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.”

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Phar­ma com­pa­ny con­tin­ues its FDA law­suit spree, this time af­ter agency de­nies fast-track des­ig­na­tion

Vanda Pharmaceuticals is making a name for itself, at least in terms of suing the FDA.

The DC-headquartered firm on Monday filed its latest suit against the agency, with the company raising concerns over the FDA’s failure to grant a fast track designation for Vanda’s potential chronic digestive disorder drug tradipitant, which is a neurokinin 1 receptor antagonist.

Specifically, Vanda said FDA’s “essential point” in its one-page denial letter on the designation pointed to “the lack of necessary safety data,” which was “inconsistent with the criteria for … Fast Track designation.”

Robert Califf (Michael Brochstein/Sipa USA via AP Images)

House Re­pub­li­cans at­tack Chi­na-on­ly da­ta in FDA sub­mis­sions, seek new in­ves­ti­ga­tion in­to re­search in­spec­tions

Three Republican representatives are calling on the FDA to take a closer look at the applications including only clinical data from China.

The letter to FDA commissioner Rob Califf late last week comes as the agency recently rejected Eli Lilly’s anti-PD-1 antibody, which attempted to bring China-only data but ran into a bruising adcomm that may crush the hopes of any other companies looking to bring cheaper follow-ons based only on Chinese data.

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Janet Woodcock (Greg Nash/Pool via AP Images)

'I re­al­ly don’t look back': Janet Wood­cock on her tran­si­tion away from drugs

Janet Woodcock may have one of the most historically long and drug-intense tenures in FDA history, but her new role is outside of all things pharma and the once-acting FDA commissioner isn’t looking back.

“No I really don’t look back,” Woodcock told Endpoints News via email on Monday morning. “Yes I will be transitioning. Longer discussion on infrastructure needed.”

Mod­er­na seeks to dis­miss Al­ny­lam suit over Covid-19 vac­cine com­po­nent, claim­ing wrong venue

RNAi therapeutics juggernaut Alnylam Pharmaceuticals made a splash in March when it sued and sought money from both Pfizer and Moderna regarding their use of Alnylam’s biodegradable lipids, which Alnylam claims have been integral to the way both companies’ mRNA-based Covid-19 vaccines work.

But now, Moderna lawyers are firing back, telling the same Delaware district court that Alnylam’s claims can only proceed against the US government in the Court of Federal Claims because of the way the company’s contract is set up with the US government. The US has spent almost $10 billion on Moderna’s Covid-19 vaccine so far.

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(Credit: Shutterstock)

Cracks in the fa­cade: Is phar­ma's pan­dem­ic ‘feel good fac­tor’ wan­ing?

The discordant effects of the Covid-19 pandemic on pharma reputation continues. While the overall industry still retains a respectable halo from its Covid-19 quick response and leadership, a new patient group study reveals a different story emerging in the details.

On one hand, US patient advocacy groups rated the industry higher-than-ever overall. More than two-thirds (67%) of groups gave the industry a thumbs up for 2021, a whopping 10 percentage point increase over the year before, according to the PatientView annual study, now in its 9th year.

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Michael Corbo, Pfizer CDO of inflammation & immunology

UP­DAT­ED: Plan­ning ahead for crowd­ed ul­cer­a­tive col­i­tis mar­ket, Pfiz­er spells out PhI­II da­ta on $6.7B Are­na drug

Pfizer has laid out the detailed results behind its boast that etrasimod — the S1P receptor modulator at the center of its $6.7 billion buyout of Arena Pharma — is the winner of the class, potentially leapfrogging an earlier entrant from Bristol Myers Squibb.

Pivotal data from the ELEVATE program in ulcerative colitis — which consists of two Phase III trials, one lasting 52 weeks and the other just 12 weeks — illustrate an “encouraging balance of efficacy and safety,” according to Michael Corbo, chief development officer of inflammation & immunology at Pfizer. The company is presenting the results as a late breaker at Digestive Disease Week.

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Todd Zavodnick, Dermavant CEO

With top­i­cal ap­proval, Der­ma­vant looks to bring new stan­dard-of-care to plaque pso­ri­a­sis pa­tients

Dermavant CEO Todd Zavodnick has been plotting to upend the plaque psoriasis market for years now. And with the company’s first approval on Tuesday, he’s pulling the trigger.

The FDA on Tuesday approved Dermavant’s “cosmetically elegant” vanishing cream tapinarof, the first steroid-free topical medication in its class, now marketed as Vtama. And unlike other launches, which could take months, Zavodnick is ready to roll it out as early as next week.

Raymond Tesi, INmune Bio CEO

Man­u­fac­tur­ing con­cerns spur clin­i­cal hold on small biotech's Alzheimer's tri­al — shares plunge

The FDA is keeping an experimental Alzheimer’s therapy out of the US for now, placing a clinical hold on INmune Bio’s IND for a Phase II trial until it can provide additional information on chemistry, manufacturing and controls.

INmune Bio, which gets its name from the approach of leveraging the innate immune system to fight disease, said it was informed about the hold via email and expects more details in a later letter. In a brief statement, it said the agency was looking for more information about CMC “of the newly manufactured XPro1595.”

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