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Moderna will not commit to previous 2028 breakeven guidance as the ripple effects of the FDA’s refusal-to-file decision spread through its pipeline.
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The rare disease drugmaker is facing potential competitors for achondroplasia drug Voxzogo. Is a big M&A deal with two approved assets enough to maintain investor interest?
The FDA issued a rare Refusal-to-File letter to Moderna over its mRNA-based influenza vaccine application, in an unusual move that sent the biotech’s shares tumbling.
A rapturous response to data published last year for Pelage’s hair loss candidate overwhelmed the biotech. Now, the company is ready to show the world the science behind the breakthrough.
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Novo Nordisk and Eli Lilly have been battling head-to-head in an exploding obesity market. They should never have been compared apples to apples.
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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.
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.