FDA user fee reau­tho­riza­tions turn in­to rare bi­par­ti­san love­fest at House hear­ing

Leav­ing any hint of par­ti­san­ship to the dust, the sub­com­mit­tee on health of the House’s en­er­gy and com­merce com­mit­tee came to­geth­er across both sides of the aisle to sup­port not on­ly the user fee reau­tho­riza­tions that pro­vide for the ma­jor­i­ty of FDA’s bud­get, but al­so the add-on bills that would re­form the agency’s ac­cel­er­at­ed ap­proval path­way among oth­er mea­sures.

On the ac­cel­er­at­ed ap­proval front, the bill, which ad­vanced to the full com­mit­tee next week by a unan­i­mous vote of 30-0, would al­low the FDA to re­quire con­fir­ma­to­ry tri­als to be un­der­way pri­or to grant­i­ng these ap­provals as part of re­forms to shore up com­pa­nies that drag their feet on such tri­als. It would al­so stream­line the FDA’s abil­i­ty to with­draw ac­cel­er­at­ed ap­provals when con­fir­ma­to­ry tri­als fail, and it re­quires the agency to ex­plain it­self when not re­quir­ing a con­fir­ma­to­ry tri­al.

Kick­ing off with an up­beat tone at Wednes­day’s markup, sub­com­mit­tee chair An­na Es­hoo (D-CA) said that the House can pass these user fee reau­tho­riza­tions and the ad­di­tion­al bills “with plen­ty time” be­fore the fi­nal Sep­tem­ber dead­line.

Like col­leagues on both side of the aisle, Es­hoo praised the clin­i­cal tri­al di­ver­si­ty rid­er pro­vi­sions in the bill, as well as oth­ers that the FDA needs to catch up with its 2-year in­spec­tion back­log, and on lev­el­ing the play­ing field for US and for­eign in­spec­tions.

How­ev­er, one of the few groups tak­ing is­sue with the cur­rent rid­ers in the House is the in­dus­try lob­by­ing group PhRMA.

“As the leg­is­la­tion moves for­ward, it is im­por­tant that the in­tegri­ty of the reau­tho­riza­tion of these crit­i­cal pro­grams not be un­der­mined by the in­clu­sion of ex­tra­ne­ous poli­cies that would slow the reau­tho­riza­tion process and jeop­ar­dize the US Food and Drug Ad­min­is­tra­tion’s (FDA) time­ly re­view and ap­proval of crit­i­cal new med­i­cines,” PhRMA said in a state­ment.

But oth­er than some ques­tions Es­hoo raised about a pro­vi­sion that would al­so re­verse a de­ci­sion from a hot­ly-con­test­ed court case, Re­pub­li­cans and De­moc­rats spent near­ly all of their time prais­ing the var­i­ous ben­e­fits of the pro­vi­sions in­clud­ed in the bill.

For in­stance, Rep. Ang­ie Craig (D-MN) praised one new rid­er that will al­low a gener­ic drug to be ap­proved even if its pro­posed la­bel­ing dif­fers from that of the brand drug (pre­vi­ous­ly not al­lowed).

Re­pub­li­cans’ top mem­ber on the sub­com­mit­tee, Brett Guthrie, (KY) said he looks “for­ward to make sure this gets signed in­to law” as it will help the US sup­ply chain, in­crease bio­phar­ma in­no­va­tion and help those with un­met needs. While pledg­ing more cures will come to mar­ket soon­er and at low­er costs, he al­so praised the in­clu­sion of the bill’s re­quire­ment that FDA is­sue fur­ther guid­ance on how RWE can help get more drugs ap­proved, Guthrie said.

Rep. Cathy Mc­Mor­ris Rodgers (R-WA) al­so of­fered her sup­port for the bill, say­ing that it will push FDA to em­brace change and use nov­el ways to de­vel­op drugs in­stead of an­i­mal mod­els.

Chair of the full E&C com­mit­tee Rep. Frank Pal­lone (D-NJ) al­so said the com­mit­tee wants to move quick­ly so the FDA doesn’t have to send out pink slips when the in­dus­try funds dry up.

He al­so not­ed that while Cures 2.0 was not a part of to­day’s markup, he’s pleased with the progress on sev­er­al of the pri­or­i­ties. He al­so not­ed that to­day’s bill in­cludes pro­vi­sions “on shared pri­or­i­ties such as ad­vanc­ing re­al world ev­i­dence, de­vel­op­ing end­points for rare dis­eases, and in­creas­ing di­ver­si­ty in clin­i­cal tri­als. We are al­so go­ing to con­tin­ue to seek tech­ni­cal as­sis­tance from the Ad­min­is­tra­tion and work on a bi­par­ti­san ba­sis to ad­vance the rest of the CURES 2.0 leg­is­la­tion.”

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

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Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Am­gen, years be­hind ri­vals, says PhI obe­si­ty drug shows dura­bil­i­ty signs

While NBC ran “The Biggest Loser” for 17 seasons, deemed toxic by critics for the reality show’s punishing exercise and diet upheavals, researchers in pharmaceutical labs have been attempting to create prescription drugs that induce weight loss — and one pharma betting it can require less frequent dosing is out with a new crop of data.

Amgen was relatively late to the game compared to its approved competitor Novo Nordisk and green light-approaching rival Eli Lilly. But early data suggested Amgen’s AMG 133 led to a 14.5% weight reduction in the first few months of dosing, buoying shares earlier this fall, and now the California pharma is out with its first batch of durability data showing that figure fell slightly to 11.2% about 150 days after the last dose. Amgen presented at the 20th World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease on Saturday afternoon.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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John Evans, Beam Therapeutics CEO

Beam's base-edit­ed al­lo­gene­ic CAR-T gets FDA go-ahead af­ter four-month wait

The FDA wanted more information on four key areas before it would let Beam Therapeutics proceed with human testing for a cell therapy in a certain type of leukemia. It appears the biotech has answered the agency’s queries.

The US regulator cleared the base-edited, off-the-shelf CAR-T, Beam said Friday morning, lifting a hold from this summer. More details on specific next steps for the Phase I will come out next year, the Boston-area biotech said.

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Tim Van Hauwermeiren, argenx CEO

Ar­genx pur­chas­es $100M+ FDA pri­or­i­ty re­view vouch­er from blue­bird bio

Argenx’s Vyvgart is due for a speedy review at the FDA, thanks to a $102 million priority review voucher (PRV).

The Netherland-based biotech picked up the PRV from bluebird bio, the companies announced on Wednesday. PRVs shorten a drug’s FDA review period from 10 months to 6 months, though they often sell on the open market for around $100 million each.

Argenx plans on using the express ticket on efgartigimod, its neonatal Fc receptor (FcRn) blocker marketed as Vyvgart for adults with generalized myasthenia gravis (gMG). While Vyvgart won its first approval last December for the chronic neuromuscular disease — which is characterized by difficulties with facial expression, speech, swallowing and breathing — CEO Tim Van Hauwermeiren said in a news release that he plans to “be active in fifteen disease targets by 2025.”

Lex­i­con slams FDA over hear­ing de­nial fol­low­ing a CRL for its SGLT2 in­hibitor can­di­date

Lexicon Pharmaceutical is not giving up on its Type I diabetes candidate, despite FDA’s repeated rejections. This week the company laid out is argument again for a hearing on sotagliflozin in response to the FDA’s most recent denial.

The issue goes back to March 2019 when the FDA made very clear to Lexicon and its now departed partner Sanofi that it would not approve their application for a potential Type I diabetes drug because it does not appear to be safe.

US month­ly costs for biosim­i­lars 'sub­stan­tial­ly high­er' than Ger­many or Switzer­land, JA­MA re­search finds

As the global biologics market is expected to hit nearly the half-trillion-dollar mark this year, new JAMA research points to the importance of timely biosimilar entry, particularly as fewer biosimilars are entering the US than in Europe, and as monthly treatment costs for biosimilars were “substantially higher” in the US compared with Germany and Switzerland.

Among the three countries, biosimilar market share at launch was highest in Germany, but increased at the fastest rate in the US, the authors from the University of Zurich’s Institute of Law wrote in JAMA Network Open today.