Know What's Happening in Pharma
Know What's Happening in Pharma
FDA approves KRESLADI, first CIRM‑funded gene‑editing therapy for LAD‑I
The FDA has granted approval to KRESLADI, a gene‑editing therapy from Rocket Pharmaceuticals that treats severe leukocyte adhesion deficiency‑I in children without a matching bone‑marrow donor. This marks the first FDA‑approved product directly funded by California’s Institute for Regenerative Medicine (CIRM), highlighting the agency’s impact on translational research.
Also developing:
By the numbers: Lupin Limited acquires VISUfarma from GHO Capital
In February 2026 the European Commission and the CHMP granted marketing authorisations and positive opinions for a slate of innovative therapies spanning COVID‑19, oncology, cardiology, immunology and rare diseases. Notable approvals include Moderna’s mNEXSPIKE COVID‑19 vaccine, Hansoh’s Aumseqa for EGFR‑mutated NSCLC, Cytokinetics’ Myqorzo for obstructive hypertrophic cardiomyopathy, ImmunityBio’s Anktiva + BCG for BCG‑unresponsive bladder cancer, and GSK’s Exdensur for type‑2 asthma and chronic rhinosinusitis with polyps. The CHMP also issued conditional approvals for Ipsen’s Ojemda in pediatric low‑grade glioma, X4’s Xolremdi for WHIM syndrome, Novartis’ remibrutinib for chronic spontaneous urticaria, and Moderna’s mCOMBRIAX combo flu/COVID vaccine. These decisions were underpinned by robust Phase II/III data demonstrating superior efficacy, improved patient‑reported outcomes and favourable safety profiles across the EU.
In this DDW Highlights episode, Bruno Quinney reviews four major stories: a Mayo Clinic study linking the Parkinson's protein alpha‑synuclein to dramatically faster Alzheimer’s progression in women; a Texas A&M‑funded project testing extracellular vesicle (EV) therapy to modulate microglia and...
Pfizer disclosed topline Phase II (FOURLIGHT‑1) data showing that atirmociclib combined with fulvestrant outperformed fulvestrant alone or everolimus + exemestane in 264 HR+, HER2‑negative metastatic breast cancer patients previously treated with CDK 4/6 inhibitors. The trial met its primary endpoint, delivering a statistically significant...
Scientists are accelerating development of mRNA cancer vaccines, with more than 120 clinical trials targeting melanoma, brain, breast, lung and prostate tumors. Early studies, such as personalized vaccines for glioblastoma, demonstrate rapid immune activation and improved survival. Simultaneously, a false...
In response to suggesting peptide drugs should have good evidence on safety and efficacy in order to weigh the risks and benefit…
U.S. lawmakers warned that China dominates the supply chain for essential hospital and outpatient medicines, especially generic drugs that fill 90% of prescriptions. Witnesses at a Senate Aging Committee hearing highlighted that China controls roughly 90% of key starting materials...
Acute asthma and COPD exacerbations remain a costly, life‑threatening gap despite advances in chronic therapies. Connect Biopharma, led by Barry Quart, is the first biotech targeting these flare‑ups with a biologic that blocks IL‑4Rα, aiming to curb upstream inflammation. The...
Scientists from VIB and KU Leuven have identified the mechanism by which lecanemab (Leqembi) clears amyloid plaques in Alzheimer’s disease. Their study shows that the antibody’s Fc fragment engages microglia, reprogramming them to phagocytose and degrade plaques. Removing the Fc...
R1 starts up with $78M, aiming for a better kidney drug https://t.co/PQQjX47ODM by @gwendolynawu #biotecjh #startups
Sleeping sickness could be wiped out with this new drug: my comments @NPR @NPRGlobalHealth https://t.co/T0mAeX8P0A
The article outlines BioCentury’s cookie policy, detailing categories of cookies used on its website. Strictly necessary cookies are always active and enable authentication, registration, and core navigation. Functional, marketing, advertising, and analytics cookies provide personalization, targeted promotions, ad tracking, and...
A Phase I/II trial of AMT‑130, an AAV‑delivered microRNA gene therapy, showed a 75% reduction in Huntington's disease progression over three years in 12 patients. The FDA initially supported using external control data from the Enroll‑HD database for the Biologics...
The blog by HotSpot Therapeutics CEO Jonathan Montagu argues that China is no longer just a manufacturing hub but a burgeoning source of innovative drug candidates, evidenced by its 17% lead over the U.S. in Nature’s Index and nearly half...
The European Commission has granted approval for AstraZeneca’s Imfinzi (durvalumab) combined with FLOT chemotherapy as a perioperative treatment for resectable, early‑stage and locally advanced gastric and gastro‑esophageal junction (G/GEJ) cancers. The decision is based on the Phase III MATTERHORN trial, which...
GEN Pharmaceuticals received BEBO Foundation ethical approval to launch a Phase II proof‑of‑concept trial of its mitochondrial‑targeting drug SUL‑238 in Parkinson’s disease. The single‑centre, randomised, double‑blind, placebo‑controlled study, named SHEPHERD, will begin patient enrolment in Groningen in April 2026. Over a 28‑day...
Semaglutide’s patent will lapse in India on March 24, 2026, unlocking generic competition that could slash prices by up to 90%. The GLP‑1 drug, sold as Ozempic and Wegovy, has been shown to cut major cardiovascular events by 20%. Indian regulators, however,...
The White House is intensifying pressure on Congress to pass a “most‑favored nation” drug‑pricing bill that would cap U.S. medication prices at levels paid by peer nations. Administration officials say the proposal would overhaul pricing for providers, insurers, federal programs...
Clinical trials are adopting integrated eCOA platforms that connect medical devices directly to digital systems, eliminating manual data entry and improving data quality. Interoperability enables real‑time monitoring and AI‑driven insights, reducing patient burden especially in long‑duration obesity studies. The obesity...
NeuroScientific Biopharmaceuticals has begun its first manufacturing run of the StemSmart mesenchymal stem cell therapy at Q‑Gen Cell Therapeutics in Brisbane, initiating a critical technology transfer. The engineering run will validate quality, potency and regulatory compliance ahead of a Phase 2...
Abeona Therapeutics reported its Q4 2018 results, highlighting progress on its lead cell therapy EB-101 for recessive dystrophic epidermolysis bullosa (RDEB). The Phase 1/2 trial demonstrated continuous type VII collagen expression for over two years, a favorable safety profile, and durable wound...
Ionis Pharmaceuticals announced that its Phase 1/2a PrProfile trial of ION717, the first PrP‑lowering antisense oligonucleotide for symptomatic prion disease, has reopened with a third dosing regimen. The study, which enrolled 56 patients in 2024, will now recruit at three...
A systematic review published in Lancet Psychiatry, analysing 54 randomized controlled trials from 1980‑2025, found no evidence that medicinal cannabis treats depression, anxiety or PTSD. The paper highlights modest benefits for conditions such as epilepsy, multiple sclerosis spasticity, pain and...
Roche announced a new AI factory, adding 2,176 Nvidia Blackwell GPUs to its existing pool and bringing total GPU count above 3,500 – the highest in the pharmaceutical sector. The purchase, likely costing $65 million or more, aims to accelerate drug...
The TrumpRx website now lists 54 prescription drugs after adding seven products from GlaxoSmithKline and Amgen. The expansion pushes the total from 47 to 54, but health‑policy analysts note that the majority are older medicines nearing the end of their...
On July 23, 2020 the FDA issued new recommendations urging health‑care professionals to discuss and consider prescribing naloxone for any patient at heightened risk of opioid overdose. The guidance covers three groups: patients on opioid pain relievers, patients receiving medications...
The FDA is revising the boxed warning for every benzodiazepine product to explicitly list risks of abuse, misuse, addiction, physical dependence, and withdrawal. Updated patient Medication Guides will mirror these warnings, and prescribing information sections such as Warnings, Precautions, and...
The FDA has issued a series of safety communications and label updates for sedative‑hypnotic drugs, including a 2019 boxed warning for sleepwalking injuries and stricter dosing guidance for zolpidem products. Recent alerts also highlight the lethal risk of combining benzodiazepines...
The FDA has approved updated labeling for all pioglitazone‑containing medicines, adding a warning that use longer than one year may increase bladder cancer risk. Healthcare professionals are instructed not to prescribe pioglitazone to patients with active bladder cancer and to...
The FDA has approved label changes for general anesthetic and sedation drugs used in children under three, adding a warning that prolonged exposure—more than three hours or multiple procedures—may harm brain development. The revisions incorporate animal study data showing neuronal...
Kennedy childhood vaccine overhaul stalled by judge The next meeting of the Advisory Committee on Immunization Practices was scheduled for this week, but has been postponed due to the ruling https://t.co/0cUIXEVgyV
Representative Chip Roy introduced the Let Injured Americans Be Legally Empowered (LIABLE) Act, which would strip COVID‑19 vaccine manufacturers of the federal immunity granted under the PREP Act. The bill would permit civil lawsuits against Pfizer, Moderna and others, even...
Scientists have engineered silica‑based nanoparticles that encapsulate rifampicin and release it only when exposed to low‑frequency ultrasound. The ultrasound both propels the particles through the protective matrix of bacterial biofilms and triggers cavitation that opens the particles, delivering the antibiotic...
Beyond the Weight Loss: The Effects of Semaglutide on Standard and Diet-Induced Obese Mice 🤔"..semaglutide improved glucose metabolic health inboth diet groups..while chronic semaglutide treatment appeared to exertanxiolytic effects in obese mice, opposit[e] effects were observed in lean animals.." https://t.co/doU6EkwT7z
GlaxoSmithKline replaced its Flovent HFA and Flovent Diskus inhalers with an identical authorized‑generic version priced substantially higher. The move was designed to sidestep Medicaid rebate requirements, prompting health plans and pharmacy benefit managers to erect new access hurdles. Senator Maggie...
FDA commissioner Makary at a CMS conference today in Baltimore calls for “big and different” IRB reforms to catch China's speedy trial starts - pre-IND phase can run 380 days in the US, he said, while China is pushing for...
NovaBridge’s VIS‑101, a dual VEGF‑A and angiopoietin‑2 inhibitor, delivered rapid and durable efficacy in a phase 2a trial of 38 wet AMD patients in China. Participants receiving 3 mg or 6 mg doses achieved mean visual gains exceeding 10 ETDRS letters and central...
Structure Therapeutics announced positive topline data from its ACCESS II trial of aleniglipron, an oral small‑molecule GLP‑1 receptor agonist. The study demonstrated statistically significant reductions in HbA1c and weight loss comparable to injectable GLP‑1 therapies. Safety signals were mild, with few...
CytomX surges on positive data for ‘masked’ ADC in colorectal cancer https://t.co/PxQMISvyri by @Lilah_Alvarado $CTMX + 66%
CytomX Therapeutics’ shares surged 66% after the company released expansion data from its Phase 1 study of Varseta‑M, a masked antibody‑drug conjugate targeting EpCAM in late‑stage colorectal cancer. The trial evaluated three dose levels, with the two highest doses achieving response...
The FDA issued a formal warning letter to Novo Nordisk, citing serious violations for failing to report adverse events such as strokes, suicidal ideation, and patient deaths linked to its GLP‑1 drugs Ozempic, Wegovy and Saxenda. Inspectors found the company...
Structure’s GLP-1 pill shows ‘best-in-class’ potential in obesity trial https://t.co/j0fzAg6GRV @ByJonGardner $GPCR $LLY $NVO $AZN $MRK #obesity
Sun Pharmaceutical's tildrakizumab (Ilumya) received FDA acceptance for a supplemental biologics license application to treat active psoriatic arthritis. The sBLA is backed by two Phase III INSPIRE studies involving more than 800 patients, which met primary endpoints with significant ACR20 improvements...
Researchers have identified that a surface‑defensive molecule on cancer cells not only sends a “don’t‑eat‑me” signal but also conceals an “eat‑me” cue that would normally trigger immune clearance. A newly engineered antibody can disrupt this masking interaction, making tumors visible...
A retrospective TriNetX analysis of 1,691 psoriasis patients compared bimekizumab with IL‑23 inhibitors and found no greater incidence of new‑onset depression, suicidal ideation, or related medication use. Over a two‑year follow‑up, the combined psychiatric event rate was 4.9% for bimekizumab...
A polygenic risk score that predicts Type 2 diabetes, obesity, and likelihood for requiring a GLP-1 drug. Across 6 ancestries. https://t.co/5hTVv8kqlK https://t.co/IgXhx8vFA6
16March: Which companies recently withdrew their planned IPOs? And how much have the stocks of this year's #biotech IPOs risen or fallen this year? Read about the IPO market plus updates on $BNTX, $IXHL, and $INKT in my latest StockWatch...
A randomized, double‑blind study published in Mayo Clinic Proceedings found that a single 25 mg dose of off‑prescription Adderall caused significant acute increases in blood pressure and heart rate among healthy young adults. Systolic pressure rose by roughly 10 mm Hg, diastolic by...
The era of digital biology, exemplified by making a bespoke and effective vaccine for cancer in a dog, is taking off. Access to the AI-Coscientist. A new feature @NatureMedicine https://t.co/AR9lqne7E1
Correct interpretation of p > 0.05: The current sample size did not yield sufficient evidence to reject, at the incredibly arbitrary 0.05 level, the supposition that the treatment does nothing, assuming data model M (describe M ...) which forgot to...
Sentynl Therapeutics, a Zydus Lifesciences subsidiary, has licensed Progerinin, an oral small‑molecule candidate for Hutchinson‑Gilford Progeria Syndrome, from PRG S&T. The agreement grants Sentynl full development rights, making Progerinin its second HGPS therapy and giving it orphan‑drug status from the FDA....
