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FDA doubles down on sham‑control for uniQure’s Huntington’s gene therapy — Tensions escalate
A senior FDA official publicly defended reviewers’ demand that uniQure run a randomized, sham‑controlled trial for its surgically delivered Huntington’s gene therapy AMT‑130, rejecting the...
Zealand’s amylin fails to match Lilly’s benchmark — Market punishes shares
Roche‑partnered petrelintide produced up to 10.7% mean weight loss at 42 weeks in a Phase II study but missed investor expectations for placebo‑corrected efficacy and was judged inferior in...
FDA grants fast approval to J&J two‑drug myeloma combo — National priority pathway used
The FDA issued a rapid approval for Johnson & Johnson’s Tecvayli plus Darzalex regimen in relapsed or refractory multiple myeloma under the Commissioner’s National Priority Voucher (CNPV) pilot,...
Kestrel clears IND for oral pan‑KRAS inhibitor — First‑in‑human trial planned
Kestrel Therapeutics obtained FDA clearance to begin a first‑in‑human Phase I trial of KST‑6051, an oral, pan‑KRAS small‑molecule intended for KRAS‑driven cancers. The company expects to start the...
Illumina expands pharma genomics with proteomics add‑on — 50K genomes to gain paired proteome
Illumina announced it will add 50,000 genomes with matched proteomics to the Alliance for Genomic Discovery dataset, expanding the pharmaceutical‑accessible resource for target discovery and...
Lonza sells capsule and health‑ingredients unit for $2.2B — Portfolio reshaped
Lonza agreed to divest its capsules and health‑ingredients business to a UK investment firm for $2.2 billion upfront, executing a strategic separation of its consumer health manufacturing assets....
PepGen faces partial FDA hold on DM1 trial — US sites paused, global program continues
PepGen reported that the FDA placed a partial clinical hold on the Freedom2‑DM1 Phase II multiple‑ascending‑dose trial of PGN‑EDODM1 in myotonic dystrophy type 1, citing concerns in preclinical...
Merck KGaA trims oncology pipeline after SpringWorks buyout — Two programs cut
Merck KGaA removed several clinical‑stage oncology assets acquired via its SpringWorks purchase, including a Hippo pathway inhibitor (SW‑682) and a PARP1 inhibitor (M9466), after a strategic...
Servier buys Day One for $2.5B — pediatric glioma drug in hand
Servier agreed to acquire Day One Biopharmaceuticals for about $2.5 billion in cash, securing Ojemda (tovorafenib), an FDA-approved therapy for pediatric low-grade glioma, and access to Day One’s...
FDA’s Vinay Prasad to leave in April — tenure marked by controversy
Vinay Prasad, head of the FDA’s Center for Biologics Evaluation and Research (CBER), will depart the agency at the end of April after a turbulent tenure that included stricter review stances on...
FDA doubles down on sham control for uniQure Huntington’s trial — agency rebuffs company
A senior FDA official defended the agency’s decision to require a randomized sham‑controlled trial for uniQure’s AMT‑130 gene therapy for Huntington’s disease, saying reviewers stand by the need...
FDA speeds Tecvayli‑Darzalex approval via priority pilot — myeloma label widened
The U.S. FDA granted rapid approval to Johnson & Johnson’s combo of Tecvayli and Darzalex Faspro for early relapsed or refractory multiple myeloma under the Commissioner’s National Priority...
Zealand’s amylin candidate falls short — ambivalent Phase II readout dents hopes
Zealand Pharma and partner Roche reported Phase II results for petrelintide showing mean weight loss of about 10.7% at 42 weeks versus 1.7% for placebo — a tolerable safety profile but efficacy...
Lilly targets employers to expand GLP‑1 access — insurers bypassed
Eli Lilly unveiled employer‑focused programs designed to broaden access to its GLP‑1 obesity medicines by offering alternative payment channels and subsidies for workforce insurance plans. The...
Japan approves first iPSC‑derived therapy for Parkinson’s — regenerative milestone
Japan’s regulators approved the world’s first therapy derived from induced pluripotent stem cells (iPSCs) to treat Parkinson’s disease, marking a regulatory milestone for regenerative medicine....
Cell therapies for neurodegeneration advance — CAR astrocytes and boosted brain phagocytes
Two preclinical advances illustrated novel cell‑based strategies against neurodegeneration. Researchers engineered astrocytes to express chimeric antigen receptors (CARs) that target and clear...
FDA policy friction grows — industry seeks clarity on single‑trial rule and advisory panels
Contract research organization group ACRO urged the FDA to issue formal guidance and open notice‑and‑comment rulemaking before expanding a new default that a single adequate trial plus...
Genomics meets proteomics and AI imaging clears margins — diagnostics scale up
Illumina announced plans to add 50,000 genomes with paired proteomic data to its Alliance for Genomic Discovery (AGD), expanding multi‑omic datasets available to pharma members and aiming to...
FDA turmoil: Vinay Prasad set to leave CBER in April
Vinay Prasad, director of the FDA’s Center for Biologics Evaluation and Research, will depart the agency at the end of April, officials confirmed. Prasad’s tenure was marked by a string of...
Servier buys Day One for $2.5B – gains pediatric glioma drug
Servier agreed to acquire Day One Biopharmaceuticals for roughly $2.5 billion in cash, a deal that transfers marketed pediatric glioma drug Ojemda (tovorafenib) and Day One’s development programs...