FDA commissioner Rob Califf (Tom Williams/CQ Roll Call via AP Images)

With drug pric­ing al­most done, Con­gress looks to wrap up FDA user fee leg­is­la­tion

The Sen­ate won’t re­turn from its sum­mer re­cess un­til Sept. 6, but when it does, it of­fi­cial­ly has 18 busi­ness days to fi­nal­ize the reau­tho­riza­tion of the FDA user fee pro­grams for the next 5 years, or else thou­sands of drug and bi­o­log­ics re­view­ers will be laid off and PDU­FA dates will van­ish in the in­ter­im.

FDA com­mis­sion­er Rob Califf re­cent­ly sent agency staff a memo ex­plain­ing how, “Our lat­est es­ti­mates are that we have car­ry­over for PDU­FA [Pre­scrip­tion Drug User Fee Act], the user fee fund­ing pro­gram that will run out of fund­ing first, to cov­er on­ly about 5 weeks in­to the next fis­cal year.”

That means Con­gress has a tiny cush­ion of about a month, un­til Nov. 4, to get its act to­geth­er.

Right now, there are vast dif­fer­ences be­tween the House-passed ver­sion of the bill from ear­ly June, and the Sen­ate’s pre­vi­ous­ly in­tro­duced ver­sion, which it has yet to vote on. While there are dif­fer­ences be­tween the two ver­sions, on ma­jor is­sues like the reg­u­la­tion of IVDs and cos­met­ics, both the House and Sen­ate agree that ac­cel­er­at­ed ap­proval path­way re­forms should be added to the user fee deals.

But in­dus­try and oth­ers are mak­ing the case for not go­ing too far with those re­forms, es­pe­cial­ly as ac­cel­er­at­ed ap­provals have helped many can­cer pa­tients ac­cess treat­ments ear­li­er than they oth­er­wise would have.

Richard Burr

Am­gen re­searchers re­cent­ly pub­lished a pa­per ex­plain­ing, “Over the 30-year his­to­ry of the ac­cel­er­at­ed ap­proval pro­gram, there have been rel­a­tive­ly few delin­quent cas­es or dan­gling ap­provals. Leg­isla­tive or oth­er changes to the pro­gram should be based on cu­mu­la­tive ex­pe­ri­ence, not out­liers.”

North Car­oli­na Re­pub­li­can and Sen­ate health com­mit­tee rank­ing mem­ber Richard Burr last month in­tro­duced a blank ver­sion of the user fee leg­is­la­tion as an at­tempt to start over with­out any of the pol­i­cy rid­ers that made it out of the com­mit­tee, like the ac­cel­er­at­ed ap­proval path­way changes that would al­low the FDA to re­quire com­pa­nies to be­gin post­mar­ket­ing stud­ies be­fore grant­i­ng the sped-up ap­provals.

But it re­mains un­clear how Con­gress will move.

Califf added that if the FDA has “rea­son­able as­sur­ance from Con­gress that they will act in time to al­low us to con­tin­ue col­lect­ing fees af­ter Sep­tem­ber 30th, then we will not need to be­gin the no­ti­fi­ca­tion process for PDU­FA-fund­ed staff.”

But in the mean­time, the FDA will have to pre­pare and FDAers are al­ready ex­pend­ing time and re­sources for that dooms­day sce­nario, even if it re­mains un­like­ly.

Tom Riga, Spectrum Pharmaceuticals CEO

Spec­trum im­plodes af­ter a harsh pub­lic slap­down and now a CRL from Richard Paz­dur

The FDA has gone out of its way several times to flatten any expectations for Spectrum’s lung cancer drug poziotinib, including slamming the regulatory door in the biotech’s face four years ago when the their executive crew came calling for a breakthrough drug designation and encouragement from the oncology wing of the FDA.

That stinging early rebuke pointed straight down the path to a corrosive in-house agency review of Spectrum’s attempt to land an accelerated approval for the oral EGFR TKI and a public whipping that included a classic takedown by none other than Richard Pazdur, who slammed the company for “poor drug development” that led to confusion over the dose needed for a slice of NSCLC patients harboring HER2 exon 20 insertion mutations.

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Vac­cine doc­u­ments, young lead­ers and mar­ket tur­moil: End­points' 10 biggest sto­ries of 2022

It’s been a volatile year in the world of biopharma. Market declines reset M&A valuations, and may be beginning to tempt bigger buyers back into dealmaking. Russia’s war in Ukraine disrupted drug sales and clinical trials. A new generation of young biotech leaders emerged in the Endpoints 20(+1) Under 40. And as capital runs dry in a tough environment for raising new funds, companies big and small are taking a look at their headcounts and operations for ways to make it through lean times.

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Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; 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.

As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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Rachael Rollins (Charles Krupa/AP Images)

US seeks jail time for co-CEO of New Eng­land com­pound­ing cen­ter af­ter dead­ly 2012 fun­gal out­break

The US attorney for the district of Massachusetts late last week called on the state’s district court to sentence the former co-owner of the now-defunct New England Compounding Center to 18 months of jail time for his role in the center’s quality deviations that led to more than 100 people dead from a fungal meningitis outbreak.

Gregory Conigliaro was convicted of conspiring with more than a dozen others at NECC to deceive the FDA and misrepresent the fact that the center was only dispensing drugs pursuant to patient-specific prescriptions.

In­dus­try groups, CVS pick apart FDA's pro­posed path­way for gener­ics to carve out OTC in­di­ca­tions

Pharma industry groups like the Association for Accessible Medicines (AAM) and PhRMA are raising pointed questions about an FDA plan to create a new pathway for marketing prescription drugs with an additional condition for nonprescription use (ACNU), which would require more safeguards than the current OTC pathway but essentially carve out new OTC uses for some generic drugs.

Chief among the concerns were: Insurance companies dropping coverage for the Rx version, new ACNU patents to block competition, and industry essentially governing the pathway.

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Robert Califf, FDA commissioner (Jose Luis Magana/AP Images)

Fourth ac­cel­er­at­ed ap­proval in Duchenne? Sarep­ta gets pri­or­i­ty re­view for gene ther­a­py amid FDA scruti­ny

Sarepta is once again on the accelerated approval path for a Duchenne drug, picking up a priority review Monday morning.

The FDA granted the accelerated review to SRP-9001, Sarepta announced Monday, which would become the biotech’s fourth Duchenne drug if approved. Much like SRP-9001 will do, each of the previous three therapies went through the accelerated approval pathway. But unlike the others, SRP-9001 is a gene therapy.

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Sanofi's new headquarters, La Maison Sanofi, in Paris (Credit: Luc Boegly)

Sanofi wel­comes 500 staffers to new Paris HQ af­ter €30M ren­o­va­tion

When Paul Hudson took the helm at Sanofi back in 2019, he promised to reinvent the pharma giant — including its Paris headquarters. This week, the company set up shop in new “state-of-the-art” digs.

La Maison Sanofi, as the new HQ is called, is officially open for business, Hudson announced on Monday. The 9,000-square-meter (just under 97,000-square-foot) space accommodates 500 employees across the company’s government and global support functions teams, including finance, HR, legal and corporate affairs — and it was built with environmental sustainability and hybrid work in mind.

Albert Bourla, Pfizer CEO (John Thys/POOL/AFP via Getty Images)

Pfiz­er CEO un­der fire from UK watch­dog over vac­cine com­ments — re­port

Pfizer CEO Albert Bourla told the BBC last December that he had “no doubt in my mind that the benefits, completely, are in favor” of vaccinating 5- to 11-year-olds for Covid-19. Almost a year later, those comments have reportedly landed him in trouble with a UK pharma watchdog.

Children’s advocacy group UsForThem filed a complaint with the UK’s Prescription Medicines Code of Practice Authority (PMCPA) last year accusing Bourla of making “disgracefully misleading” statements during the BBC interview, including one that “Covid in schools is thriving.” At the time, UK regulators had not yet cleared the vaccine for the 5 to 11 age group, though the vaccine did have a positive opinion from the EMA’s human medicines committee.

FDA tells Catal­ent to fix is­sues at two man­u­fac­tur­ing sites on its own

The CDMO Catalent will have to fix issues at two manufacturing plants in the US and Europe that were subject to inspections by the FDA this summer, giving the company room to correct the issues without facing further regulatory action.

The FDA gave Catalent a “voluntary action indicated” response to two inspections at the contract manufacturer’s site in Bloomington, IN, and Brussels, Belgium. Fixing the issues on its own is a preferable outcome to facing an “official action indicated” response, meaning that an official warning would be sent out or a sit-down with the FDA would be required.

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