Stephen Hahn, FDA commissioner (Alex Edelman/Pool via AP Images)

Hahn in­sists FDA will stick to guide­lines for Covid-19 vac­cine de­ci­sions

Speak­ing at the Fi­nan­cial Times’ US Phar­ma and Biotech Sum­mit on Tues­day, FDA com­mis­sion­er Stephen Hahn said the agency will stick to the stan­dards and cri­te­ria it has laid out to ap­prove or au­tho­rize ap­pli­ca­tions for vac­cines to pre­vent coro­n­avirus dis­ease.

In re­cent weeks, Hahn has made nu­mer­ous state­ments as­sert­ing that the agency will not ap­prove or au­tho­rize a vac­cine that does not meet the agency’s “rig­or­ous ex­pec­ta­tions,” de­spite re­peat­ed claims by Pres­i­dent Don­ald Trump that a vac­cine could be green­light­ed be­fore the No­vem­ber elec­tion.

Hahn’s state­ments al­so fol­low news that FDA has de­vel­oped guid­ance on its ex­pec­ta­tions for emer­gency use au­tho­riza­tions (EUAs) for Covid-19 vac­cines that are re­port­ed­ly un­der re­view by the White House.

“The cri­te­ria that we’ve es­tab­lished for a vac­cine, are the cri­te­ria we are go­ing to use. We’ve been very clear about that with spon­sors, very clear about that with the pub­lic,” Hahn said. While Hahn specif­i­cal­ly ad­dressed FDA’s June 30 guid­ance on vac­cine de­vel­op­ment and li­cen­sure, he did not make men­tion of the EUA-spe­cif­ic guid­ance.

Nonethe­less, Hahn said the June 30 guid­ance lays out the cri­te­ria FDA will be look­ing at to make a de­ci­sion about safe­ty and ef­fi­ca­cy and that the agency con­tin­ues to have con­ver­sa­tions with spon­sors about the da­ta it wants to see. Hahn added that it is up to vac­cine mak­ers to de­ter­mine the tim­ing of an ap­pli­ca­tion, but that when one is sub­mit­ted, “We will ap­ply the stan­dards that we’ve stat­ed to look at those da­ta and we’ll make a de­ci­sion.”

Hahn al­so said he could not pre­dict when a vac­cine would be avail­able. “I don’t have a crys­tal ball here,” he said and af­firmed that, “We won’t cut cor­ners, we will use our rig­or­ous cri­te­ria.”

In re­marks made Mon­day at the Vac­cine World Con­gress, Philip Krause, deputy di­rec­tor of the Of­fice of Vac­cines Re­search and Re­view with­in the CBER, ad­dressed re­ports about the agency’s EUA guid­ance.

Julie Ger­berd­ing

“Of course, as you’ve read in the pa­per, there is FDA guid­ance which ex­ists but hasn’t yet been re­leased,” Krause said dur­ing a pan­el dis­cus­sion with Paul Stof­fels, CSO at John­son & John­son, and Julie Ger­berd­ing, EVP and chief pa­tient of­fi­cer at Mer­ck.

Krause asked whether the two phar­ma­ceu­ti­cal ex­ec­u­tives feel it is im­por­tant for the EUA guid­ance to be re­leased to the pub­lic, ref­er­enc­ing a pledge by the CEOs of nine vac­cine mak­ers to not seek ap­proval or au­tho­riza­tion for a vac­cine be­fore demon­strat­ing safe­ty and ef­fi­ca­cy through Phase III tri­als.

Paul Stof­fels

“In the con­text of your CEOs’ pledge to fol­low FDA guid­ance, how im­por­tant do you think it is that this guid­ance be made pub­licly avail­able so that the pub­lic can judge whether or not your CEOs are do­ing what they said you would do,” he asked.

“I think it is im­por­tant that the guid­ance against which we will be judged will be avail­able,” said Stof­fels. Ger­berd­ing con­curred, say­ing that, “Trans­paren­cy re­al­ly mat­ters right now, even if there is no shock­ing per­spec­tive that is part of this new guid­ance, just get­ting it out there so that peo­ple can see it, I think we re­al­ly have to err on the side of trans­paren­cy.”

For a look at all End­points News coro­n­avirus sto­ries, check out our spe­cial news chan­nel.

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Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; 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.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

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#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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In­dus­try groups call to block WTO IP waiv­er ex­pan­sion to Covid-19 ther­a­peu­tics

The WTO’s TRIPS Council in mid-October is expected to debate whether to extend the IP waiver for Covid-19 vaccines to therapeutics and diagnostics too.

While the Biden administration backed the original vaccine waiver, which critics note has not done much to expand access to vaccines as demand has dried up, US trade officials haven’t offered any perspective yet on whether to expand the waiver to Covid treatments.

Jerome Durso, Intercept Pharmaceuticals CEO

In­ter­cep­t's OCA fails a PhI­II NASH tri­al, rais­ing fresh doubts about its years­long quest for an OK

Intercept Pharmaceuticals has run into another big setback in its yearslong quest to win an approval for OCA in NASH. The biotech put out word Friday morning that its Phase III REVERSE study failed the primary endpoint for the liver disease, sending its share price into a tailspin.

There was no significant improvement in fibrosis among the patients suffering from cirrhosis who were treated with obeticholic acid, with investigators hunting for a minimum 1-stage histological improvement in the disease after 18 months of therapy.

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