Susan Galbraith, AstraZeneca oncology chief

Af­ter 18 years in de­vel­op­ment, As­traZeneca’s CT­LA-4 an­ti­body gets the red car­pet treat­ment at the FDA

Af­ter a decade, a sec­ond CT­LA-4 an­ti­body may fi­nal­ly be on its way to ap­proval.

As­traZeneca an­nounced Mon­day that the FDA had giv­en pri­or­i­ty re­view to treme­li­mum­ab, its long-ges­tat­ing CT­LA-4 an­ti­body, in com­bi­na­tion with the PD-L1 in­hibitor Imfinzi for pa­tients with liv­er can­cer that can’t be treat­ed with surgery.

The Big Phar­ma’s ap­pli­ca­tion is based on a mul­ti-year, 1,324-pa­tient study that showed 30.7% of pa­tients who re­ceived a “prim­ing dose” of treme­li­mum­ab fol­lowed by Imfinzi were alive af­ter three years, com­pared to 20.2% of pa­tients who re­ceived Bay­er’s so­rafenib, the stan­dard of care.

Su­san Gal­braith, As­traZeneca’s can­cer R&D chief, called that da­ta “un­prece­dent­ed.” Oth­ers raised ques­tions about how the da­ta held up com­pared to an al­ter­na­tive treat­ment reg­i­men be­ing de­vel­oped by Roche, and how much ben­e­fit the CT­LA-4 an­ti­body of­fered, giv­en that pa­tients who re­ceived Imfinzi alone showed near­ly iden­ti­cal sur­vival at the two-year mark. The dif­fer­ence on­ly emerged af­ter three years.

Nev­er­the­less, the three-year da­ta were no­table giv­en how oth­er PD-1 in­hibitors have done in the same set­ting when test­ed on their own. Op­di­vo failed to show an ad­van­tage over stan­dard of care in one tri­al, and Mer­ck has strug­gled to show that Keytru­da of­fers a mean­ing­ful ad­van­tage ei­ther.

Treme­li­mum­ab has had plen­ty of its own stum­bles. Pfiz­er first put the drug in tri­als in 2004, but aban­doned it soon af­ter, fol­low­ing dis­ap­point­ing ear­ly re­sults. It was still years be­fore Bris­tol My­ers Squibb showed block­ing CT­LA-4 could dra­mat­i­cal­ly ex­tend the lives of a sub­set of melanoma pa­tients, jump­start­ing the im­muno-on­col­o­gy field and mak­ing tens of bil­lions of dol­lars for Mer­ck and Bris­tol My­ers.

As­traZeneca li­censed Pfiz­er’s drug in 2011, hop­ing to get in on the still nascent im­muno-on­col­o­gy field, but it failed con­tin­u­al­ly in tri­als, in­clud­ing in lung, urothe­lial, and head and neck can­cer.

Al­though the drug would on­ly be OK’d for a lim­it­ed in­di­ca­tion, an ap­proval would make it the sec­ond CT­LA-4 an­ti­body on the mar­ket and the first con­tender to Bris­tol My­ers’ Yer­voy since it was ap­proved in 2011.

As­traZeneca is al­so hop­ing to get an ap­proval soon in com­bi­na­tion with Imfinzi in metasta­t­ic lung can­cer, al­though it is un­clear how much ben­e­fit the new drug of­fers over Imfinzi alone.

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 warns Proc­ter & Gam­ble over NyQuil la­bel's in­gre­di­ent list­ings

The FDA on Tuesday released a warning letter sent earlier this month to the Mason, OH-based site of Procter & Gamble Manufactura, raising questions about the list of ingredients on the label and in the electronic filing.

The warning says that for P&G’s over-the-counter Vicks Nyquil Severe Hot Remedy Cold and Flu Plus Congestion, there’s a “mismatched” list of active ingredients between the labeling and the electronic listing file. The listing file for the active ingredients did not match the active ingredients in the electronic file.

No­vo Nordisk re­mains un­der UK scruti­ny as MHRA con­ducts its own re­view in 'in­cred­i­bly rare' case

The UK’s Medicines and Healthcare products Regulatory Agency is now reviewing Novo Nordisk’s marketing violation that resulted in its loss of UK trade group membership last week. Novo Nordisk was suspended on Thursday from the Association of the British Pharmaceutical Industry (ABPI) for two years after an investigation by its regulatory arm found the pharma broke its conduct rules.

MHRA said on Tuesday that its review of the Prescription Medicines Code of Practice Authority (PMCPA) investigation is standard practice. An MHRA spokesperson emphasized in an email to Endpoints News that the situation with Novo Nordisk is “incredibly rare” while also noting ABPI took “swift and proportionate action.”

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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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Andy Plump, Takeda R&D chief (Jeff Rumans for Endpoints News)

What kind of PhI­Ib da­ta is worth $4B cash? Take­da’s Andy Plump has some thoughts on that

A few months back, when Takeda caused jaws to drop with its eye-watering $4 billion cash upfront for a mid-stage TYK2 drug from Nimbus, it had already taken a deep dive on the solid Phase IIb data Nimbus had assembled from its dose-ranging study in psoriasis.

Now, it’s rolling that data out, eager to demonstrate what inspired the global biopharma to go long in a neighboring, but new, disease arena for the pipeline. And the most avid students of the numbers will likely be at Bristol Myers Squibb, who will have a multi-year head start on pioneering the TYK2 space with Sotyktu (deucravacitinib) as Takeda makes its lunge for best-in-class status.

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FTC says patent bat­tle over Parkin­son's drug could have 'sig­nif­i­cant im­pli­ca­tion­s' for pa­tients

The Federal Trade Commission has gotten involved in a patent feud over Supernus’ Parkinson’s drug Apokyn, a case the agency said may have ‘‘significant implications” for patients who rely on the drug.

Sage Chemical won the first generic approval for its Apokyn formulation (also known as apomorphine hydrochloride injection) back in 2022. The non-ergoline dopamine agonist is approved to treat Parkinson’s symptoms during “off episodes,” such as difficulty moving, tremors and intense cramping. However, regulators specified that the approval pertained to the generic drug cartridges only, not the injector pen required for administration.

Growth hor­mone from No­vo Nordisk is in short­age over man­u­fac­tur­ing de­lays

Novo Nordisk’s growth hormone Norditropin is in shortage because of manufacturing delays, according to an FDA site that tracks drug shortages as well as the American Society of Health-System Pharmacists’ shortages list.

The FDA has shortages of the drug listed for its 5, 10, 15 and 30 mg doses, while the pharmacists’ group, also known as ASHP, reported shortages of the same doses, except for the 15 mg version.

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PhRMA calls for more di­verse in­fra­struc­ture up­grades to US emer­gency tri­als frame­work

The White House’s Office of Science and Technology Policy (OSTP) last year sought to find ways to better coordinate large-scale clinical trials in the US — as the UK lead by example during the pandemic — especially for these emergency clinical trials.

The lobbying group PhRMA Tuesday called for more clinical trial diversity in underserved areas, including by making participation less of a burden, and expanding eligibility criteria when appropriate.

Mar­ket­ingRx roundup: What could a US Tik­Tok ban mean for phar­ma? Pfiz­er, Lil­ly lead phar­ma March Mad­ness ad­ver­tis­ers

Just as pharma marketers finally make moves into TikTok, the threat of a US ban on the social media channel is now looming. Already banned on federal employee phones by an initial Congressional act, more bills and maybe bans are on the way. With rare bipartisan agreement, lawmakers have introduced legislation that would give the US president the power to ban TikTok (although not mentioned by name) and other foreign-owned technology platforms that represent a security threat to the US.

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