Pharma News and Headlines
Pharma News and Headlines
The EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended six new medicines, including three orphan drugs already approved by the FDA—Ojemda for pediatric low‑grade glioma, Palsonify for acromegaly, and Xolremdi for WHIM syndrome. The agency also gave a positive opinion to Moderna’s combined flu‑COVID‑19 mRNA vaccine, diverging from the FDA’s recent refusal to file the standalone flu candidate. Additionally, CHMP rejected two therapies that the FDA had previously declined, underscoring a regulatory split between Europe and the United States.
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![[Review] Autosomal Dominant Polycystic Kidney Disease](/cdn-cgi/image/width=1200,quality=75,format=auto,fit=cover/https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/e5c772f073be33a83118f46a1ac91f13.webp)
Autosomal dominant polycystic kidney disease (ADPKD) remains the most common hereditary cause of chronic kidney disease, imposing substantial morbidity, mortality, and healthcare costs worldwide. Recent advances in molecular genetics and high‑resolution imaging have sharpened diagnostic criteria and enabled more accurate...
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Human pluripotent stem cells (hPSCs) are emerging as a transformative platform for Parkinson’s disease, with recent phase I/II trials showing successful engraftment of hESC‑ and iPSC‑derived dopaminergic neurons. Yet extensive data reveal that cultured hPSCs frequently acquire recurrent genetic lesions—most...
Minnesota’s Department of Health reports that hospitals and clinics in the state earned at least $1.34 billion in 2024 from the 340B drug discount program. Participants received $3.045 billion in discounted medicines but paid $1.53 billion plus $165 million in administration fees. The largest...
Ultrasonic spray coating is emerging as a core engineering discipline for drug‑eluting implants, with parameters such as drug‑to‑polymer ratio, nitrogen carrier‑gas flow, nozzle height, and spray power dictating coating thickness, profile, and elution behavior. The article explains how variations in...
Researchers at Cornell’s Weill Institute introduced QUASARR‑seq, a high‑throughput assay that measures promoter and enhancer activity simultaneously. The study found that most human regulatory elements can function as both promoters and enhancers, following a unified regulatory logic. A bidirectional feedback...
The iShares U.S. Pharmaceuticals ETF (IHE) is anchored by Johnson & Johnson and Eli Lilly, whose earnings per share have nearly doubled in the past year. IHE’s aggregate one‑year EPS growth estimate is an impressive 19.07% while trading at a modest...
President Trump’s "most‑favored nation" (MFN) drug pricing agreements, touted as a safeguard against excessive prescription costs, have been revealed to run for three years for several participants. SEC filings show that 16 pharmaceutical firms have entered these deals, each with...
Researchers have engineered size‑shifting lipid nanoparticles that grow from ~100 nm to >300 nm after intraperitoneal injection, exploiting a capsule‑filter mechanism that blocks entry into the liver and spleen while allowing passage to the pancreas. The enlarged particles deliver mRNA payloads—including CRISPR‑Cas9...
Eli Lilly unveiled LillyPod, a DGX SuperPOD built with 1,016 NVIDIA Blackwell Ultra GPUs delivering more than 9,000 petaflops of AI performance. The system powers genomics, protein‑diffusion, small‑molecule graph neural networks and foundation models, allowing billions of in‑silico experiments. Constructed...
Bayer's HYRNUO (sevabertinib) received accelerated FDA approval on November 19, 2025 for adult patients with HER2‑mutated, non‑squamous non‑small cell lung cancer who have progressed after prior therapy. The approval is based on the single‑arm SOHO‑01 trial, which enrolled 122 patients across 78...
A year-long trastuzumab emtansine (T‑DM1) regimen showed 98% three‑year invasive‑disease‑free survival in early‑stage HER2‑positive breast cancer, comparable to standard paclitaxel‑plus‑trastuzumab. The ATEMPT trial of nearly 500 patients found similar overall survival but markedly lower neuropathy and hair loss with T‑DM1,...
The FDA granted accelerated approval to Boehringer Ingelheim’s oral HER2 kinase inhibitor Hernexeos six weeks after the company filed its application, marking the second approval under the new Commissioner’s National Priority Voucher (CNPV) program. The decision expands Hernexeos use to...
Today’s brief highlights four pre‑clinical advances: Paratus Sciences unveiled PS‑1001, a pan‑inflammasome blocker targeting IL‑1β and IL‑18 for hidradenitis suppurativa; researchers identified the SCAN circuit as a core pathway disrupted in Parkinson’s disease; RX‑10616 demonstrated enhanced radiotherapy response in head‑and‑neck...
Prospect Therapeutics announced the identification of a new series of small‑molecule inhibitors targeting Janus kinase 1 (JAK1) and tyrosine kinase 2 (TYK2). The lead compounds demonstrate nanomolar potency and selectivity in biochemical assays and effectively suppress cytokine signaling in cellular...
The Consolidated Appropriations Act of 2026 bans pharmacy benefit managers (PBMs) from receiving compensation tied to drug list prices and mandates 100% rebate pass‑through to plan sponsors by August 2028. The law also allocates roughly $190 million to the Centers for...
The FDA’s Drug Trials Snapshots program publishes concise, consumer‑focused summaries of the demographic makeup and outcomes of pivotal clinical trials for newly approved drugs. Launched in 2015, the tool covers only New Molecular Entities and original biologics approved after that...
Ionis Pharmaceuticals received FDA acceptance of its supplemental NDA for olezarsen and a priority‑review designation, with a PDUFA action date of June 30, 2026. The decision is backed by two Phase III CORE studies—CORE (n=617) and CORE2 (n=446)—that evaluated once‑monthly subcutaneous...
GSK’s depemokimab, marketed as EXDENSUR, received FDA approval on December 16, 2025 as a six‑month subcutaneous add‑on for severe eosinophilic asthma in patients aged 12 and older. Approval was based on two 52‑week, double‑blind, placebo‑controlled trials (SWIFT‑1 and SWIFT‑2) that...
The FDA issued a suite of new guidance documents in early 2026 aimed at easing development of therapies for rare diseases, including the Rare Disease Evidence Pathway, Plausible Mechanism Pathway, and innovative trial designs for cellular and gene therapies. The...
Quotient Sciences has extended its commercial partnership with Ipsen to manufacture Sohonos (Palovarotene), the approved therapy for fibrodysplasia ossificans progressiva (FOP), an ultra‑rare disease affecting fewer than 1,000 people worldwide. The deal includes Ipsen's investment in a pneumatic closed‑transfer system...
Eli Lilly disclosed Phase III ACHIEVE‑3 results showing its oral GLP‑1 agonist orforglipron outperformed oral semaglutide in lowering A1C and inducing weight loss. The 52‑week, open‑label trial enrolled 1,698 patients across six countries and compared two doses of each drug. Orforglipron also...
Bayer announced that its phase‑III PEACE‑3 trial demonstrated a statistically significant overall‑survival benefit for the combination of Xofigo (radium‑223) and enzalutamide versus enzalutamide alone in patients with metastatic castration‑resistant prostate cancer (mCRPC) and bone metastases. Median overall survival extended to...
PMV Pharmaceuticals reported Phase I results of rezatapopt, a small‑molecule p53 reactivator, in 77 patients with advanced solid tumors carrying the TP53 Y220C mutation. The oral drug was generally well tolerated, with few dose‑limiting toxicities, allowing the selection of a...
The FDA has warned consumers against buying ULTRA ADVANC3 on Amazon and ULTRA ADVANC3 GOLD on NaturistaRex after laboratory tests revealed hidden prescription drugs. The supplements contain undeclared dexamethasone, diclofenac, and methocarbamol, each carrying serious health risks. The agency advises immediate discontinuation and...
Fulgent Genetics reported Q4 2025 revenue of $84.1 million, a sequential increase and strong year‑over‑year growth across its Precision Diagnostics and Biopharma segments. Non‑GAAP gross margin improved to 44.3% while operating expenses fell, delivering a $0.7 million adjusted EBITDA and a $4.5 million...
Senator Ron Johnson (R‑WI) is urging Congress to draft a “Right to Try 2.0” bill that would force the FDA to grant broader, faster access to experimental therapies for rare diseases. FDA Commissioner Martin Makary pushed back, defending the agency’s...
The U.S. Department of Justice filed an amicus brief supporting AbbVie in its effort to overturn a Colorado statute that prohibits pharmaceutical manufacturers from limiting discounts under the federal 340B drug pricing program when hospitals use contract pharmacies. The Colorado...
The FDA granted accelerated approval to zongertinib (Hernexeos) for adults with unresectable or metastatic non‑squamous NSCLC that carry activating HER2 TKD mutations, extending its use to treatment‑naive patients. The decision rests on the Beamion LUNG‑1 trial, which reported a 76%...
RIKEN scientists have shown that an intrinsically disordered region (IDR) of the DEAD‑box helicase DDX3X confers selective binding to specific mRNA structures, a mechanism uncovered using solution NMR spectroscopy. The discovery clarifies how DDX3X distinguishes target transcripts, linking its specificity...
ARUP Laboratories has unveiled the Innovation Central Laboratory, a dedicated hub for co‑development with pharmaceutical and biotech partners. The facility is designed to validate technologies and move next‑generation diagnostics from concept through to commercial readiness. It will host projects ranging...
The FDA approved KOMZIFTI (ziftomenib) on November 13 2025 as an oral 600 mg capsule for adult patients with relapsed or refractory acute myeloid leukemia (AML) harboring an NPM1 mutation. Approval rests on the single‑arm KO‑MEN‑001 trial, which enrolled 112 participants across seven...
The Centers for Medicare & Medicaid Services entered the third year of its Medicare drug price negotiation program, selecting 15 drugs—including, for the first time, therapies covered under Medicare Part B. Patient advocacy groups have until March 1 to submit written comments...
Bristol Myers Squibb reported that its antibody‑drug conjugate iza‑bren, licensed from Chinese partner SystImmune, achieved statistically significant improvements in progression‑free and overall survival versus chemotherapy in a Phase III trial for advanced triple‑negative breast cancer. The study, conducted in mainland China...
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Adding androgen deprivation therapy (ADT) to definitive radiotherapy improves overall survival for men with high‑risk prostate cancer, but its benefit when combined with postoperative radiotherapy (PORT) after prostatectomy is less clear. Randomised trials such as GETUG‑AFU 16, SPPORT, and the RADICALS‑HD...
The FDA announced it will default to a single pivotal trial for new drug applications, extending a practice long used in oncology and rare‑disease approvals. In 2024, 66% of new molecular entities were cleared based on one trial, signaling a...
The latest episode of BioSpace’s Denatured podcast spotlights a growing partnership between ALS patients and pharmaceutical developers, featuring insights from EverythingALS founder Indu Navar and VectorY Therapeutics CMO Dr. Olga Uspenskaya. The discussion highlights how patient‑driven collaborations are accelerating trial timelines...
The U.S. Food and Drug Administration rejected a rare‑disease cell therapy that had already secured approval in Europe, despite earlier internal support from the agency. The decision comes under the FDA’s new leadership and has sparked debate over whether the...
Atara Biotherapeutics’ stock jumped 20% after STAT reported that internal FDA inconsistencies may have driven the rejection of its EBV‑positive PTLD cell therapy, Ebvallo. The FDA denied the U.S. application citing insufficient evidence of effectiveness, yet former reviewers said data...
Boehringer Ingelheim announced a potential $500 million investment in UK biotech Sitryx to obtain global rights to an undisclosed small‑molecule immunology programme. The partnership covers upfront fees, milestones and royalties, giving Boehringer full responsibility for research, clinical development and commercialization. Sitryx’s...
ViVE 2026 in Los Angeles marked a decisive shift from digital ambition to operational accountability across the health ecosystem. Leaders emphasized that AI adoption now hinges on governance, measurable ROI, and clinician trust, while interoperability is being treated as core...
Researchers have captured the fleeting i‑DNA structure inside living cells, showing it acts as a molecular switch for genes that drive cancer. The study demonstrates that i‑DNA formation and resolution are tightly timed during DNA replication, influencing gene expression and...
Continuous cardiac monitoring via wearable ECG devices is reshaping clinical trials and post‑market care. Recent studies show that 14‑day monitoring after cardiac surgery uncovered atrial fibrillation in 24% of patients, many of which were missed until three‑month follow‑ups. In epilepsy...
Pfizer has signed a global gene‑editing partnership with Beam Therapeutics, reviving its genetics ambitions after withdrawing the hemophilia B therapy Beqvez a year earlier. The agreement includes a $300 million upfront payment and up to $1.05 billion in milestones, granting Pfizer exclusive worldwide...
Cara Medical announced that the U.S. FDA granted 510(k) clearance for its CARA System, a non‑invasive solution that creates patient‑specific 3‑D visualizations of the cardiac conduction system using CTA data. The platform pairs the CARA Metis™ Simulator for pre‑procedural mapping...
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Oral GLP-1 receptor agonists have moved from injectable peptides to a tablet form, highlighted by oral semaglutide combined with the absorption enhancer SNAC. The formulation requires patients to fast overnight, take the dose with a small amount of water, and...
GlaxoSmithKline announced that China’s National Medical Products Administration has accepted the New Drug Application for linerixibat and placed it under priority review for treating cholestatic pruritus in primary biliary cholangitis (PBC). The filing is backed by the Phase III GLISTEN trial,...
Merck Animal Health announced that the U.S. FDA has approved Numelvi (atinvicitinib) for controlling pruritus linked to allergic dermatitis in dogs six months and older, with commercial availability slated for spring 2026. Numelvi is a second‑generation JAK inhibitor that demonstrates...
The FDA has rejected Regenxbio’s gene therapy for mucopolysaccharidosis type 2, citing a need for additional data that could postpone approval by years. This decision adds to at least four other recent rejections of cell or gene therapies targeting deadly rare...
