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Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like MPS II moving into the spotlight.
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After advancing in lockstep through the pandemic, the fortunes of the biotechs have diverged as their use of COVID-19 windfalls has taken shape.
After suffering in the wake of expired tax incentives for pharmas, the island is trying to take advantage of geopolitics to grow its drug manufacturing sector.
AstraZeneca’s $15 billion pledge to its China operations highlights the country’s advantages. But other regions are also hoping to host more clinical studies.
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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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The Federal Trade Commission seeks to codify its heavier-handed approach, already displayed by its actions regarding Amgen’s acquisition of Horizon, among other deals.
Ulotaront failed to meet primary endpoints in two studies. The companies said high COVID-19 placebo effect “may have masked molecule’s therapeutic effect” and plan to discuss next steps with the FDA.
Amid U.S. shortages in crucial drugs, House Republicans have filed their own draft bill that seeks to increase the accountability of the FDA and provide more flexibility for generics manufacturers.
Biogen’s $7.3 billion Reata acquisition and layoffs dominated this week’s news, while BMS and Roche reported second-quarter earnings and BioSpace looked at 12 late-stage neuro companies.
Following a sweeping cost-cutting plan announced earlier this week, including an 11% workforce reduction, Biogen is acquiring Reata Pharmaceuticals to bolster its neurological and rare disease pipeline.
The British drugmaker’s rare disease subsidiary Alexion will take on a number of Pfizer’s preclinical gene therapy programs and technologies to advance its genomic medicines pipeline.
Despite beating analyst expectations, Takeda sustains a mid-stage defeat in a rare genetic disorder, leaving the fate of an investigational enzyme replacement therapy uncertain.
Two late-stage trial wins for Merck’s V116 investigational pneumococcal vaccine could make it a serious challenger to Pfizer’s Prevnar 20 in the space.
Data from two late-stage studies show that participants on Eli Lilly’s tirzepatide, which targets both GIP and GLP-1 receptors, achieved up to 26.6% mean weight loss.
The FDA has three upcoming action dates in the next two weeks, including a highly anticipated decision regarding Biogen and Sage’s depression drug zuranolone.