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BioMarin’s investigational therapy failed to elicit clinical improvements in patients with ENPP1 deficiency, while also missing key secondary endpoints of rickets severity and growth.
FEATURED STORIES
Nusano will bring a massive new radioisotope facility in Salt Lake City online by the end of the year, establishing a supply of starting materials for the next generation of radiopharmaceuticals.
Last month, Revolution Medicines’ RAS inhibitor doubled survival in a Phase 3 pancreatic cancer trial. On the biotech’s heels are Immuneering, Actuate Therapeutics, Erasca and more, looking to improve on that result with increased tolerability—and more time for patients.
The recent approval of Regeneron’s Otarmeni underscores the maturation of gene therapies across a range of diseases. Here, BioSpace reviews genetic medicines in development for the central nervous system, retinal, cardiac and neuromuscular diseases.
FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
The Department of Health and Human Services is spinning its wheels, unable to establish steady leadership at three major divisions—the CDC and the FDA’s two primary review units.
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Keytruda can now be used in the European Union for patients with resectable non-small cell lung cancer at high risk of recurrence in combination with platinum chemotherapy, then continued as a monotherapy afterwards.
April kicks off with three FDA target action dates, including one that could potentially set the stage to move CAR-T therapies into earlier lines of treatment.
After an initial rejection due to safety issues, followed by a dispute and deferred actions, Akebia Therapeutics on Wednesday finally won the FDA’s nod for vadadustat as a treatment for anemia caused by chronic kidney disease.
Continuing 2024’s biotech initial public offering rally, Boundless Bio will debut Thursday on the Nasdaq with the proceeds used to advance its pipeline of extrachromosomal DNA cancer assets.
Citing a JAMA study that found Ozempic could be profitably produced at under $5 per month, Senator Bernie Sanders on Wednesday called on Novo Nordisk to lower prices for the diabetes treatment and the weight-loss drug Wegovy.
Big Pharma’s appetite for safe and effective oral IBD drugs with novel mechanisms of action continues to grow, with my former company just the latest in a string of acquisitions in the space.
Nanoscope Therapeutics nabbed a victory on the changed primary endpoint of its Phase IIb vision loss gene therapy trial Tuesday, teeing the biotech up to file for FDA approval of the candidate this year.
Novocure’s stock spiked more than 15% Wednesday morning after the company announced its Tumor Treating Fields therapy met the primary endpoint in a late-stage trial.
To avoid overextending yourself and harming your work-life balance, how should you set and maintain boundaries at work?
While disease-modifying therapies largely steal the spotlight in Alzheimer’s drug development, several companies are working to solve this less-discussed but disruptive facet of the illness.