CBER Director Peter Marks (Greg Nash/Pool via AP Images)

Op­er­a­tion Warp Speed for rare dis­eases: CBER leader says pi­lot is com­ing soon

The next gen­er­a­tion of Op­er­a­tion Warp Speed is com­ing soon, and this time it’s go­ing to take aim at rare dis­eases, Pe­ter Marks, the di­rec­tor of the FDA’s Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search, told at­ten­dees of the Bio­phar­ma Con­gress in Wash­ing­ton, DC on Mon­day.

“We’re get­ting ready to pi­lot that pro­gram,” Marks said, mak­ing clear the goal is to help ar­eas with high un­met needs.

I think the goal is to take a drug with some promise in the rare dis­ease space for dis­eases that don’t have al­ter­na­tives … and then take these prod­ucts, maybe they have break­through or ad­vanced ther­a­py des­ig­na­tion, they have promise and there is a prod­uct in de­vel­op­ment and not just a con­cept. And then give them the op­por­tu­ni­ty with not just chem­istry, man­u­fac­tur­ing and con­trols, which we have a pi­lot for, but al­low the clin­i­cal de­vel­op­ment to hap­pen in con­stant com­mu­ni­ca­tion and shar­ing of po­ten­tial re­sults be­fore a sub­mis­sion of an NDA or BLA. That’s the idea, is to move things as fast as pos­si­ble.

He said “it’s not un­like­ly in some of these rare dis­ease sit­u­a­tions that there will be hic­cups” and if those FDA-spon­sor con­ver­sa­tions can oc­cur in re­al time, they can re­solve those is­sues more quick­ly.

Celia Wit­ten

Marks stressed that he trusts his deputy Celia Wit­ten to find new tal­ent for the FDA’s re­tir­ing lead­er­ship as the agency tran­si­tions from its Of­fice of Tis­sues and Ad­vanced Ther­a­pies to the su­per-of­fice to be known as the Of­fice of Ther­a­peu­tic Prod­ucts, or OTP.

He al­so ex­plained how he’s look­ing in­to us­ing a Pro­ject Or­bis-like ap­proach to ex­pand­ing the ap­proval of gene and cell ther­a­pies through­out oth­er high-in­come coun­tries (e.g. UK, Japan, EU) as the FDA’s On­col­o­gy Cen­ter of Ex­cel­lence cur­rent­ly does for some new can­cer drugs and in­di­ca­tions.

“And we want to take it one step fur­ther for these rare dis­eases. For vac­cines, there’s WHO-pre­qual­i­fi­ca­tion, which is ba­si­cal­ly, WHO says this vac­cine has been li­censed in a ref­er­ence coun­try, it’s good enough for us, it should be good enough for you, and the idea is to quick­ly do that for gene ther­a­py so low­er-in­come coun­tries could po­ten­tial­ly make use of these ap­provals. Why is that im­por­tant? For some gene ther­a­pies, the on­ly way peo­ple will get treat­ment is if they get gene ther­a­py be­cause those coun­tries can­not af­ford sup­port­ive care, for in­stance,” Marks said, not­ing that “there may be ways to deal” with some of the mas­sive up­front costs.

When asked why there has been an in­crease in clin­i­cal holds from CBER, Marks not­ed mul­ti­ple caus­es in­clud­ing an in­crease in clin­i­cal tri­als, less ex­pe­ri­enced spon­sors in this cell and gene ther­a­py space, and

we see a va­ri­ety of dif­fer­ent type of INDs, some of which are miss­ing key pieces, and some­times those can be fixed with in­ter­ac­tive dis­cus­sions dur­ing the 30 days. But 30 days is not a lot of time if some­one doesn’t have a man­u­fac­tur­ing sec­tion to their IND [laugh­ter in the au­di­ence] — don’t laugh, don’t laugh, it hap­pens enough, we have a rule of threes that I wouldn’t tell you about it if it didn’t hap­pen at least three times … There are some cas­es where we have bona fide spon­sors who have is­sues that prob­a­bly could be re­solved with in­ter­ac­tive re­view where we just don’t have the band­width.

OTP is look­ing to bring on about 125 new staffers to help with the cell and gene ther­a­py re­views thanks to the lat­est it­er­a­tion of the user fee deals, but the mar­ket for tal­ent is fierce­ly com­pet­i­tive.

FDA’s out­go­ing OTAT di­rec­tor Wil­son Bryan re­cent­ly an­nounced his in­tent to re­tire but told End­points News on Mon­day that he’s look­ing for­ward to the next chal­lenge, and want­ed to hand off the reins of the of­fice to the next set of in­com­ing lead­er­ship as the tran­si­tion hap­pens.

Look­ing ahead, Bryan ex­pressed can­did fears that the agency may not end up pulling cell or gene ther­a­pies quick­ly when ap­proved un­der the ac­cel­er­at­ed path­way, and even when there might be not enough ev­i­dence of ef­fi­ca­cy from the con­fir­ma­to­ry tri­al.

He said he does think the ac­cel­er­at­ed path­way, which has on­ly been used once so far for a cell/gene ther­a­py, will be fre­quent­ly used and that the con­fir­ma­to­ry stud­ies could take years to con­firm ben­e­fit, es­pe­cial­ly if it’s a slow­ly de­vel­op­ing dis­ease.

“We’re go­ing to get in­to trou­ble with ac­cel­er­at­ed ap­proval for gene ther­a­pies if it takes years to tell us if some­thing works,” Bryan said. “Be­cause then that means it takes years to tell us if it doesn’t work.”

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

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Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to clinicaltrials.gov, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

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Bet­ter Ther­a­peu­tics cuts 35% of staff while await­ing dig­i­tal ther­a­peu­tic ap­proval

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.