HHS Secretary Xavier Becerra (Jacquelyn Martin/AP Images)

UP­DAT­ED: FDA calls for Hatch-Wax­man tweak, ac­cel­er­at­ed ap­proval re­forms in Pres­i­dent Biden's 2023 bud­get pro­pos­al

As the FDA has re­ceived its fair share of vil­i­fi­ca­tion and staffers work­ing ex­ceed­ing­ly long hours since the pan­dem­ic be­gan, Pres­i­dent Biden’s 2023 bud­get re­quest is look­ing to pro­vide the agency with a ma­jor in­fu­sion of new funds — $2.1 bil­lion, or 34% above FDA’s bud­get this year.

That vast ma­jor­i­ty of those new funds will be di­rect­ed at prep­ping for the next pan­dem­ic, or the next it­er­a­tion of the cur­rent one. With­in an HHS-wide to­tal of $81.7 bil­lion in 2023 pan­dem­ic funds, $1.6 bil­lion is in­clud­ed for FDA to ex­pand and mod­ern­ize pan­dem­ic-re­lat­ed reg­u­la­to­ry ca­pac­i­ty, IT and lab in­fra­struc­ture.

On FDA’s end, the agency said it al­so sub­mit­ted to Con­gress a com­ple­men­tary pack­age of leg­isla­tive pro­pos­als “de­signed to bol­ster the FDA’s au­thor­i­ties” — no­table pro­pos­als men­tioned in­clude:

  • Ac­cel­er­at­ed ap­proval re­forms to help en­sure that the re­quired con­fir­ma­to­ry stud­ies will fin­ish in a time­ly man­ner and “reap high-qual­i­ty, in­ter­pretable re­sults. This will help min­i­mize the time that a prod­uct is mar­ket­ed be­fore its clin­i­cal ben­e­fit can be con­firmed.”

Specif­i­cal­ly, the FDA said on these leg­isla­tive pro­pos­als that it’s look­ing on Con­gress to re­vise the FD&C Act to al­low the agency to re­quire, as a con­di­tion of an ac­cel­er­at­ed ap­proval ap­pli­ca­tion’s ac­cep­tance for fil­ing or ap­proval that a spon­sor must first demon­strate that a pro­posed con­fir­ma­to­ry study is ad­e­quate­ly de­signed to ver­i­fy and de­scribe clin­i­cal ben­e­fit and can be com­plet­ed in a time­ly man­ner.

The FDA’s al­so look­ing to re­vise the law so that it can fol­low its typ­i­cal dis­pute res­o­lu­tion pro­ce­dures for drug ap­pli­ca­tions when with­draw­ing an ac­cel­er­at­ed ap­proval. The agency’s on­col­o­gy ad­comm met last sum­mer to dis­cuss how to pro­ceed on sev­er­al ac­cel­er­at­ed ap­provals that ei­ther failed to fin­ish or failed to prove clin­i­cal ben­e­fit in their con­fir­ma­to­ry tri­als.

FDA said it is al­so propos­ing a tech­ni­cal fix to re­vise the with­draw­al stan­dard so that it mir­rors the anal­o­gous with­draw­al stan­dard set forth in sec­tion 505(e) for drugs with tra­di­tion­al ap­provals.

Mean­while, as part of a push to in­crease the avail­abil­i­ty of gener­ic drugs up­on ap­proval, the agency said it’s seek­ing to amend the Hatch-Wax­man 180-day patent chal­lenge ex­clu­siv­i­ty pro­vi­sions,

“so that the FDA can ap­prove sub­se­quent gener­ic drug ap­pli­ca­tions un­less and un­til a first ap­pli­cant be­gins com­mer­cial mar­ket­ing of a drug and trig­gers the ex­clu­siv­i­ty pe­ri­od, at which point ap­proval of sub­se­quent ap­pli­ca­tions would be blocked by 180 days. This would en­sure that the ex­clu­siv­i­ty on­ly lasts 180 days, as in­tend­ed, rather than mul­ti­ple years. This would sub­stan­tial­ly in­crease the like­li­hood that gener­ic ver­sions of patent-pro­tect­ed drugs come in­to the mar­ket in a time­ly fash­ion and would al­low mul­ti­ple ver­sions of gener­ic prod­ucts to be ap­proved quick­ly lead­ing to sig­nif­i­cant cost sav­ings.”

Back in No­vem­ber, the FDA pub­lished re­search show­ing how of the gener­ic drugs that were el­i­gi­ble for ex­clu­sive mar­ket­ing for 180 days at ap­proval, on­ly 50% had mar­ket­ed 6 months af­ter ap­proval.

Build­ing on the suc­cess of Rick Paz­dur’s FDA’s On­col­o­gy Cen­ter of Ex­cel­lence, the bud­get seeks to pro­vide the cen­ter with an ad­di­tion­al $20 mil­lion in new, one-time funds to sup­port pro­grams that in­cor­po­rate pa­tient voice, re­al-world ev­i­dence, and col­lab­o­ra­tions with FDA’s glob­al part­ners (see Pro­ject Or­bis) to fa­cil­i­tate faster pa­tient ac­cess to new can­cer ther­a­pies.

“With this in­vest­ment, FDA will build on ex­ist­ing ef­forts to con­tin­ue to fa­cil­i­tate and ex­pand in­ter­nal and ex­ter­nal col­lab­o­ra­tions to ex­pe­dite the de­vel­op­ment of on­col­o­gy and ma­lig­nant hema­tol­ogy prod­ucts as well as in­crease di­ver­si­ty and speed progress against the most dead­ly and rare can­cers, in­clud­ing child­hood can­cers, and fos­ter the de­vel­op­ment of nov­el ther­a­peu­tics for pa­tients with ul­tra-rare can­cers,” the HHS bud­get re­quest re­leased on Mon­day says.

An ad­di­tion­al $30 mil­lion next year will al­so go to help­ing the opi­oid cri­sis, with funds to sup­port the de­vel­op­ment of opi­oid over­dose re­ver­sal treat­ments and treat­ments for opi­oid use dis­or­der, as well as to stop ship­ments of coun­ter­feit drugs, and new fund­ing to de­vel­op, eval­u­ate, and ad­vance dig­i­tal health med­ical de­vices to ad­dress opi­oids use dis­or­der.

An­oth­er $6 mil­lion in­crease is re­quest­ed to help build the foun­da­tion for im­ple­ment­ing the 21st Cen­tu­ry Roadmap for mod­ern­iz­ing FDA’s safe­ty sur­veil­lance and over­sight pro­gram for mar­ket­ed drug prod­ucts, an in­crease of $5 mil­lion above FY 2022 en­act­ed to con­tin­ue the im­ple­men­ta­tion of the Pre­dic­tive Tox­i­col­o­gy Roadmap, which will en­hance FDA’s abil­i­ty to quick­ly and more ac­cu­rate­ly pre­dict po­ten­tial tox­i­c­i­ties–and re­duce as­so­ci­at­ed risks to the pub­lic.

The bud­get al­so in­cludes an in­crease of $54 mil­lion for ca­pac­i­ty-build­ing ef­forts at the agency, in­clud­ing sup­port for es­sen­tial ser­vices, such as re­spond­ing to in­quiries and sub­ject mat­ter ex­per­tise on FOIA re­quests, le­gal ser­vices, and sup­port ef­forts that re­duce risk from lab­o­ra­to­ry work, en­hance lab­o­ra­to­ry se­cu­ri­ty and da­ta qual­i­ty, and in­crease ef­fi­cien­cies across the safe­ty and health pro­gram.

Biden’s bud­get re­quest al­so fea­tures an in­crease of $34 mil­lion over last year to par­tial­ly fund an­tic­i­pat­ed in­creas­es in pay costs for FDA’s over 19,000 em­ploy­ees.

The Al­liance for a Stronger FDA en­dorsed the Pres­i­dent’s re­quest­ed in­crease in FDA’s bud­get au­thor­i­ty ap­pro­pri­a­tions (ex­clud­ing user fees) of ap­prox­i­mate­ly $356 mil­lion over the FY 2022 ap­pro­pri­at­ed amounts, or a to­tal of ap­prox­i­mate­ly $3.67 bil­lion.

Ed­i­tor’s note: Ar­ti­cle up­dat­ed to in­clude de­tails from FDA’s re­quest.

Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Scoop: Roche scraps one of two schiz­o­phre­nia PhII tri­als af­ter fail­ing the pri­ma­ry end­point

Roche has terminated one of two Phase II trials testing its drug ralmitaront in patients with schizophrenia, the Big Pharma confirmed to Endpoints News.

The study was terminated last month, according to a June 22 update to the registry on clinicaltrials.gov. Begun in September 2020, the trial was looking at ralmitaront in patients with acute schizophrenia. The trial enrolled 286 patients out of an originally planned 308.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Years af­ter link­ing arms with Bris­tol My­ers and both Mer­cks, Sutro finds its lat­est part­ner in Tokyo

Astellas and Sutro Biopharma are linking arms on a new field of antibody-drug conjugates that they hope will improve upon existing cancer immunotherapies.

The Tokyo pharma will dole out $90 million in cash for the collaboration, the companies said Monday afternoon. That upfront payment will extend the South San Francisco biotech’s runway from late 2023 into the first half of 2024, Cowen analysts noted.

Pearl Huang, Dunad Therapeutics CEO (Ken Richardson, PR Newswire)

Long­time biotech leader Pearl Huang takes the reins as CEO of No­var­tis-backed up­start

It has only been a few months since Pearl Huang exited the top seat at Cygnal Therapeutics, but now she’s back at the helm of another biotech.

After taking a few months off — passing an exam in that time to get her captain’s license from the US Coast Guard — she’s been named CEO of Dunad Therapeutics, a biotech focused on developing a small molecule covalent therapies that was founded in 2020. Huang told Endpoints News that two factors attracted her to going back to the c-suite: the company’s technology and its co-founders.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

Matt Gline, Roivant CEO (John Sciulli/Getty Images for GLG)

Roivant chops sick­le cell gene ther­a­py, der­ma­tol­ogy drugs to fo­cus on 'high­er val­ue pro­ject­s'

Roivant is sweeping a suite of drugs, including a gene therapy for sickle cell disease already in the clinic, out of its pipeline.

Six programs from four of its “vants” are being wound down as part of “a company-wide cost optimization and pipeline reprioritization initiative to reduce our expected operating expenses and prioritize our capital resources.”

When reached by Endpoints News, a spokesperson said, “We don’t anticipate a material reduction in headcount but we will likely reassign some folks to higher value projects as part of winding down specific programs.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

Laurence Reid, Decibel CEO

Still in pre­clin­i­cal test­ing for ear gene ther­a­pies, Deci­bel touts small snap­shot of chemo-in­duced hear­ing loss drug

Though Decibel Therapeutics has largely pivoted toward gene therapies for the inner ear, its lead clinical candidate simply aims to protect cancer patients from chemotherapy-induced hearing loss. On Tuesday, the biotech presented its first efficacy data for the program, and execs like what they see.

Decibel reported interim results from a Phase Ib study showing the experimental drug, dubbed DB-020, largely protected a small group of patients from losing their hearing. Researchers used a particularly unique study design, administering the compound in one of each patients’ ears before they received cisplatin chemotherapy and placebo in the other.