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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.
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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.
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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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Partners Biogen and Sage Therapeutics had sought approval for the therapy in both postpartum depression and major depressive disorder, but the FDA rejected the application for the latter.
Thursday, Gilead Sciences reported lower second-quarter profit due to a drop in sales of the company’s Veklury antiviral and a $525 million legal settlement, while slightly raising its sales outlook for the year.
The market opportunity for pulmonary arterial hypertension drugs stretches well beyond the 500–1,000 Americans diagnosed with the disease each year—and biopharma is taking note.
For the second time, the regulator has blocked Mesoblast’s attempt to have remestemcel-L approved for pediatric steroid-refractory acute graft versus host disease, citing the need for adult data.
Following the FDA’s approval last month, a Centers for Disease Control and Prevention panel has recommended AstraZeneca’s and Sanofi’s Beyfortus for immunizing infants and high-risk young children.
The company reported mid-stage and late-stage trial victories, one in small-cell lung cancer and another in colorectal cancer, as part of its second-quarter earnings on Thursday. No specific data was provided.
A Louisiana woman has filed a lawsuit alleging that Novo Nordisk’s Ozempic and Eli Lilly’s Mounjaro did not have warnings about the risk of severe gastrointestinal problems.
During Regeneron’s second-quarter earnings call Thursday, the company said that the regulator could make its decision on 8-mg Eylea in the third quarter—much earlier than market expectations.
The biotech Thursday reported that second-quarter sales of its COVID shot dropped 94%. However, it expects $6 billion to $8 billion in revenue this year, up from a previous forecast of $5 billion.
The regulator Wednesday approved expanding Lonsurf’s label for previously treated metastatic colorectal cancer, authorizing a combination regimen with Roche’s bevacizumab.