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The cancers were diagnosed 19 to 92 months after Skysona treatment.
After extending its review period to evaluate additional submissions, the FDA ultimately denied Applied Therapeutics’ govorestat for galactosemia, citing “deficiencies” with the application. The biotech plans to meet with the regulator to discuss the best way forward for the drug.
At the conference, AstraZeneca and Daiichi Sankyo will present their case for Dato-DXd in NSCLC, while BioNTech and Merus will reveal promising mid-stage data for their respective cancer candidates.
Despite hotly debated biomarkers and failed or delayed confirmatory trials, the accelerated approval program has a track record of propelling R&D for some of medicine’s most challenging illnesses.
Emboldened by technological advances and a deeper knowledge of glioblastoma, Merck, Kazia Therapeutics, CorriXR Therapeutics and others are targeting the often-fatal brain tumor.
Projected to be worth over $38 billion in the global healthcare market by 2032, AI simulations have the potential to streamline clinical trials and help address inequities in underserved patient populations.
Eli Lilly topped the list of the 20 biggest pharmas by market cap with a more than 39% improvement year-to-date in its share price. Other companies have not been so lucky.
Despite the “unfortunate” failure, William Blair analysts do not believe that the utreloxastat readout will heavily affect PTC, instead postulating that the upcoming FDA decision on its phenylketonuria candidate sepiapterin will be a stronger driver of the biotech’s stock.
Trump is rounding out his health cabinet with another controversial figure: one of the authors of the Great Barrington Declaration, which advocated for herd immunity through infection during the COVID-19 pandemic.
BridgeBio’s Attruby wins approval for transthyretin amyloid cardiomyopathy while the FDA accepts Alnylam’s application for Amvuttra in the indication; Cassava’s controversial Alzheimer’s drug flunks Phase III; Amgen’s MariTide fails to impress investors, Donald Trump’s controversial nominations continue.
With Elevidys expansion in hand, Sarepta commits up to $10 billion to develop short interfering RNA–based drugs to build out its pipeline.
With Amgen’s MariTide results at the lower end of investors’ expectation of 20% to 25% weight loss, the much-anticipated readout sent the company’s shares tumbling.
Currently, Eli Lilly’s GLP-1 drugs Mounjaro and Zepbound (tirzepatide) are not on the FDA’s shortage list but compounded pharmacies are still making them. That’s unprecedented.
Alector is kicking off a resource realignment effort that will include a workforce reduction. The biotech expects its current cash position to last it through 2026.
Truqap’s positive clinical data comes after it failed a late-stage study in metastatic triple-negative breast cancer. It helps AstraZeneca position itself as a top player in the prostate cancer space, alongside its Big Pharma colleagues.
While taldefgrobep alfa failed to show improved motor function in spinal muscular atrophy, treated patients saw a marked reduction in body fat. Biohaven plans to launch a Phase II trial in obesity by the end of the year.
Cassava Sciences has revealed the late-stage clinical failure of controversial Alzheimer’s drug simulfilam. The company had pledged to share the results whether “good, bad or ambiguous.”
The FDA has accepted Alnylam’s supplemental New Drug Application for Amvuttra on the heels of BridgeBio’s Attruby nod, potentially giving Pfizer’s tafamidis franchise another competitor.
Adcendo joins a growing group of biotechs working to usher the next generation of antibody-drug conjugates to the market.
Career conservative and former congressman Dave Weldon will, if confirmed, act as director of the Centers of Disease Control and Prevention, where his anti-vaccine views will mesh with those of selected Department of Health and Human Services head Robert F. Kennedy Jr.