Pipeline
Sanofi will take venglustat to regulators for Gaucher disease but an application for Fabry disease is less clear after the failure of a Phase III trial.
Despite Sanofi CEO Paul Hudson’s confidence in vaccines, the French pharma has cut at least one mRNA flu shot program.
The deal will help bolster Eli Lilly’s growing hearing loss portfolio, which is anchored by the gene therapy AK-OTOF.
Growing opposition to vaccines in the U.S., driven by recent government policy changes, makes it difficult to see a return on investment in vaccine development, Moderna CEO Stéphane Bancel said this week.
2026 is shaping up to be a pivotal year for rare disease drugmakers, with key approvals, filings and readouts lined up for this year.
Three years after the accelerated approval of its anti-amyloid Alzheimer’s therapy, Biogen—neck and neck in the market with Eli Lilly and its Kisunla offering—is focused on a near-term FDA decision for a subcutaneous induction dose of Leqembi, a presymptomatic readout in 2028 and a clutch of next-generation candidates.
After a period of diversification, Novo Nordisk is returning to its roots by focusing on the 2 billion people with diabetes, obesity or overweight.
Biopharma companies—including AstraZeneca, BioNTech and Agios—peered farther into the future on the second day of JPM, setting both revenue and R&D targets through the end of the decade.
AstraZeneca is relying on several upcoming products to help hit its target of $80 billion in revenue by 2030, including drugs for hypertension, breast cancer and generalized myasthenia gravis, all of which are currently under FDA review.
Jefferies analysts forecast a $1 billion market opportunity for each of Sarepta’s siRNA programs for facioscapulohumeral muscular dystrophy and myotonic dystrophy type 1.
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