STAT+: Peter Marks on creating Operation Warp Speed, but for rare diseases
Peter Marks wants drug developers to ask more stupid questions. It’s part of the top…
Category: drug development
STAT+: European regulators consider appeal over Amylyx ALS drug
LONDON — This week, European regulators are considering an appeal from Amylyx Pharmaceuticals after initially…
Want a biotech buyout? Target cancer
Want to stay on top of the science and politics driving biotech today? Sign up to get…
STAT+: Akero’s treatment for NASH falls short in cirrhosis study
Akero Therapeutics said Tuesday that an experimental medicine failed to show a significant benefit for…
STAT+: A Q&A with the global head of Boehringer Ingelheim’s innovation unit
LONDON — At an event here last week, the German pharmaceutical firm Boehringer Ingelheim outlined…
NIH funds expanded access projects for ALS
Want to stay on top of the science and politics driving biotech today? Sign up to get…
Moderna’s plan to make a single vaccine for Covid, influenza, and RSV is on track
Want to stay on top of the science and politics driving biotech today? Sign up to get…
Scientists want to put carbon footprints on drugs. But it’s hard to get accurate numbers
Anyone picking up a prescription from their pharmacy is used to the reams of paper…
STAT+: Failed biotech gives a rare peek inside the tough breaks — and tough choices — that led to its demise
SAN DIEGO — It was spring 2020, and Histogen, a small biotech with big ambitions…
The major biotech news updates you need to read today
Want to stay on top of the science and politics driving biotech today? Sign up to get…
When is a Wall Street disappointment ‘good enough’ for the FDA?
Want to stay on top of the science and politics driving biotech today? Sign up to get…
STAT+: Mirum’s Chenodal succeeds in late-stage trial for rare metabolic disorder
Mirum Pharmaceuticals reported on Monday that a drug it recently acquired to treat cerebrotendinous xanthomatosis,…
STAT+: Just how much money do drugmakers gain from patent extensions?
Extending patent protection doesn’t just stretch a drug’s profits — in some cases, doing so…
STAT+: Early data are promising for autoimmune disease drug from Roivant Sciences’s Immunovant
Results released Tuesday from an early study of an autoimmune disease drug from Roivant Sciences’s…
STAT+: Ionis inches toward first solo drug launch with late-stage trial success for rare genetic disease therapy
Ionis Pharmaceuticals, a California biotech that has spearheaded the development of RNA-targeting medicines, announced on…
STAT+: Travere kidney drug narrowly fails confirmatory trial following FDA approval
Travere Therapeutics said Thursday that a study meant to confirm the benefit of its newly…
STAT+: Approval setback for EpiPen alternative puts spotlight on common anti-competition tactic
Americans who suffer severe allergic reactions won’t have alternatives to autoinjectors like the Epipen for…
.jpg)
FDA asks for more data before considering needle-free anaphylaxis treatment
ARS Pharma had high hopes that the FDA on Tuesday would approve its flagship product,…
STAT+: New obesity drugs are in the works trying to out-blockbuster Wegovy and Mounjaro
The exploding popularity of drugs like Wegovy and Mounjaro has propelled dozens of companies to…
STAT+: Here are the dozens of weight loss drugs in development to catch a booming market
The blazing success of weight loss drugs like Wegovy and Mounjaro has electrified the pursuit…
ALS advocates say criticism of new drugs misses bigger picture
A diagnosis of ALS has long been seen as a death sentence. But in recent…
STAT+: Why Novartis is changing the name of its research labs
Swiss drugmaker Novartis’ research and development hub has a new name — and a new…
STAT+: Pharmalittle: This fall’s COVID booster rollout; approval for Pfizer’s RSV vaccine
Good morning Pharmalittle students. This is Allison DeAngelis, filling in for Ed Silverman while he’s…
STAT+: Pharmalittle: Mid-year price hikes for U.S. drugs reaches new heights; onetime biotech CEO Vivek Ramaswamy sparks debate at GOP debate
Good morning, Pharmalittle readers. Reporter Andrew Joseph here filling in for Ed from STAT’s London…
STAT+: Drug makers already bet big on mRNA for cancer. Why is Biden going in, too?
WASHINGTON — President Biden’s fledgling health agency, designed to accelerate under-funded research, just set its…
Vivek Ramaswamy’s brashness made waves in the Republican debate. He did the same in biotech
The youngest candidate on Wednesday night’s debate stage didn’t make any friends among his fellow…
STAT+: In the case of a devastating disease, the FDA weighs an experimental drug’s muddled data and a desperate need
Emma Albee knows the experimental drug she takes is not a cure. It won’t allow…
STAT+: Novo Nordisk celebrates landmark Wegovy study, even as supply issues persist
The release of Novo Nordisk’s latest earnings report on Thursday provided a chance for executives…
STAT+: Wegovy’s successful heart trial raises a vital question about weight loss
A landmark study that found the blockbuster obesity drug Wegovy reduces the chance of heart…
STAT+: Drug from Stoke shows lower activity against childhood epilepsy, prompting search for better dose
Stoke Therapeutics is still seeking an effective dose for an experimental drug to treat a…
STAT+: Flagship, Pfizer team up in deal worth $7 billion
Pharma giant Pfizer is turning to venture firm Flagship for new drug development, signing a…
STAT+: Argenx antibody drug significantly slows progression of nerve disorder that robs patients of muscle strength
Belgian drugmaker Argenx said Monday that its antibody treatment called Vyvgart significantly delayed the progression…
STAT+: With momentum on Alzheimer’s therapies, Europe sees delivery challenges ahead, too
LONDON — Starting Sunday, Alzheimer’s specialists will gather for a major conference in Amsterdam, convening at…
A young girl’s custom gene therapy hints at a framework for tailored rare disease treatments
When Timothy Yu developed milasen, a custom drug for a young girl named Mila with…
STAT+: AstraZeneca shares fall after lung cancer drug underwhelms in trial
LONDON — AstraZeneca said on Monday that a new lung cancer treatment outperformed standard-of-care chemotherapy…
STAT+: Moonlake’s less-than-transparent data disclosures produced a cash windfall. Risks remain
For two days starting on Sunday, Moonlake Immunotherapeutics happily crunched numbers and shared results from…
STAT+: After late start, Eli Lilly starts pulling ahead in race for the $30 billion weight loss market
SAN DIEGO — It was Novo Nordisk’s Ozempic and Wegovy that set off a new…
Opinion: A seemingly small semantic issue is a major roadblock to develop treatments for rare diseases
Finding a new medicine is never easy. But developing treatments for patients with rare diseases…
STAT+: U.K. agency declines to recommend Eli Lilly’s Mounjaro for type 2 diabetes
LONDON — A U.K. government agency on Tuesday said that it won’t recommend Eli Lilly’s…
STAT+: Fatty liver disease was alleviated by Lilly’s ‘triple-G’ obesity drug in small study
SAN DIEGO — An experimental obesity drug from Eli Lilly significantly reduced liver fat in…
Ozempic 3.0? Lilly’s ‘triple-G’ drug shows biggest weight loss yet in mid-stage trial
SAN DIEGO — Ozempic and Wegovy became weight loss sensations by targeting a single hormone,…
STAT+: Lilly’s obesity pill cuts 15% of weight at highest dose in mid-stage trial
SAN DIEGO – An experimental pill from Eli Lilly led to 14.7% weight loss on…
STAT+: EU poised to reject Amylyx’s ALS drug, as the company vows an appeal
LONDON — European regulators are on the verge of rejecting an ALS drug from Amylyx…
STAT+: After promising early data, gene therapy trial for Huntington’s takes puzzling turn
Last year, after a string of high-profile trial failures, the Huntington’s disease community got positive…
STAT+: Moving past regulatory obstacles, Protagonist Therapeutics looks to pivotal trial for its rare blood disorder therapy
FRANKFURT, Germany — Protagonist Therapeutics has run into some speed bumps as it’s developed its…
STAT+: FDA panel unanimously endorses Eisai’s Alzheimer’s drug
Expert advisers to the Food and Drug Administration on Friday voted unanimously in favor of expanding…
STAT+: Trailing other sickle cell drug makers, Editas still sees opportunity for its CRISPR therapy
FRANKFURT — Even as other cutting-edge genetic therapies for sickle cell disease move closer to…
STAT+: Sen. Cassidy wants to put a stop to FDA delays in drug review process
WASHINGTON – Sen. Bill Cassidy (R-La.) is looking into ways to speed up the Food…
.jpg)
STAT+: ‘We don’t agree’ on drug pricing policy, FDA chief tells biotech leaders
BOSTON — The drug industry has spent the past year speaking against new mechanisms that…
STAT+: After missing vaccine glory in pandemic, GSK looks to dominate other diseases
BOSTON — GSK has been developing vaccines under one corporate guise or another for 140…