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.”

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Eu­ro­pean Com­mis­sion de­lays pro­pos­al for ma­jor changes to phar­ma leg­is­la­tion

The European Commission has once again delayed the release of its proposal for an overhaul of the continent’s pharmaceutical legislation.

The release, previously anticipated on March 29, will occur “slightly later” than expected due to the “very busy College agendas of the last few weeks,” a Commission spokesperson told Endpoints News via email.

While the agency hasn’t provided an updated timeline, the spokesperson said the agenda is “always indicative and adoption dates of Commission proposals may change any time, especially when these proposals concern reforms of complex legislations of major importance.”

In­cyte wins ac­cel­er­at­ed ap­proval for PD-1 in rare skin can­cer

Incyte touted an accelerated approval for its PD-1 retifanlimab in a rare skin cancer on Wednesday, roughly a year and a half after the drug suffered a rejection in squamous cell carcinoma of the anal canal (SCAC).

Retifanlimab, marketed as Zynyz, was approved for metastatic or recurrent locally advanced Merkel cell carcinoma (MCC), a fast-growing skin cancer typically characterized by a single, painless nodule. It’s roughly 40 times rarer than melanoma, according to the nonprofit Skin Cancer Foundation — but incidence is growing, particularly among older adults, Incyte said in its announcement.