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Facing declining valuations and funding challenges, public biotechs like bluebird bio are going private to restructure, reduce regulatory burdens and refocus on long-term growth.
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The FDA’s proposed Rare Disease Evidence Principles review process is a starting point for getting rare disease therapies across the finish line, but industry leaders say there are more concrete steps the regulator could take to help patients.
With AbbVie’s $1.2 billion acquisition of Gilgamesh Pharmaceuticals’ lead depression drug, the psychedelic therapeutics space has soundly rebounded from Lykos’ rejection last year. There are now seven programs in Phase III trials across the sector, with multiple companies vying for that first approval.
A decade-long journey has come to an end for Stealth BioTherapeutics and the Barth syndrome community with the first-ever treatment for this uncommon mitochondrial disease. CEO Reenie McCarthy called it a “pivotal victory” that “offers hope for expedited regulatory attention to other ultra-rare diseases.”
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Pfizer Inc. and BioNTech SE announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency has recommended marketing authorization for the companies’ Omicron XBB.1.5-adapted monovalent COVID-19 vaccine administered as a single dose for individuals 5 years of age and older, regardless of prior COVID-19 vaccination history.
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The FDA has vowed to fix a pharma ad loophole—but they’re targeting the wrong one.
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GSK, Gilead and Arcellx, Vertex and more present new data at the American Society of Hematology annual meeting just as sickle cell therapies Casgevy and Lyfgenia have a new outcomes-based payment model; Eli Lilly and Novo Nordisk pump new funds into manufacturing; and AbbVie makes a Cerevel comeback while uniQure clears a path toward accelerated approval in Huntington’s disease.
Carisma’s second workforce reduction this year likely leaves the company with 44 full-time employees as turns its focus to developing therapies for fibrosis, oncology and autoimmune diseases.
In a Type B meeting, the FDA signified that it will allow uniQure to use a natural history control, the composite Unified Huntington’s Disease Rating Scale, and neurofilament light chain levels to support the accelerated approval of its gene therapy AMT-130.
The overall survival edge over J&J’s Darzalex will help GSK strengthen its case as it plots the market comeback of Blenrep, which was pulled after a failed confirmatory study.
Anito-cel has shown no signs of delayed neurotoxicity at around 9 months of follow-up, hinting at a safety profile that could set it apart from J&J and Legend’s Carvykti.
According to Jake Van Naarden, president of Lilly Oncology, the excess deaths could be due to the high rate of crossover in BRUIN CLL-321.
Relmada Therapeutics will halt two Phase III trials of a major depressive disorder drug after a futility assessment and explore strategic alternatives including a potential sale to maximize shareholder value.
Tavapadon improved motor and daily living complications at week 26. The news comes nearly one month to the day after AbbVie announced the Phase II failure of another key Cerevel asset, emraclidine.
Analysts called the data “very competitive” but raised questions about safety. Merck gained ownership of the ADC when it acquired VelosBio in November 2020 for $2.75 billion.
The discontinuation of STRIDES is a rare stumble for the next-generation obesity field and comes just weeks after Amgen announced underwhelming mid-stage data for MariTide.