FDA accepts uniQure's AMT-130 Huntington's gene therapy BLA for priority review, decision due May 2027 with adcomm planned
The FDA granted priority review and set a May 1, 2027 PDUFA date, and appears likely to convene an advisory committee for the first Huntington's disease gene th
Biopharmadive · +15 outlets
Democrats win Senate control, gaining power to push back on Trump health care agenda
Democratic control of the Senate reshapes the outlook for health policy, Medicare Advantage, and insurance legislation heading into the next session.
STAT · +14 outlets
Inovio's DNA immunotherapy Voceryq wins FDA approval for rare HPV-linked airway disease
The accelerated approval makes Voceryq the first DNA-based immunotherapy cleared for recurrent respiratory papillomatosis, sending Inovio shares sharply higher.
Fierce Pharma · +7 outlets
Servier's $1.55B muscular dystrophy drug sevasemten fails first major trial
The setback hits Edgewise's Becker muscular dystrophy program, a condition with no approved treatments and no clear path forward in the study.
STAT · +2 outlets
BioNTech beats Q3 estimates but defends mRNA cancer strategy after trial misses
Executives kept mRNA cancer therapies central to the pipeline while acknowledging a missed endpoint and mixed lung and breast cancer signals.
GenomeWeb · +5 outlets
Karyopharm to sell itself in bankruptcy, with Gamida Cell parent as lead bidder
The debt-laden cancer drug developer will hold a court-supervised auction after striking a forbearance deal with creditors.
Biopharmadive · +1 outlets
Child dies of measles in New York, state's first such death in 30 years
The death in rural northern New York underscores gaps in vaccination coverage amid national measles resurgence.
STAT · +14 outlets
Compass Pathways set to file psilocybin depression drug for approval imminently
The filing would put Compass in a tight race to win the first US approval of a psychedelic medicine, against a nonprofit competitor.
BioSpace · +5 outlets
Person in Washington state dies of bird flu after exposure to infected cattle, first US death in a year
The third US bird flu fatality overall highlights ongoing H5N1 spillover risk from dairy herds to farm workers.
STAT · +13 outlets
FDA places partial clinical hold on Bright Minds' BMB-101 Phase 2 DEE seizure trial
The hold, tied to toxicity seen in mice, halts development of the epilepsy candidate and cut Bright Minds' share price in half.
Biopharmadive · +13 outlets
Lilly's Jaypirca approved by FDA for first-line CLL/SLL treatment
The label expansion moves the BTK inhibitor earlier in the treatment sequence for chronic lymphocytic leukemia and small lymphocytic lymphoma patients.
scanx.trade · +14 outlets
BioNTech, Moderna mRNA cancer vaccine race intensifies as Moderna stock surges on Keytruda patent cliff
Personalized mRNA cancer vaccine progress is colliding with upcoming biosimilar pressure on Merck's Keytruda, reshaping oncology competition.
BigGo Finance · +7 outlets
Personalised melanoma vaccine cuts risk of recurrence
The result adds to evidence that tailored mRNA vaccines can reduce relapse in high-risk melanoma patients.
Lab + Life Scientist · +14 outlets
FDA approves Bayer's Kerendia to slow chronic kidney disease in type 1 diabetes
The approval extends the nonsteroidal MRA's use to a patient group with few kidney-protective options.
scanx.trade · +14 outlets
FDA finalizes cell and gene therapy potency guidance
The final guidance aims to standardize potency testing for cell and gene therapies, a persistent source of regulatory friction for developers.
RAPS · +6 outlets
Medicare Part D premiums to spike 50% on average in 2027, KFF finds
The think tank urged seniors to compare plans during open enrollment given wide variation in costs across Part D offerings.
Biopharmadive · +13 outlets
STAT+: Psilocybin's lasting benefits prove elusive in mice, multi-lab study finds
Five Bay Area labs compared psilocybin's durable effects on anxiety and depression-like behavior in mice with limited results, raising questions for clinical cl
STAT
FDA OKs generic HIV prevention shot, widening access for lower-income countries
Tentative approval for Dr. Reddy's generic version of lenacapavir is a key step toward expanding global access to the prevention drug.
STAT · +4 outlets