FDA push­es back a re­view date for TG's 'U2' com­bo as the biotech steels it­self for up­com­ing ad­comm

The FDA has pushed back its re­view of TG Ther­a­peu­tics’ U2 com­bo (ubli­tux­imab plus Ukoniq) in pa­tients with chron­ic lym­pho­cyt­ic leukemia and small lym­pho­cyt­ic lym­phoma af­ter the drug­mak­er sub­mit­ted up­dat­ed sur­vival da­ta in Feb­ru­ary the agency deemed a ma­jor amend­ment to its ap­pli­ca­tion, ac­cord­ing to a re­lease.

The agency set a new re­view date of June 25, TG said.

The news wasn’t un­ex­pect­ed giv­en some of TG’s re­cent com­ments. In a state­ment, CEO Michael Weiss had this to say:

As men­tioned on our earn­ings call ear­li­er this week, we be­lieved an ex­ten­sion of the PDU­FA date was a like­ly sce­nario es­pe­cial­ly giv­en the pro­posed tim­ing of the up­com­ing ODAC meet­ing. We hope this ex­ten­sion pro­vides the time need­ed to give prop­er at­ten­tion and re­view to the U2 BLA/sN­DA.

With its re­view date pushed out, TG is now star­ing di­rect­ly at an up­com­ing ad­comm for U2’s ap­pli­ca­tion here, which the FDA called back in No­vem­ber. The agency at the time in­di­cat­ed to the biotech that it had more ques­tions on the OS end­point and safe­ty da­ta in the piv­otal Phase III study un­der­gird­ing its ap­pli­ca­tion.

In an up­date Thurs­day, TG said the FDA had sent a list of po­ten­tial ques­tions for that ad­comm, for which a date still hasn’t been sched­uled. Here’s what the com­pa­ny said could be on the dock­et:

The FDA has no­ti­fied the Com­pa­ny that po­ten­tial ques­tions and dis­cus­sion top­ics for the ODAC in­clude: the ben­e­fit-risk of the U2 com­bi­na­tion in the treat­ment of CLL or SLL, and the ben­e­fit-risk of Ukoniq in re­lapsed/re­frac­to­ry mar­gin­al zone lym­phoma (MZL) or fol­lic­u­lar lym­phoma (FL). In ad­di­tion, as part of the ben­e­fit-risk analy­sis, the over­all safe­ty pro­file of the U2 reg­i­men, in­clud­ing ad­verse events (se­ri­ous and Grade 3-4), dis­con­tin­u­a­tions due to ad­verse events, and dose mod­i­fi­ca­tions, is ex­pect­ed to be re­viewed. The FDA’s con­cern giv­ing rise to the ODAC meet­ing ap­pears to stem from an ear­ly analy­sis of over­all sur­vival from the UNI­TY-CLL tri­al.

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.

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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Alaa Halawa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

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Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

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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.

FDA re­jects Ab­b­Vie's in­fu­sion ther­a­py for Parkin­son's, re­quests more in­fo on pump de­vice

The FDA rejected AbbVie’s 24-hour infusion therapy for Parkinson’s, saying it needs more information on a device used to administer the treatment before it can clear it.

The Chicago-area drugmaker said in a press release that the complete response letter from the agency didn’t include any requests for more efficacy or safety trials related to the drug, known as ABBV-951. The company said it aims to “resubmit the NDA as soon as possible.”

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FDA+ roundup: Leg­isla­tive asks for 2024 fo­cus on more au­thor­i­ties, gener­ic drug com­pe­ti­tion

The FDA’s legislative priorities for the next year highlight the agency’s focus on expanding generic drug competition, backstopping the supply chain and growing its current authorities.

On the new authorities front, FDA is seeking to expand its mandatory recall authority for all drugs, as the agency has been embroiled in a long process to remove some from the market. Covis Pharma refused to pull its preterm birth drug Makena, which won accelerated approval, for almost five years after failing its confirmatory trial. The company has since reversed course after a negative adcomm.

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FDA in­di­cates will­ing­ness to ap­prove Bio­gen ALS drug de­spite failed PhI­II study

Ahead of Wednesday’s advisory committee hearing to discuss Biogen’s ALS drug tofersen, the FDA appeared open to approving the drug, newly released briefing documents show.

Citing the need for flexibility in a devastating disease like ALS, regulators signaled a willingness to consider greenlighting tofersen based on its effect on a certain protein associated with ALS despite a failed pivotal trial. The documents come after regulatory flexibility was part of the same rationale the agency expressed when approving an ALS drug last September from Amylyx Pharmaceuticals, indicating the FDA’s openness to approving new treatments for the disease.

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Sanofi, Re­gen­eron boast PhI­II win with Dupix­ent in COPD, clear­ing first bar for ex­pan­sion

Dupixent, the blockbuster anti-inflammatory drug from Sanofi and Regeneron, has cleared a high-stakes Phase III study in chronic obstructive pulmonary disease, the companies announced Thursday morning.

If they hold up in a second, identical trial, the data pave the way for Dupixent to become the first biologic to treat patients whose COPD remains uncontrolled despite being on maximal standard-of-care inhaled therapy — the patient population studied in the pivotal program. The companies had spotlighted this as a key readout as they look to expand the Dupixent franchise and explore its full potential.