FDA and Bio­gen dis­cussed Aduhelm's ac­cel­er­at­ed ap­proval as ear­ly as 2019 — re­port

Top FDA neu­ro­science of­fi­cial Bil­ly Dunn has been a vo­cal ad­vo­cate for Bio­gen and its Alzheimer’s drug Aduhelm through­out the en­tire re­view process all the way up to the drug’s di­vi­sive ac­cel­er­at­ed ap­proval. That en­thu­si­asm — which had drawn the ire of ad­vo­ca­cy groups and the ad­comm of out­side ex­perts — seems to stem from a se­ries of meet­ings be­tween the com­pa­ny and Dunn dat­ing back as far as 2019, STAT re­ports, when the Cam­bridge, MA drug­mak­er launched a se­cret cam­paign dubbed Pro­ject Onyx to win ap­proval for ad­u­canum­ab.

Aaron Kessel­heim

Those meet­ings led to dis­cus­sions with of­fi­cials in Dunn’s of­fice about the use of the ac­cel­er­at­ed ap­proval path­way to move Aduhelm through the line. The use of that path­way is what Har­vard med­ical school pro­fes­sor and ad­comm mem­ber Aaron Kessel­heim at­trib­uted to his de­par­ture from the FDA com­mit­tee. By June 2019, just a month af­ter Bio­gen ex­ec­u­tives met with Dunn, of­fi­cials in his of­fice pro­posed the ac­cel­er­at­ed ap­proval route — de­spite doc­u­ments from the FDA that said the agency first dis­cussed the pos­si­bil­i­ty of ac­cel­er­at­ed ap­proval on March 31, ac­cord­ing to the STAT re­port.

Bil­ly Dunn

“It was clear that Bil­ly Dunn was an al­ly, so the job for Bio­gen be­came fig­ur­ing out how to sup­port his ef­forts with­in the FDA,” a for­mer Bio­gen em­ploy­ee anony­mous­ly told STAT.

The FDA grant­ed ad­u­canum­ab — now mar­ket­ed as Aduhelm — ap­proval in ear­ly June, as the first drug to slow the pro­gres­sion of Alzheimer’s. In the years lead­ing up to the OK, near­ly every ef­fort to treat Alzheimer’s had failed, even as the num­ber of Amer­i­cans liv­ing with the dis­or­der grew to more than 6 mil­lion. The last ap­proved drug came in 2003 and on­ly tem­porar­i­ly boosts mem­o­ry. The ap­proval re­mains high­ly con­tro­ver­sial as the FDA ig­nored the near-unan­i­mous rec­om­men­da­tion of its ad­vi­so­ry com­mit­tee, which said Bio­gen cher­ry-picked da­ta af­ter the drug failed two clin­i­cal tri­als.

Al San­drock

Ad­u­canum­ab seemed dead in the wa­ter in 2019 when da­ta from those two clin­i­cal tri­als showed a flop on the pri­ma­ry end­points. Bio­gen’s stock dropped 30% and the com­pa­ny lost $16 bil­lion in mar­ket val­ue. When sci­en­tists lat­er found in post hoc analy­ses that the drug did demon­strate ef­fec­tive­ness against Alzheimer’s, Bio­gen reached out to Dunn and asked for his help. Near­ly im­me­di­ate­ly af­ter, chief sci­en­tist Al San­drock met with Dunn in Philadel­phia for an off-the-record, off-the-books meet­ing, STAT re­port­ed.

Dunn, now the di­rec­tor of the FDA’s of­fice of neu­ro­science, laid out a red car­pet for the com­pa­ny, of­fer­ing a hearty en­dorse­ment of the drug de­spite what Caleb Alexan­der, an epi­demi­ol­o­gist at Johns Hop­kins Uni­ver­si­ty and ad­comm mem­ber, called “dozens of red flags” in the da­ta in front of a room filled with skep­tics.

Dur­ing that No­vem­ber 2020 pan­el dis­cus­sion, Dunn brushed aside an FDA sta­tis­ti­cian’s con­cerns about the lack of ef­fi­ca­cy. His tone was deemed pro­mo­tion­al.

“At times, Dunn was ad­vo­cat­ing more strong­ly for Aduhelm than [Saman­tha] Budd Hae­ber­lein, the Bio­gen ex­ec­u­tive pre­sent­ing the com­pa­ny’s clin­i­cal da­ta,” the STAT sto­ry reads. “When she strug­gled to an­swer ques­tions posed by the pan­el, Dunn stepped in to re­spond.”

Three mem­bers of the FDA’s Pe­riph­er­al and Cen­tral Ner­vous Sys­tem Drugs Ad­vi­so­ry Com­mit­tee re­signed af­ter the ap­proval, with Kessel­heim call­ing it “prob­a­bly the worst drug ap­proval de­ci­sion in re­cent U.S. his­to­ry.”

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.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

When will the FDA re­scind a break­through des­ig­na­tion? New draft guid­ance spells out the com­pli­cat­ed de­tails

Although it’s exceedingly rare for the FDA to rescind a breakthrough designation once it’s granted, there has been a recent uptick — as in 2020 and 2021 combined, the agency rescinded 17 BTDs, compared to just 18 rescinded from 2015 to 2019 combined.

Protagonist Therapeutics saw this reality up close and personal in April after a clinical hold lifted on its experimental blood cancer drug, as the company revealed that the FDA sought to revoke the BTD. The decision, Protagonist says, stems from “observed malignancies” related to the hold, initially imposed in September 2021.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

FDA warns Mex­i­can glyc­erin man­u­fac­tur­er for re­fus­ing an in­spec­tion

A drug manufacturing facility in Mexico is drawing the ire of the FDA after it ignored the US regulator’s inspection requests and phone calls.

According to the warning letter issued on June 13, Glicerinas Industriales refused a pre-announced inspection during a phone call with FDA prior to the inspection at the company’s facility in Zapopan, Mexico, a city next to Guadalajara, which was planned for May 16 to May 20.