E&C chair Frank Pallone (Kevin Dietsch/Pool via AP Images)

Ac­cel­er­at­ed ap­proval re­forms take cen­ter stage at House hear­ing, but a way for­ward re­mains un­clear

The House En­er­gy & Com­merce com­mit­tee’s health sub­com­mit­tee on Thurs­day dug in­to al­most two dozen dif­fer­ent FDA- and phar­ma-re­lat­ed bills that may be tacked on­to a larg­er bill or even the bio­phar­ma user fee bills, which have to be reau­tho­rized be­fore the end of Sep­tem­ber.

The mas­sive batch of bills mak­ing their way through the House in­cludes two crit­i­cal bills that seek to re­form the FDA’s ac­cel­er­at­ed ap­proval process in dif­fer­ent ways.

On the one hand is E&C chair Frank Pal­lone (D-NJ), who, to the cha­grin of in­dus­try, is seek­ing ex­pi­ra­tion dates (5 years max) on ac­cel­er­at­ed ap­provals giv­en the fact that com­pa­nies of­ten drag their feet on con­fir­ma­to­ry tri­als. His bill is run­ning in­to op­po­si­tion from Re­pub­li­can Cathy Mc­Mor­ris Rodgers of Wash­ing­ton, who sought to make clear the cur­rent ac­cel­er­at­ed path­way is func­tion­ing well, but that the path­way needs to be mod­ern­ized for some new drugs, like those for ALS, that don’t have a clear sur­ro­gate end­point.

Mc­Mor­ris Rodgers’ bill would make it so that the ev­i­dence used to sup­port that an end­point is rea­son­ably like­ly to pre­dict a clin­i­cal ben­e­fit un­der an ac­cel­er­at­ed ap­proval “may in­clude epi­demi­o­log­i­cal, patho­phys­i­o­log­i­cal, ther­a­peu­tic, phar­ma­co­log­ic, or oth­er ev­i­dence de­vel­oped us­ing bio­mark­ers, for ex­am­ple, or oth­er sci­en­tif­ic meth­ods or tools.”

Pal­lone, mean­while, of­fered the ex­am­ple of Bio­gen’s con­tro­ver­sial new Alzheimer’s drug Aduhelm, which won an ac­cel­er­at­ed ap­proval last June.

“Here we are nine months lat­er, and the spon­sor has not screened a sin­gle pa­tient for its re­quired con­fir­ma­to­ry tri­al,” Pal­lone said. “Oth­er drugs have stayed on the mar­ket for eight or nine years with­out prov­ing a clin­i­cal ben­e­fit.”

He al­so not­ed how CDER Di­rec­tor Pa­trizia Cavaz­zoni tes­ti­fied last month on how “the process for re­mov­ing these drugs from the mar­ket is cum­ber­some and can take months or even years.”

Rank­ing mem­ber Rep. Brett Guthrie (R-KY), how­ev­er, took the oth­er side on the Aduhelm de­bate, say­ing that CMS’ de­ci­sion to re­strict cov­er­age to on­ly clin­i­cal tri­als could have a chill­ing ef­fect on oth­er in­vest­ments in Alzheimer’s.

Sup­port­ing the ac­cel­er­at­ed ap­proval sta­tus quo, Jeff Allen, CEO of Friends with Can­cer Re­search, not­ed that ac­cel­er­at­ed ap­provals con­vert­ing to full ap­proval took a me­di­an time of 3.1 years. He al­so said that if ac­cel­er­at­ed ap­proval drugs are in­ap­pro­pri­ate­ly lim­it­ed, there could be de­lays in ac­cess, but the hall­mark of the ac­cel­er­at­ed path­way is bal­anc­ing these un­cer­tain­ties.

“I think the leg­is­la­tion pro­posed to­day to strength­en the post-mar­ket sur­veil­lance side will help to ex­pand the de­vel­op­ment of sur­ro­gate end­points,” he not­ed.

Resh­ma Ra­machan­dran, physi­cian-fel­low in Yale’s Na­tion­al Clin­i­cian Schol­ars Pro­gram, tes­ti­fied that phar­ma com­pa­nies of­ten mar­ket drugs via the ac­cel­er­at­ed path­way for long pe­ri­ods of time pri­or to the con­fir­ma­to­ry tri­al re­sults. And some­times that mar­ket­ing con­tin­ues even af­ter con­fir­ma­to­ry tri­als have failed.

She not­ed that Pal­lone’s bill builds in flex­i­bil­i­ty for FDA on when ap­pro­pri­ate con­fir­ma­to­ry tri­al com­ple­tion date can be set, and it al­lows for ne­go­ti­a­tions be­tween the spon­sor and FDA. That would mean drugs won’t be au­to­mat­i­cal­ly with­drawn if such a drug is still in a con­fir­ma­to­ry tri­al.

In ad­di­tion to ac­cel­er­at­ed ap­provals, the bills dis­cussed at the hear­ing al­so in­clude fur­ther fund­ing and struc­ture for ARPA-H, a new NIH-housed agency (with $1 bil­lion in starter funds) that will cor­rect the gap be­tween the ba­sic re­search at NIH and the de­vel­op­ment of prod­ucts in the pri­vate sec­tor, ac­cord­ing to health sub­com­mit­tee chair An­na Es­hoo (D-CA). Es­hoo al­so praised the years’ worth of work in the lat­est it­er­a­tion of the 21st Cen­tu­ry Cures Act.

Rep. Fred Up­ton (R-MI), who co-spon­sored both Cures bills with Rep. Di­ana DeGette (D-CO), not­ed on Thurs­day that Covid “has taught us a valu­able les­son that FDA can work quick­ly and that RWE will im­prove its de­ci­sion­mak­ing.”

Three oth­er bills dis­cussed would seek to im­prove clin­i­cal tri­al di­ver­si­ty, while oth­ers will help with gener­ic and biosim­i­lar de­vel­op­ment, in­clud­ing one that will al­low some gener­ics to be ap­proved with a “tem­porar­i­ly dif­fer­ent” la­bel than its ref­er­ence prod­uct.

Vas Narasimhan (Photographer: Jason Alden/Bloomberg via Getty Images)

No­var­tis de­tails plans to axe 8,000 staffers as Narasimhan be­gins sec­ond phase of a glob­al re­org

We now know the number of jobs coming under the axe at Novartis, and it isn’t small.

The pharma giant is confirming a report from Swiss newspaper Tages-Anzeiger that it is chopping 8,000 jobs out of its 108,000 global staffers. A large segment will hit right at company headquarters in Basel, as CEO Vas Narasimhan axes some 1,400 of a little more than 11,000  jobs in Switzerland.

The first phase of the work is almost done, the company says in a statement to Endpoints News. Now it’s on to phase two. In the statement, Novartis says:

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Bob Nelsen (Lyell)

As bear mar­ket con­tin­ues to beat down biotech, ARCH clos­es a $3B ear­ly-stage fund

One of the biggest names in biotech investing has a whole lot of new money to spend.

ARCH Venture Partners closed its 12th venture fund early Wednesday morning, the firm said, bringing in almost $3 billion to invest in early-stage biotechs. The move comes about a year and a half after ARCH announced its previous fund, for almost $2 billion back in January 2021.

In a statement, ARCH managing director and co-founder Bob Nelsen appeared to brush off concerns about the broader market troubles, alluding to the downturn that’s seen several biotechs downsize and the XBI fall back to almost pre-pandemic levels.

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Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

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How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

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Aurobindo Pharma co-founders P. V. Ram Prasad Reddy (L) and K. Nityananda Reddy

Au­robindo Phar­ma re­ceives warn­ing let­ter from In­di­a's SEC fol­low­ing more FDA ques­tion marks

Indian-based generics manufacturer Aurobindo Pharma has been in the crosshairs of the FDA for several years now, but the company is also attracting attention from regulators within the subcontinent.

According to the Indian business news site Business Standard, a warning letter was sent to the company from the Securities Exchange Board of India, or SEBI.

The letter is related to disclosures made by the company on an ongoing FDA audit of the company’s Unit-1 API facility in Hyderabad, India as well as observations made by the US regulator between 2019 and 2022.

Ankit Mahadevia, Spero CEO

Spero’s UTI can­di­date gets the CRL ham­mer as the com­pa­ny falls in­to pen­ny stock sta­tus

Spero Therapeutics has been struggling in the past few years, dealing with FDA holds and staff reductions amidst a rough biotech market, and the latest news from the Massachusetts-based company confirms what it anticipated in May: a CRL.

The company was slapped with the no-go for its NDA, the biotech disclosed Monday. The company was seeking approval for tebipenem HBr oral tablets, intended for the treatment of adult patients with complicated urinary tract infection, or cUTI, including pyelonephritis. The FDA had set a PDUFA date of June 27.

Sanofi to cut in­sulin prices for unin­sured from $99 to $35, match­ing the in­sulin cap com­ing through Con­gress

As the House-passed bill to cap the monthly price of insulin at $35 nationwide makes its way for a Senate vote soon, Sanofi announced Wednesday morning that beginning next month it will cut the monthly price of its insulins for uninsured Americans to $35, down from $99 previously.

The announcement from Sanofi, which allows the uninsured to buy one or multiple Sanofi insulins (Lantus, Insulin Glargine U-100, Toujeo, Admelog, and Apidra) at $35 for a 30-day supply effective July 1, follows House passage (232-193) of the monthly cap in March, with just 12 Republicans voting in favor of the measure.

Hank Safferstein, Generian CEO

Astel­las sub­sidiary to part­ner with Pitts­burgh up­start in search for 'un­drug­gable' pro­teins

As Astellas continues its drive to build out its gene therapy portfolio and capabilities, a subsidiary of the Japanese pharma company has entered into a collaboration with a little-known Pittsburgh biotech.

Astellas-owned Mitobridge and Generian Pharmaceuticals announced on Wednesday that they will work together in a new deal for “undruggable” protein targets. Generian will net an undisclosed upfront payment and could get up to $180 million in milestones, should anything from its platform prove successful, as well as single-digit royalties on global net sales.

Adam Simpson, Icosavax CEO

Reel­ing from Covid flop, Icosavax says its RSV can­di­date passed ear­ly test. But in­vestors need some more con­vinc­ing

Three months separated from a disappointing readout of its Covid-19 vaccine, Icosavax is back with what it calls positive topline data for a different VLP vaccine candidate — although investors aren’t impressed.

IVX-121, a vaccine candidate for respiratory syncytial virus (RSV), appeared to generate “robust” immune responses among both young and older adults, as measured by neutralizing antibodies, and appeared generally well-tolerated, Icosavax reported.