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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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Corcept’s overall survival data “look competitive” with AbbVie’s Elahere and Merck’s blockbuster Keytruda, Truist Securities said Thursday.
Merck had previously offered anywhere from $28 billion to $32 billion to swallow Revolution Medicines.
Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging field.
From opening new therapeutic mechanisms to repairing neuronal damage, investigational molecules from Ventyx Therapeutics, AC Immune, Gain Therapeutics and more could shape the future of Parkinson’s disease treatment.
The mixed data from the Phase III COAST 2 trial follows an underwhelming data drop from COAST 1 in September that Leerink Partners said “fell well below expectations.”
The $1.2 trillion budget package will now move to the Senate, which is expected to hold a vote next week.
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.
Corxel will use the fundraising proceeds to advance the oral GLP-1 therapy CX11 through mid-stage development in the U.S., as well as prepare for its Phase III studies.
Growing opposition to vaccines in the U.S., driven by recent government policy changes, makes it difficult to see a return on investment in vaccine development, Moderna CEO Stéphane Bancel said this week.
“I don’t like established science,” ACIP chairperson Kirk Milhoan said in an interview on the Why Should I Trust You? podcast. “Science is what I observe.”