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. 

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.

FDA Commissioner Rob Califf joined the heads of the CDC and NIH in the hot seat today before a key House subcommittee, explaining that there needs to be a much faster, more coordinated way to oversee vaccine safety, and that foreign biopharma inspections, halted for years due to the pandemic, are slowly ramping up again.

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.”

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.

The chief executive has kicked off a strategic review, he announced during Teva’s quarterly call, which will continue over the next several months and produce results sometime in the middle of 2023. That means some pipeline cuts may be in store, he told Endpoints News, while declining to offer much more detail.