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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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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 status could support staged transitions to new manufacturing processes, potentially mitigating some risks of high-stakes switches.
What China is accomplishing in R&D “has implications for everyone playing in the R&D or innovation world,” McKinsey’s Fangning Zhang says.
The agency’s sweeping rollout and staff challenge underscore rising momentum behind agentic AI: advanced, multiagent systems now fueling early pilots in medical writing, patient engagement and regulatory workflows across the industry.
Although still in Phase I, Wave Life Sciences’ injectable RNA weight loss treatment achieved results that impressed analysts, with 4% fat reduction after three months, beating Novo Nordisk’s semaglutide at a similar time point.
The 2025 meeting of the American Society of Hematology features some of the newest developments in blood cancers and rare diseases.
Dyne’s exon-skipping therapy zeleciment rostudirsen resulted in an approximately sevenfold increase in dystrophin levels at six months and elicited functional improvements that are the “best ever” for this treatment class, Stifel analysts said.
GSK and Ideaya first linked up in 2020 to advance novel therapies for solid tumors. It is unclear why the pharma terminated the partnership.
Analysts at Jefferies see blockbuster potential in zorevunersen in Dravet syndrome, with sales potentially reaching $1 billion to $4 billion.
The pharmaceutical supply chain and device development have become intricately linked. Harmonizing formulation development with drug delivery device design—and leveraging a single‐vendor ecosystem—can deliver significant time, cost, and regulatory advantages for US‑focused drug products, according to industry experts.
As the FDA unveils a parade of initiatives aimed at accelerating drug development for rare diseases, experts appeal for a consistent approval process that will support and further catalyze momentum.