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The discovery of a tumor in a patient who received REGENXBIO’s gene therapy for Hurler syndrome prompted the FDA to place a hold on that program along with the company’s Hunter syndrome program, which is awaiting an FDA decision on or before Feb. 8.
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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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The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
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A February executive order on pharmaceutical price transparency does nothing to change the incentives that keep costs opaque. But drug companies and other stakeholders would reap the benefits of such disclosures.
InflaRx’s vilobelimab met the bar for futility in a Phase III trial for the rare skin disease pyoderma gangrenosum.
In a video posted to social media on Tuesday, HHS Secretary Robert F. Kennedy, Jr. claimed that there is a lack of clinical data to support the repeat COVID-19 booster strategy in children.
The layoffs will take effect on July 22 and particularly target those working in sales, data engineering and product management.
Rouzbeh Haghighat was serving on the board of Chinook Therapeutics at the time of the Novartis acquisition and, according to the DOJ’s allegations, used confidential information to conduct insider trading.
HHS Secretary RFK Jr. removes the COVID-19 vaccine recommendation for healthy kids and pregnant women—the latest in a string of changes to vaccine policies; judge issues an order to halt HHS’ reorganization and mass layoff plans; Rocket Pharmaceuticals’ pivotal Danon disease trial is on hold after a patient death; and President Trump has named Mehmet Oz to spearhead his Most Favored Nation drug pricing policy.
Nearly two years in with Zurzuvae, Biogen tackles an ‘all of the above market’ to find patients and battle stigma in postpartum depression.
R&D spending across the global pharmaceutical sector climbed 1.5% in 2024, according to unreleased data from Evaluate Pharma.
The acquisition of SiteOne provides a bit of diversification for Lilly, which has burrowed into the obesity and diabetes space with mega-blockbuster tirzepatide and several follow-on molecules.
In addition to a $140 million series D, GRIN Therapeutics has signed a global licensing deal for the epilepsy disorder drug radiprodil worth $50 million upfront.