CFO

Ex­clu­sive: Ver­i­ly wel­comes Atara Bio­ther­a­peu­tics vet­er­an as new CFO

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Utpal Koppikar joins Verily after a nearly five-year stint as CFO and senior VP at Atara, though his résumé also boasts roles at Gilead and Amgen.

The news follows a major reshuffling at Verily, including several senior departures earlier this year and a round of layoffs.

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Singer Nick Jonas is back at work for Dexcom, this time for its new G7 glucose monitor.

Dex­com's spokescelebri­ty Nick Jonas re­turns to Su­per Bowl in new glu­cose mon­i­tor com­mer­cial

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Jonas’ sleight-of-hand tricks populate the commercial — he pinches his empty fingers together and pops them open to reveal the small CGM — even as he ends the ad, saying, “It’s not magic. It just feels that way.” Jonas then disappears in a puff of smoke.

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Richard Francis, newly-appointed Teva CEO (Novartis via Facebook)

New Te­va CEO Richard Fran­cis repri­or­i­tizes to 'get back to growth'

Six weeks into his new role at the helm of Teva Pharmaceutical, Richard Francis said it’s time to “get back to growth,” starting with a good look at the company’s priorities.

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Sanofi is renewing its #VaccinesForDreams campaign with more stories, such as Juan's in Argentina (Sanofi)

Sanofi re­news so­cial cam­paign to re­mind that vac­cines let peo­ple ‘Dream Big’

Sanofi is highlighting people’s dreams — both big and small — to make the point that vaccines make them possible.

The renewed “Dream Big” global social media campaign’s newest dreamer is Juan, a teacher in the Misiones rainforest in Argentina whose story is told through videos on Instagram and Sanofi’s website with the hashtag #VaccinesForDreams.

The campaign ties to Sanofi’s broader umbrella initiative “Vaccine Stories” to promote the value of vaccines and drive awareness of the need for improved vaccination coverage.

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