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Saol Therapeutics received a complete response letter for its pyruvate dehydrogenase complex deficiency treatment a week after the FDA unveiled its Rare Disease Evidence Principles program. On Dec. 18, in a Type A meeting, the biotech will attempt to convince the agency that its drug fits perfectly into the framework.
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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.
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.
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Nearly one-third of biotech and pharma professionals are thinking about leaving the U.S. to find biopharma jobs, according to a BioSpace LinkedIn poll. Career coaches discuss why people are considering relocating and what they should do before heading for the exit.
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Novo Nordisk, under new CEO Maziar Mike Doustdar, has a new attitude. It’s making Pfizer livid.
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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.
The R&D pipeline for depression therapies faced a demoralizing 2025 as five high-profile candidates, including KOR antagonists by Johnson & Johnson and Neumora Therapeutics, flunked late-stage clinical trials, underscoring the persistent challenges of CNS drug development.
Innovative outcome measures coupled with a focus on patient-centered clinical differentiation can help the biopharma industry make meaningful progress in the highly complex area of neuroscience.
Commissioner Marty Makary said that the FDA will soon start requiring only one pivotal trial, instead of two, for companies seeking approval for new drugs.
Hypersensitivity reactions in a mouse model prompted the agency to suspend Denali’s planned Phase I development for DNL952 for Pompe disease.
Praxis Precision Medicines has also announced a “successful” pre-NDA meeting with the FDA for its essential tremor drug candidate ulixacaltamide, for which an approval application is slated for early 2026.
The ACIP voted 8-3 to recommend delaying the hepatitis B vaccine, commonly given just after birth. The CDC itself has said the shot is safe and effective.