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What’s in Today’s Brief? (February 14th Preview)
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FDA rejects Disc’s porphyria drug — biomarker link questioned
The FDA rejected Disc Medicine’s experimental porphyria therapy bitopertin, citing uncertainties about the blood-based biomarker used as the efficacy endpoint. The agency said the surrogate marker’s correlation with clinical benefit remained unclear, blocking a pathway that had been accelerated under a newly launched commissioner-level priority review program. Disc, which had been an early beneficiary of the administration’s voucher initiative, saw its shares plunge after the decision. The rejection underscores regulatory skepticism about biomarker-driven approvals and raises questions about the practical value of the new expedited-review vouchers. Clinicians and investors will watch for whether Disc can supply additional clinical outcome data or pursue another regulatory strategy.
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FDA refuse-to-file blindsides Moderna — flu program under scrutiny
The FDA issued a refuse-to-file letter for Moderna’s mRNA influenza vaccine, asserting concerns about trial design and the choice of comparator, and declined to proceed with a formal review. Moderna said the decision was unexpected given prior communications, while company leaders have publicly described the letter as coming “out of the blue.” Agency officials including the vaccine review office have pointed to comparator choice and supporting analysis as unresolved issues. The action has rippled across the vaccine ecosystem: executives, investors and policy observers note growing regulatory uncertainty for respiratory vaccine programs and are recalibrating late-stage investments and development timelines accordingly.
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Infigratinib phase III clears bar — BridgeBio adds oral option for achondroplasia
BridgeBio reported positive top-line results from its global phase III Propel 3 trial of oral infigratinib in children with achondroplasia, meeting growth and body-composition endpoints. The drug, an FGFR3 inhibitor, produced growth outcomes that matched the marketed therapy Voxzogo and offers an oral alternative to existing injectable options. BridgeBio plans regulatory submissions later this year and positions infigratinib as a differentiated, oral FGFR3-targeting therapy. The readout adds competitive pressure in the achondroplasia market and could influence payer and prescriber choices where route of administration matters for pediatric adherence and long-term management.
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All of Us reaches 1 million participants — diversity milestone for precision medicine
NIH’s All of Us research program has reached its target of collecting genetic and health-related data from one million Americans, the agency announced. The dataset aims to represent the country's demographic diversity, enabling broader population-level analyses and more equitable biomarker and drug-discovery research. Researchers and industry partners will use the cohort to support translational studies, real-world evidence generation, and diverse cohort recruitment for clinical trials. The milestone removes a long-standing sample-size barrier for many precision-medicine applications and could accelerate discovery of population-specific biomarkers and therapeutic targets.
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Lyell launches head‑to‑head CAR‑T trial — first direct test against marketed products
Cell therapy developer Lyell announced the start of a first‑of‑its‑kind trial that will test its experimental CAR‑T product directly against marketed CAR‑T therapies. The randomized design pits Lyell’s engineered T‑cell approach against standard-of-care cell therapies to measure comparative efficacy and safety. The trial could set a precedent for next‑generation cell‑therapy development by moving beyond single‑arm pivotal designs toward randomized comparisons. Investors and competitors will track enrollment, endpoints and any signals that differentiate manufacturing, durability, or toxicity profiles.
...and 5 more selected Biotech stories in today’s full edition — or archive.
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