Peter Marks (Greg Nash/Pool via AP Images)

FDA cen­ter di­rec­tors on lessons from the EUA path­way: Flex­i­bil­i­ty serves us well

The flex­i­bil­i­ty of emer­gency use au­tho­riza­tions, and al­low­ing the FDA to ac­cept more un­cer­tain­ty dur­ing the pan­dem­ic so far, has ul­ti­mate­ly aid­ed its re­sponse to Covid-19, cen­ter di­rec­tors said Tues­day at a work­shop at the Na­tion­al Acad­e­mies of Sci­ences, En­gi­neer­ing and Med­i­cine.

Pa­trizia Cavaz­zoni

Pa­trizia Cavaz­zoni, di­rec­tor of the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search, point­ed to the agili­ty of the EUAs, and the abil­i­ty to quick­ly re­voke any EUAs, as well as the trans­paren­cy around those de­ci­sions as to what led to the evo­lu­tion of the types of ther­a­peu­tics that have been au­tho­rized (see chart be­low).

“Agili­ty is very im­por­tant, and the EUA al­lows us that,” she said, stress­ing the abil­i­ty to re­voke EUAs very quick­ly and say­ing FDA wish­es it had that abil­i­ty, when the da­ta war­rant­ed, out­side of the pan­dem­ic.

“The big take­away for me, based on the past sev­er­al months, is the im­por­tance of col­lab­o­ra­tion across the US gov­ern­ment agen­cies and with in­ter­na­tion­al coun­ter­parts and the re­search com­mu­ni­ty,” Cavaz­zoni said. “The oth­er im­por­tant take­away is hav­ing a reg­u­la­to­ry frame­work that al­lows us to be ag­ile and flex­i­ble.”

It’s im­por­tant for the pub­lic to un­der­stand the ev­i­dence and da­ta gen­er­a­tion con­tin­ues even af­ter an EUA, Cavaz­zoni said, of­fer­ing the ex­am­ple of hy­drox­y­chloro­quine, which had its EUA re­voked:

What has been lost in this dis­cus­sion is that au­tho­riza­tion was in­cred­i­bly nar­row and the fact sheet specif­i­cal­ly said it should be lim­it­ed to pa­tients who could not be in clin­i­cal tri­als. Al­most im­me­di­ate­ly af­ter these tri­als read out, the EUA was re­voked. De­spite that, there was so much noise around this and it’s part of our lessons learned on com­mu­ni­cat­ing facts.

With the au­tho­rized mon­o­clon­al an­ti­bod­ies for Covid-19, Cavaz­zoni not­ed that it was clear these could save lives but “what we saw was very slow up­take last fall and in­to the spring un­til this sum­mer where fi­nal­ly the mes­sage went through and now we have lim­it­ed sup­plies. We need to work to­geth­er more ef­fec­tive­ly to break through the noise.”

Josh Sharf­stein

Josh Sharf­stein, a Johns Hop­kins pro­fes­sor who was once con­sid­ered for the top FDA com­mis­sion­er po­si­tion, al­so ques­tioned the cen­ter di­rec­tors ear­li­er on Tues­day on how in­dus­try of­ten leads the con­ver­sa­tion around the da­ta on new Covid-re­lat­ed ther­a­peu­tics and vac­cines.

Cavaz­zoni said her cen­ter is “con­strained by laws and reg­u­la­tions on what we com­mu­ni­cate” and from its per­spec­tive, “we would wel­come broad­er au­thor­i­ties when it comes to com­mu­ni­ca­tion about the pro­grams, not on­ly on is­suance of EUAs and ed­u­ca­tion, as well as EUAs that end up be­ing de­clined. I think this is an area prime for fur­ther think­ing.”

Pe­ter Marks, di­rec­tor of the FDA’s Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search, added that he’s hes­i­tant to call for any ma­jor changes to the EUA path­way. “It’s not that usu­al that I think of a par­tic­u­lar statu­to­ry au­thor­i­ty as in­cred­i­bly well craft­ed but our emer­gency use au­tho­riza­tion is craft­ed quite well to deal with a range of things that can come our way and it’s up to us to en­sure it’s used prop­er­ly, not to mess around with the statute.”

“If we’re think­ing about any­thing in this area, it’s mi­nor re­dec­o­ra­tion, not ren­o­va­tion,” he said. Ear­li­er in the pan­el dis­cus­sion Marks men­tioned the idea of po­ten­tial­ly com­bin­ing the ef­forts of the FDA’s vac­cine ad­comm VRB­PAC and the CDC’s sep­a­rate ad­comm ACIP. “The process ul­ti­mate­ly works in that sci­ence cor­rect­ed some of the er­rors that were made. Yes, it might have been nice if they weren’t made in re­al time but I think the trans­paren­cy helps.”

“The thing I won­der in the vac­cine realm if we could have bet­ter co­or­di­nat­ed things to es­sen­tial­ly col­lapse the VRB­PAC process with the Ad­vi­so­ry Com­mit­tee on Im­mu­niza­tion Prac­tices process,” he said. “We need to look care­ful­ly at this, ACIP was de­vel­oped for rou­tine vac­cines and for that mat­ter, for coun­ter­mea­sures not for the mid­dle of an emer­gency.”

And in a coun­try where there’s al­ways been vac­cine hes­i­tan­cy, “that’s not the best time to have any air space be­tween two ad­vi­so­ry com­mit­tees,” Marks said. “I think we as a fed­er­al gov­ern­ment need to do a re­al­ly good job to make sure our mes­sag­ing is har­mo­nized.”

Jeff Shuren

The com­ments come as the FDA’s and CDC’s ad­comms and lead­ers re­cent­ly clashed over what groups of peo­ple should first re­ceive boost­er shots fol­low­ing the re­cent EUA. Two of the top vac­cine ex­perts work­ing un­der Marks re­cent­ly an­nounced their re­tire­ments were due at least in part to con­cerns with the way the boost­er EUA process played out.

On the med­ical de­vice side, CDRH head Jeff Shuren not­ed that the EUA au­thor­i­ties have been in­cred­i­bly help­ful giv­en the vol­ume of prod­ucts com­ing in through the door. “With­out a na­tion­al test­ing strat­e­gy, we’re do­ing all com­ers, rather than hav­ing a tar­get­ed ap­proach,” he said. He al­so not­ed the EUA path­way was in­valu­able in speed­ing new tests and di­ag­nos­tics, adding, “I re­al­ly wish we had that flex­i­bil­i­ty in peace time.”

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; 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.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

EMA signs off on 3 drugs re­cent­ly re­ject­ed by FDA, in­clud­ing Bio­Mar­in's new he­mo­phil­ia gene ther­a­py

The EMA’s human medicines committee on Friday recommended three new drugs for approval or conditional approval, even as their US counterparts have rejected these three for various reasons.

In a major move, CHMP offered a thumbs-up to a conditional marketing authorization for the first gene therapy to treat severe hemophilia A, although the agency cautioned that it’s so far unknown how long the effects of infusion will last.

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Sanofi, GSK tout 72% Omi­cron ef­fi­ca­cy in PhI­II tri­al of next-gen, bi­va­lent shot — with an eye to year-end roll­out

Sometimes, being late can give you an advantage.

That’s what Sanofi and GSK are trying to say as the Big Pharma partners report positive results from a late-stage trial of their next-gen bivalent Covid-19 vaccine, which was designed to protect against both the original strain of the SARS-CoV-2 virus and the Beta variant. Specifically, against Omicron, they note, the vaccine delivered 72% efficacy in all adults and 93.2% in those previously infected.

Alarmed by side ef­fect, FDA slaps clin­i­cal hold on Sarep­ta's next-gen Duchenne drug

Sarepta Therapeutics’ next-gen Duchenne muscular dystrophy drug has been hit with a clinical hold after investigators flagged a serious case of low magnesium levels in one patient’s blood.

Screening and dosing will be halted in what is known as Part B of the Phase II MOMENTUM study, which has enrolled about half of the planned patients. Sarepta said it will be submitting information on all cases of the condition, known as hypomagnesemia, per the FDA’s request and proposing some changes to the risk mitigation and safety monitoring plan.

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