FDA accepts uniQure's AMT-130 for priority review, sets May 2027 decision on Huntington's gene therapy
A May 2027 PDUFA date and a likely advisory committee make this the furthest-advanced gene therapy for Huntington's disease, based on a small externally control
Biopharmadive · +15 outlets
FDA grants accelerated approval to Inovio's DNA immunotherapy Voceryq for rare HPV-linked airway disease
The first DNA-based therapy approval marks a shift in the field from vaccines toward therapeutic use in recurrent respiratory papillomatosis.
Fierce Pharma · +12 outlets
Bright Minds' Phase 2 DEE epilepsy trial halted as FDA imposes partial clinical hold on BMB-101
The hold, tied to toxicity seen in mice, cut the biotech's share price roughly in half and delays its lead seizure program.
Dealroom · +8 outlets
Servier's $1.55B muscular dystrophy drug sevasemten fails first major test in Becker MD
The setback removes a potential first treatment for Becker muscular dystrophy, a form with no approved therapies.
STAT · +2 outlets
FDA approves Bayer's Kerendia to slow chronic kidney disease in type 1 diabetes
The label expansion extends the nonsteroidal MRA beyond type 2 diabetes to a broader CKD population.
scanx.trade · +14 outlets
Lilly's Jaypirca wins FDA approval for first-line CLL/SLL treatment
The front-line clearance expands use of the BTK inhibitor into newly diagnosed chronic lymphocytic leukemia patients.
scanx.trade · +6 outlets
Karyopharm, deep in debt, to sell itself in Chapter 11 bankruptcy auction
Gamida Cell's parent is the lead bidder in a court-supervised auction after the company struck a forbearance deal with lenders.
Biopharmadive · +1 outlets
BioNTech beats Q3 estimates, holds to mRNA cancer strategy despite trial misses
The company reported Q3 revenue above estimates and reiterated its commitment to mRNA cancer therapies after a mixed month of data.
GenomeWeb · +5 outlets
Vertex executives defend povetacicept launch outlook ahead of FDA decision in IgA nephropathy
Wall Street views the IgA nephropathy drug as central to Vertex's pipeline, and management used its earnings call to calm investor concerns.
Biopharmadive · +4 outlets
Child dies of measles in New York, the state's first such death in 30 years
The death in rural northern New York underscores slipping vaccination coverage amid national measles outbreaks.
STAT · +14 outlets
Washington state resident dies of bird flu after cattle exposure, first U.S. death this year
The third U.S. H5N1 death overall raises fresh questions about surveillance of the virus in dairy herds.
STAT · +13 outlets
Medicare Part D premiums to jump 50% on average next year, KFF finds
KFF urged seniors to shop carefully during open enrollment given wide cost variance between plans.
Biopharmadive · +10 outlets
FDA finalizes cell and gene therapy potency guidance
The guidance gives developers a standardized framework for potency assays, a long-running bottleneck in CGT approvals.
RAPS · +9 outlets
FDA approves generic lenacapavir HIV prevention shot, widening access for lower-income countries
Tentative approval for Dr. Reddy's generic version of the twice-yearly shot is a key step in broadening global PrEP access.
STAT · +14 outlets
FDA advisers narrowly vote to add six peptides to the drug compounding list
The close vote keeps alive a fight over compounded peptides such as BPC-157 and semaglutide copies.
The American Journal of Managed Care · +7 outlets
CNBC: little-known biotech's cancer bet pays off, sending shares up twofold
An unnamed small-cap biotech doubled after reporting positive data from a high-risk oncology program.
CNBC · +1 outlets
Psilocybin's lasting benefits prove elusive in mice, five-lab study finds
The pooled mouse results come as Compass Pathways and a nonprofit vie to win the first psychedelic approval.
STAT · +1 outlets
CRISPR Therapeutics reports growing Casgevy use ahead of key pipeline data, RBC says
Analysts flagged expanding uptake of the sickle cell gene therapy as investors await readouts from the company's next programs.
Yahoo Finance · +1 outlets