No new guid­ance, but here's some ad­vice: FDA draws a line in the sand for Covid-19 vac­cine de­vel­op­ers

Less than a day af­ter re­ports emerged that the White House cit­ed in­dus­try op­po­si­tion in a move to block stricter Covid-19 vac­cine guide­lines, the FDA is spelling out ex­act­ly what reg­u­la­tors have been com­mu­ni­cat­ing to de­vel­op­ers look­ing for an emer­gency use au­tho­riza­tion.

In do­ing so, the reg­u­la­tors are of­fi­cial­ly draw­ing a line in the sand amid an in­creas­ing­ly chaot­ic cam­paign to bring a vac­cine to mar­ket at record speed.

In the brief­ing doc­u­ments for the Vac­cines and Re­lat­ed Bi­o­log­i­cal Prod­ucts Ad­vi­so­ry Com­mit­tee ahead of its Oct. 22, 2020 meet­ing, the agency tucked in two ap­pen­dices. The first was a lengthy guid­ance from June, unedit­ed, on de­vel­op­ment and li­cen­sure of a vac­cine to pre­vent Covid-19. But the sec­ond — a doc­u­ment ti­tled “Sum­ma­ry of Ad­vice Pro­vid­ed to In­di­vid­ual Spon­sors in Re­sponse to Ques­tions Re­gard­ing Emer­gency Use Au­tho­riza­tion of Vac­cines to Pre­vent COVID-19” — is new to the pub­lic.

Per the 5-page sum­ma­ry, the FDA has sug­gest­ed to spon­sors that their EUA re­quests should meet a high­er bar than what’s out­lined in the guid­ance. These ad­di­tion­al safe­ty and ef­fi­ca­cy re­quire­ments match the stricter stan­dards that the FDA was about to pub­lish be­fore the White House over­ruled them.

As­sum­ing com­pa­nies fol­low the ad­vice (and CBER chief Pe­ter Marks has em­pha­sized that they “knew what we’re ex­pect­ing”), it would be all but im­pos­si­ble for any vac­cine de­vel­op­er to sub­mit an up-to-par ap­pli­ca­tion be­fore Elec­tion Day, let alone get cleared for EUA as Pres­i­dent Don­ald Trump has promised.

For in­stance, the FDA said it’s told spon­sors to re­port da­ta with a me­di­an fol­low-up of at least two months af­ter “com­ple­tion of the full vac­ci­na­tion reg­i­men” to mon­i­tor ad­verse events; safe­ty num­bers on “well over 3,000 vac­cine re­cip­i­ents”; and “a to­tal of 5 or more” se­vere Covid-19 cas­es in the place­bo group to as­suage con­cerns about vac­cine-in­duced en­hanced res­pi­ra­to­ry dis­ease.

Then there’s the ad­vice for com­pa­nies to pro­vide a de­tailed de­scrip­tion of their man­u­fac­tur­ing process and con­trols no less than one month pri­or to seek­ing an EUA. That means for an EUA to be is­sued by Nov. 3, a com­pa­ny would have sub­mit­ted this in­for­ma­tion days ago.

Each of these sub­mis­sions, the FDA added, will be scru­ti­nized in­di­vid­u­al­ly by the same pan­el of out­side ex­perts it has con­vened in two weeks:

FDA ex­pects to con­vene an open ses­sion of FDA’s VRB­PAC pri­or to the is­suance of any EUA for a COVID-19 vac­cine, to dis­cuss whether the avail­able safe­ty and ef­fec­tive­ness da­ta sup­port au­tho­riza­tion of an EUA for the spe­cif­ic re­quest un­der re­view. This dis­cus­sion will be spe­cif­ic to the par­tic­u­lar vac­cine that is the sub­ject of the EUA re­quest and will be sep­a­rate from, and in ad­di­tion to, any gen­er­al dis­cus­sion by the VRB­PAC re­gard­ing the de­vel­op­ment, au­tho­riza­tion and/or li­cen­sure of vac­cines to pre­vent COVID-19.

Oct. 22, though, will be when the gen­er­al dis­cus­sion hap­pens. Reg­u­la­tors have asked the ad­comm to ex­plore what ad­di­tion­al stud­ies vac­cine de­vel­op­ers should con­duct, ei­ther be­fore or af­ter an OK, to eval­u­ate their can­di­dates. Should they be do­ing tri­als in chil­dren and preg­nant women? Is there a need for post-mar­ket­ing safe­ty stud­ies fol­low­ing a full BLA ap­proval? What about post-EUA safe­ty fol­low-up?

Pfiz­er/BioN­Tech, Mod­er­na, As­traZeneca/Ox­ford, J&J and a slate of oth­ers will sure­ly be tun­ing in.

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

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Ex­elix­is pulls a sur­prise win in thy­roid can­cer just days ahead of fi­nal Cabome­tyx read­out

Exelixis added a thyroid cancer indication to its super-seller Cabometyx’s label on Friday — months before the FDA was expected to make a decision, and days before the company was set to unveil the final data at #ESMO21.

At a median follow-up of 10.1 months, differentiated thyroid cancer patients treated with Cabometyx (cabozantinib) lived a median of 11 months without their disease worsening, compared to just 1.9 months for patients given a placebo, Exelixis said on Monday.

Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

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President Biden and Pfizer CEO Albert Bourla (Patrick Semansky/AP Images)

Chaot­ic ad­comm sees Pfiz­er/BioN­Tech boost­ers re­ject­ed for gen­er­al pop­u­la­tion, but rec­om­mend­ed for old­er and high-risk pop­u­la­tions

With just days before President Joe Biden’s Covid-19 booster rollout is set to go into effect, an FDA advisory committee appeared on the verge of not recommending boosters for anyone in the US before a last-minute change of wording laid the groundwork for older adults to have access to a third dose.

The FDA’s adcomm on Vaccines and Related Biological Products (VRBPAC) roundly rejected Pfizer/BioNTech booster shots for all individuals older than 16 by a 16-2 vote Friday afternoon. Soon after, however, the agency posed committee members a new question limiting booster use to the 65-and-older population and individuals at high risk of disease due to occupational exposure or comorbidities.

Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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Rafaèle Tordjman (Jeito Capital)

Con­ti­nu­ity and di­ver­si­ty: Rafaèle Tord­j­man's women-led VC firm tops out first fund at $630M

For a first-time fund, Jeito Capital talks a lot about continuity.

Rafaèle Tordjman had spotlighted that concept ever since she started building the firm in 2018, promising to go the extra mile(s) with biotech entrepreneurs while pushing them to reach patients faster.

Coincidentally, the lack of continuity was one of the sore spots listed in a report about the European healthcare sector published that same year by the European Investment Bank — whose fund is one of the LPs, alongside the American pension fund Teacher Retirement System of Texas and Singapore’s Temasek, to help Jeito close its first fund at $630 million (€534 million). As previously reported, Sanofi had chimed in €50 million, marking its first investment in a French life sciences fund.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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