News
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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The PD-1 inhibitor Keytruda significantly improved overall survival in a late-stage trial when used with chemoradiotherapy to treat patients with newly diagnosed advanced cervical cancer.
The FDA approved Bristol Myers Squibb’s Breyanzi for chronic lymphocytic leukemia and small lymphocytic leukemia prior to Friday’s adcomm for the company’s other CAR-T therapy, Abecma.
Despite skepticism from FDA reviewers, the Oncologic Drugs Advisory Committee on Thursday strongly supported Geron’s imetelstat for the treatment of anemia in patients with lower-risk myelodysplastic syndromes.
After several delays, BeiGene on Thursday finally secured the FDA’s approval for its PD-1 inhibitor Tevimbra for the treatment of unresectable or metastatic esophageal squamous cell carcinoma.
If you’re confused by the NASH versus MASH indication, you’re not alone.
Madrigal Pharmaceuticals’ Rezdiffra (resmetirom) is the first-ever approved therapy for metabolic dysfunction-associated steatohepatitis—a decision experts say could signal a sea change in treatment of the disease.
The U.K. National Institute for Health and Care Excellence on Thursday recommended against funding Vertex Pharmaceuticals’ CRISPR-based sickle cell disease therapy Casgevy unless uncertainties can be cleared up.
This episode focuses on a healthy discussion regarding the IRA, particularly the unintended consequences to small molecule development within the industry and for patients.
At the center of the deal is Amolyt Pharma’s late-stage candidate eneboparatide for the rare disease hypoparathyroidism. AstraZeneca also gains ownership of AZP-3813, which is being assessed for acromegaly in a Phase I trial.
A lawsuit filed by the Pharmaceutical Research and Manufacturers of America failed to block an Arkansas law that empowers hospitals to use outside pharmacies to dispense discounted drugs.