FDA grants priority review to uniQure's AMT-130 for Huntington's disease, sets May 2027 decision date
The FDA accepted uniQure's BLA for its Huntington's disease gene therapy and scheduled a May 1, 2027 PDUFA date, with an advisory committee expected.
Biopharmadive · +15 outlets
Democrats win control of Congress, putting Medicare for All backers in charge of key health committees
Democratic gains in the midterms hand the party power to push back on the Trump administration's health care agenda and set committee leadership.
STAT · +9 outlets
Analysts say biopharma stocks likely to climb after Democratic 'blue wave'
Historical data and analysts suggest the sector outperforms after midterms, with Democrats focused on priorities other than drug pricing.
Biopharmadive · +1 outlets
Former Madrigal Pharmaceuticals executive sues, alleging coverup of drug safety issues
The federal lawsuit claims the company concealed safety concerns tied to its drug Rezdiffra.
STAT
Ultragenyx to cut about 20% of staff after another rare disease study fails
The layoffs come as the company ends development of two rare disease drugs following a failed trial.
STAT · +4 outlets
Servier's $1.55 billion Becker muscular dystrophy drug fails first major trial
The setback hits a form of muscular dystrophy that currently lacks approved treatments.
STAT · +7 outlets
Child dies of measles in New York, state's first such death in 30 years
The death in rural northern New York highlights resurgent measles transmission amid slipping vaccination rates.
STAT · +14 outlets
FDA advisers narrowly vote to add six peptides to drug compounding list
The close advisory vote could affect availability of compounded peptide therapies, with regulators weighing next steps.
Health Affairs · +6 outlets
Compass Pathways to file psilocybin depression drug for approval imminently after stock swings
Compass has been seen as a frontrunner to win the first psychedelic drug approval, though a nonprofit rival may make the race tight.
BioSpace · +6 outlets
Karyopharm, deep in debt, to sell itself through bankruptcy auction
Gamida Cell's parent will serve as lead bidder in a court-supervised auction after Karyopharm negotiated a forbearance deal on debt payments.
Biopharmadive · +2 outlets
Medicare Part D premiums to spike 50% on average next year, KFF finds
The think tank urged seniors to shop carefully during open enrollment given wide variation in plan costs.
Biopharmadive · +7 outlets
Vertex executives defend IgA nephropathy kidney drug ahead of FDA decision
On an earnings call, Vertex expressed conviction in the coming launch of povetacicept, viewed as important to its future.
Biopharmadive · +6 outlets
First US bird flu death of the year reported in Washington state after cattle exposure
It is the first such death this year in the US and the third linked to the ongoing outbreak.
STAT · +13 outlets
FDA finalizes cell and gene therapy potency guidance
The guidance sets expectations for potency testing in cell and gene therapy development.
RAPS · +6 outlets
CVS plans to split up, as Trump administration surveys pharma supply chain vulnerabilities
The administration will survey key pharmaceutical production components to identify domestic supply chain gaps.
STAT · +8 outlets
Clinical trial investigators write to FDA urging approval of Capricor's deramiocel
The letter from trial investigators precedes a regulatory decision on the Duchenne muscular dystrophy therapy.
Yahoo Finance · +5 outlets
FDA grants tentative approval to Dr. Reddy's for generic HIV prevention shot
The approval covers a generic version of a groundbreaking HIV prevention medicine, alongside Lonza's purchase of US facilities.
STAT · +1 outlets
Lame duck session to shape health funding as Congress faces government deadline
Before Democrats take control, Congress must fund expiring health care programs and confirm HHS nominees.
STAT · +1 outlets