November 25, 2019 04:17 PM ESTUpdated 04:51 PM
R&D
FDA+

Make that 2 new sick­le cell dis­ease block­buster hope­fuls OK’d by the FDA in just a few days

John Carroll

Editor & Founder

Just days after Novartis scored a ‘breakthrough’ OK with their new drug crizanlizumab (Adakveo) to prevent vaso-occlusive crises (VOCs) among sickle cell disease patients, Global Blood Therapeutics $GBT has followed up and nabbed their own regulatory green light in the field.

Based on data from a study demonstrating a 51% increase in the rate of hemoglobin response among patients — compared to only 6.5% in the placebo group — regulators stamped an approval on Oxbryta, better known until now as voxelotor. The company calls this drug “transformative.” But the therapy failed to show a statistically significant improvement in the rate of VOCs, which is what sends patients to the hospital.

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March 20, 2023 06:00 AM EDT

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

Sean Dodge

Healthcare Technology Analyst, RBC Capital Markets

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.

March 20, 2023 11:47 AM EDTUpdated 11:58 AM
R&D
FDA+

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

Max Gelman

Senior Editor

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)
March 18, 2023 07:00 AM EDT
Deals
R&D

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

John Carroll

Editor & Founder

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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March 21, 2023 03:46 PM EDT
FDA+
Manufacturing

FDA warns Proc­ter & Gam­ble over NyQuil la­bel's in­gre­di­ent list­ings

Tyler Patchen

News Reporter

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.

March 21, 2023 03:16 PM EDT
Pharma
Marketing

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

Beth Snyder Bulik

Senior Editor

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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Vipin Garg, Altimmune CEO
March 21, 2023 11:24 AM EDT
R&D

Al­tim­mune’s shares halved af­ter in­ter­im look at PhII weight loss drug da­ta

Kyle LaHucik

Associate Editor

Altimmune’s attempt to catch up to Novo Nordisk and Eli Lilly’s GLP-1 drugs hit an investor snag Tuesday after the biotech shared interim Phase II weight loss data.

The Maryland biotech’s pemvidutide is a GLP-1/glucagon dual receptor agonist meant to activate GLP-1 receptors to squash appetite and glucagon to ramp up energy use. The 2.4 mg dose showed a placebo-adjusted weight loss of 9.7% at week 24 of 48, which Jefferies analysts said would be comparable to Novo Nordisk’s semaglutide (Wegovy) and Eli Lilly’s tirzepatide (Mounjaro).

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March 21, 2023 03:26 PM EDT
Pharma
Law

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

Nicole DeFeudis

Editor

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.

March 21, 2023 03:06 PM EDT
Pharma
Manufacturing

Growth hor­mone from No­vo Nordisk is in short­age over man­u­fac­tur­ing de­lays

Tyler Patchen

News Reporter

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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March 21, 2023 03:05 PM EDT
Pharma

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

Paul Schloesser

Associate Editor

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.

Make that 2 new sick­le cell dis­ease block­buster hope­fuls OK’d by the FDA in just a few days

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

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

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

FDA warns Proc­ter & Gam­ble over NyQuil la­bel's in­gre­di­ent list­ings

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

Al­tim­mune’s shares halved af­ter in­ter­im look at PhII weight loss drug da­ta

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

Growth hor­mone from No­vo Nordisk is in short­age over man­u­fac­tur­ing de­lays

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