FDA accepts uniQure's Huntington's gene therapy AMT-130 for priority review, May 2027 decision date
The FDA's priority review sets up a decision on what would be the first gene therapy for Huntington's disease, based on a small, externally controlled dataset.
HDBuzz · +15 outlets
Washington state resident dies of bird flu after cattle exposure, first US death this year
The death, the third US bird flu fatality linked to the ongoing H5N1 outbreak in dairy cattle, renews scrutiny of the virus's pandemic risk.
STAT · +14 outlets
FDA issues final guidance on cell and gene therapy potency testing
The finalized potency guidance aims to standardize quality and testing standards for cell and gene therapies, a long-sought regulatory clarity for developers.
Oncodaily · +10 outlets
Medicare Part D premiums to spike 50% on average in 2027, KFF finds
The steep premium increase and wide plan-to-plan variance put pressure on seniors during open enrollment and on policymakers debating drug pricing.
Biopharmadive · +8 outlets
FDA grants tentative approval to Dr. Reddy's generic version of HIV prevention shot lenacapavir
The generic approval is intended to widen access to the twice-yearly HIV prevention drug in dozens of lower-income countries.
STAT · +10 outlets
Child dies of measles in northern New York, state's first such death in 30 years
The death underscores resurgent measles transmission amid falling vaccination rates in parts of the US.
STAT · +14 outlets
'Three-parent IVF' mitochondrial replacement under scrutiny as studies raise safety doubts
STAT reporting finds the technique is marketed as a fertility treatment for older women despite limited evidence it works, raising ethical and safety concerns.
STAT · +3 outlets
Karyopharm to sell itself in bankruptcy; Gamida Cell parent Ayrmid is lead bidder
The court-supervised auction follows a forbearance deal on debt and marks a further consolidation among cash-strapped cell therapy developers.
Biopharmadive · +2 outlets
Keytruda biosimilars approach US market but face an uphill battle
Merck's blockbuster faces a patent cliff, yet pharmacy benefit managers and contracting dynamics may limit biosimilar uptake.
Biopharmadive · +3 outlets
Inovio shares surge after FDA approval of Voceryq
The approval gives Inovio its first US-cleared product and a stock rally after years of clinical and financial setbacks.
The Pharma Letter · +14 outlets
Compass Pathways and nonprofit rival compete to win first psychedelic approval
The race to secure the first FDA-approved psychedelic therapy is tightening, with Compass facing unexpected competition.
STAT · +3 outlets
FDA places partial hold on Bright Minds seizure trial over mouse toxicity
The clinical hold delays a key program for Bright Minds and reflects FDA caution following preclinical toxicity signals.
Fierce Biotech · +7 outlets
mRNA vaccine advances renew interest in cancer applications
Reports on patients who defied lethal cancer and a CEO's caution that personalized vaccines are experimental frame the state of the field.
NPR · +12 outlets
Kymera eczema readout looms as high-stakes test of a would-be Dupixent rival
Mid-stage results for Kymera's experimental therapy are closely watched as a gauge of the immunology pipeline's competitive depth.
STAT
AstraZeneca to restructure, Alnylam shares slide and Regeneron posts record quarter in Q3 earnings
The earnings roundup captures diverging fortunes among large biopharmas as cost cuts and pipeline worries shape investor sentiment.
Biopharmadive · +2 outlets
Hyderabad biotech builds personalized mRNA cancer vaccine operation
The Indian RNA startup is developing personalized cancer vaccines, a field where PopVax's CEO cautions the approach remains experimental, not a cure.
NDTV · +3 outlets
BridgeBio prepares rare muscular dystrophy launch with FDA approval in sight
A near-term FDA decision would give BridgeBio a second commercial product and expand options in a rare disease with limited treatments.
BioSpace · +1 outlets
Four drugmakers allegedly paid tens of millions to Russian middlemen to supply medicines
The reported payments raise compliance and sanctions questions about pharmaceutical supply chains into Russia.
STAT · +1 outlets