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New draft guidance from the FDA on multiple myeloma endpoints reflects the new technology available to assess disease and how patient journeys have changed with better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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With the acquisition, AstraZeneca will gain access to Icosavax’s investigational combination vaccine IVX-A12, which is being developed for respiratory syncytial virus and human metapneumovirus in older adults.
Bristol Myers Squibb has bought the rights to develop and commercialize SystImmune’s potentially first-in-class, bi-specific antibody-drug conjugate for lung and breast cancer as well as other solid tumors.
Following the regulator’s administrative complaint and threat of a lawsuit in federal court, Sanofi has decided to terminate its licensing deal with Maze Therapeutics to avoid a long litigation process.
The trial demonstrated that C3G glomerulopathy patients treated with the oral factor B inhibitor saw a significant reduction in protein in their urine. The results come days after iptacopan won the FDA’s green light for a rare blood disease.
The company’s fiscal report for 2023 details revenue losses of $539 million and layoffs of 1,100 employees but notes that future GLP-1 manufacturing revenues could help stabilize its finances.
The New York-based pharma reported at ASH that odronextamab had an 80% objective response rate, with 73% of patients experiencing a complete response.
Patients treated with the investigational sickle cell therapy saw an increase in hemoglobin levels and improved red blood cell parameters.
The investigational allogeneic CAR-T therapy P-BCMA-ALLO1 appears to be more effective following strong immunosuppressive preconditioning.
Friday’s FDA approval of Vertex-CRISPR’s Casgevy and bluebird bio’s Lyfgenia has immediately revealed startling differences between these two gene therapies: price and a black-box warning.
Along with CRISPR/Cas9-based Casgevy—developed by Vertex Pharmaceuticals and CRISPR Therapeutics—the regulator on Friday approved bluebird bio’s Lyfgenia, a second gene therapy for sickle cell disease.