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New draft guidance from the FDA on multiple myeloma endpoints reflects the new technology available to assess disease and how patient journeys have changed with better treatments.
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With the biopharma industry performing better of late, analysts, executives and other industry watchers are “cautiously optimistic”—a term heard all over the streets of San Francisco at the J.P. Morgan Healthcare Conference earlier this month.
Bristol Myers Squibb, GSK and Merck are contributing drug ingredients as part of their deals with the White House but are keeping many of the terms of their agreements private.
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
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Phacilitate’s annual event dawns as cell and gene therapies reach a new tipping point: the science has hit new heights just as regulatory and government policies spark momentum and frustration.
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Analysts said the deal with Novo was likely giving Hims “‘credibility’ or increased consumer traffic,” adding that the “litigation risk is back on the table” now that the Danish pharma has stepped away.
The industry sector focused on aging is only about 10 years old, but acting on what scientists already know, a new crop of biotechs, backed by investors, are taking a disease-centric approach to extending the human lifespan.
BrainStorm Cell Therapeutics issued a statement Tuesday supporting a Citizens’ Petition submitted to the FDA requesting the approval of its cell therapy NurOwn, whose BLA was withdrawn in 2023. A Phase IIIb trial was scheduled to begin last month.
The deal marks an end for CAR T company Cargo Therapeutics, which has been slashing its workforce and cutting programs since the January decision to halt its lead candidate for a certain type of aggressive large B cell lymphoma.
President Donald Trump’s One Big Beautiful Bill, signed into law last week, reintroduces broader exemptions for orphan drugs from the IRA’s drug price negotiation program—a move welcomed by the biopharma industry. The new tax law also cuts Medicaid funding, posing a minimal risk to pharma’s bottomlines and potentially jeopardizing hospitals’ 340B status. It does not, however, include new rules for pharmacy benefit managers that had been in an earlier draft.
After issues with a batch of Jasper Therapeutics’ investigational antibody led to “lower” therapeutic effects in several patients, analysts at BMO Capital Markets said they “believe investors won’t feel comfortable coming back to the story.”
Societies, including the American Academy of Pediatrics, allege that Kennedy’s directive to remove COVID-19 from vaccination guidelines for healthy pregnant women and healthy children puts these vulnerable groups at risk of serious illness.
A readout from the company’s SUMMIT trial put its small molecule bezuclastinib on a collision course with rival Blueprint’s Ayvakit, which Leerink analysts said does not sufficiently treat all patients.
The partnership will give Chugai access to Gero’s artificial intelligence technology to discover novel targets in aging-related diseases. Chugai will then develop antibody-based drugs based on the findings.
After a season of regulatory upheaval, obesity and rare genetic diseases will likely remain major themes for biopharma in 2025, according to Jefferies.