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.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; 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.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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Richard Pazdur, FDA's OCE director (Flatiron Health via YouTube)

FDA's OCE makes the case for ac­cel­er­at­ed ap­proval rid­er in user fee reau­tho­riza­tion

Four experts from the FDA’s Oncology Center of Excellence took to the New England Journal of Medicine yesterday to make the case for not only improving the agency’s ability to expeditiously pull dangling accelerated approvals when, on the rare occasion, confirmatory trials fail, but also better building “quality and efficiency into the AA on-ramp.”

The timely perspective arrives as Congress has exactly one week left to draft, release and sign off on the reauthorized user fee deals before layoff notices will be sent to drug reviewers. That package, which is likely to hitch a ride with the continuing resolution, may or may not include several policy riders (opposed by Republicans), including one that would allow the FDA to require confirmatory trials to be underway before an AA is granted, and would improve the process by which FDA can withdraw AAs.

Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.

FDA's out­side ex­perts vote in fa­vor of Fer­ring's fe­cal trans­plant for C. dif­fi­cile, set­ting the stage for Seres

FDA’s outside advisors voted in favor of Ferring Pharmaceuticals’ RBX2660, an experimental poop-based drug implant that the company says would be the first microbiota-based live biotherapeutic to receive an FDA green light.

That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

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No­var­tis takes Gilenya fight to Supreme Court as gener­ic com­pe­ti­tion looms

Novartis stands to lose more than a quarter billion dollars in sales this year if Gilenya generics are allowed to market. So it’s taking the fight all the way to the Supreme Court.

The Swiss pharma giant announced on Wednesday that a US federal appeals court has refused to rehear its Gilenya patent case against private Chinese biotech HEC Pharm. Next up? Novartis plans on petitioning the Supreme Court to uphold the validity of its patent No. 9,187,405, referred to in court documents as the ‘405 patent, covering a 0.5 mg dosing regimen of the multiple sclerosis drug.

An­oth­er Cipla site lands a Form 483 over clean­ing is­sues and QC con­trols

A Cipla drug manufacturing site in India has once again landed in the crosshairs of FDA inspectors.

The facility in question is Cipla’s drug manufacturing facility in the village of Verna, in the state of Goa in India’s southwest. In a sign that foreign inspections might ramp up again, the FDA’s visit from Aug. 16 to Aug. 22 uncovered six observations.

The 11-page report noted that environmental monitoring at the site did not properly ensure that microbial contaminants were not making any impact in the aseptic filling areas. It also found that procedures meant to stop microbial contamination were not adequately conducted in aseptic areas of the facility.

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