White House Announces MFN Agreement with Drug Manufacturer Regeneron
The White House unveiled a most‑favored‑nation (MFN) pricing agreement with Regeneron covering its blockbuster eye‑disease drug Eylea. The pact obligates Regeneron to extend to Medicare the lowest net price it offers any private payer, with annual price reviews built into the contract. In addition, the deal expands eligibility for low‑income beneficiaries and requires the company to share cost‑savings data with the administration. The agreement is part of a broader federal push to rein in prescription‑drug spending and improve access to high‑cost biologics.
FDA Fast-Tracks the Development of Psychedelic Medications Following President’s Executive Order
The FDA announced an accelerated regulatory pathway for psychedelic‑based therapies after the April 18 executive order targeting serious mental illness. It will prioritize serotonin‑2A agonists for treatment‑resistant depression, PTSD and substance‑use disorders, and issue national‑priority vouchers for psilocybin and methylone studies...
FDA Grants Three Priority Vouchers to Psychedelics
The FDA has granted three Commissioner's National Priority Vouchers to psychedelic‑focused firms—Compass Pathways, Transcend Therapeutics and the Usona Institute—following President Trump’s executive order to accelerate mental‑health treatments. All three companies already hold Breakthrough Therapy Designations, and the vouchers promise faster...
ALONE-AF: Stopping OAC After Ablation Doesn’t Reduce Cognitive Function
A substudy of the ALONE‑AF trial found that stopping oral anticoagulation (OAC) after successful catheter ablation did not lead to cognitive decline over two years. Montreal Cognitive Assessment (MoCA) scores rose modestly in both the OAC and no‑OAC groups, with...
Tortugas Neuroscience Nabs $106M
Tortugas Neuroscience announced a $106 million seed and Series A financing round, one of the larger early‑stage biotech raises this year. The capital, led by Cure Ventures and co‑led by The Column Group and AN Venture Partners, will fund the completion of...
The BioPharm Brief: Innovation, Gene Therapy Momentum, and Next-Gen ADCs Lead Today’s Headlines
The latest Ron Lanton Report shows biopharma innovation moving beyond Boston and San Francisco into emerging regions powered by academic collaborations, venture capital, and niche talent pools. The European Medicines Agency issued a positive opinion on Novartis’ intrathecal onasemnogene abeparvovec, expanding...
The Slow March of Clinical Biomarkers to Become Surrogate Endpoints
Clinicians are urging the FDA to recognize more clinical biomarkers as surrogate endpoints, hoping to accelerate drug approvals and reduce trial durations. Regulators remain cautious, approving only six biomarkers through the 2007 Biomarker Qualification Programme and often demanding definitive clinical...
Alphamab Oncology to Present Phase 1 Data on Bispecific ADC JSKN016 in HER2-Negative Breast Cancer at ASCO 2026
Alphamab Oncology will unveil Phase 1 results for its bispecific antibody‑drug conjugate JSKN016 at ASCO 2026, focusing on HER2‑negative breast cancer. The first‑in‑human trial in China demonstrated antitumor activity and a manageable safety profile in heavily pretreated patients, including triple‑negative cases. JSKN016...
Semaglutide NAION Risk Higher than Risk with SGLT2 Inhibitors
A VA‑based study published in JAMA Ophthalmology found that patients with type 2 diabetes taking semaglutide experienced more than double the risk of nonarteritic anterior ischemic optic neuropathy (NAION) compared with those on SGLT2 inhibitors. The analysis covered 102,361 veterans, with...
Policy Watch: FDA Moves to Implement Trump Order on Psychedelic Drugs
President Trump’s recent executive order tasked the FDA and ARPA‑H with accelerating psychedelic research. In response, the FDA issued Commissioner’s National Priority Vouchers to three firms—Compass Pathways, Transcend Therapeutics and the Usona Institute—fast‑tracking review of psilocybin and methylone candidates for...
STAT+: FDA to Speed up Review of Three Psychedelics as Mental Health Treatments
The FDA announced it will grant priority‑review vouchers to accelerate the evaluation of three psychedelic therapies—Compass Pathways’ psilocybin for treatment‑resistant depression, Usona Institute’s psilocybin for major depressive disorder, and Transcend Therapeutics’ MDMA‑like compound for PTSD. The move is part of...
Protecting Innovation for Psychedelic Therapies Fast-Tracked Under New Executive Order
President Donald J. Trump signed an executive order that fast‑tracks FDA approval for breakthrough psychedelic therapies targeting serious mental illness. The order creates a Commissioner’s National Priority Voucher program, establishes pre‑approval patient access pathways, and earmarks $50 million in federal funding...
EU Biotech Act: The European Commission’s Landmark Proposal to Strengthen Biotechnology in Europe
On December 2025 the European Commission unveiled the European Biotech Act, a sweeping proposal aimed at cementing the EU’s position as a global biotech leader against the United States and China. The Act introduces fast‑track regulatory pathways, a strategic‑project framework...
Re: England Athletics Promotion of Medical Knee Procedure to Runners on Eve of London Marathon Is Condemned by Experts
England Athletics recently promoted Arthrosamid, a Class IIb implantable knee device, to runners ahead of the London Marathon. The product, marketed as a "drug," bypasses many safety tests because it carries a CE mark that allows manufacturer self‑certification. A UK...
FDA Awards Three Priority Review Vouchers For Psychedelics
On April 24, 2026 the FDA announced it will issue three priority review vouchers to companies developing psychedelic therapies. The vouchers cover two psilocybin programs targeting treatment‑resistant and major depressive disorder, and a methylone program for post‑traumatic stress disorder. The...
FDA Unveils Three Psychedelic-Focused Commissioner Vouchers
The U.S. Food and Drug Administration announced it has issued fast‑track “commissioner vouchers” to three psychedelic‑focused companies. The agency withheld the identities of the sponsors, a move that surprised investors and analysts. The vouchers are designed to expedite regulatory review,...
AI-Designed Drugs by a DeepMind Spinoff Are Headed to Human Trials
Isomorphic Labs, the DeepMind spinoff behind AlphaFold, announced that its AI‑designed drug candidates will soon enter human clinical trials. The company’s new IsoDDE engine claims to double the accuracy of AlphaFold 3 in predicting protein‑small‑molecule interactions. Partnerships with Eli Lilly and Novartis...
Designer Baby Companies Are in Turmoil
Two high‑profile germline‑editing startups—Bootstrap Bio and Manhattan Genomics—have ceased operations within a year of launching. Bootstrap Bio folded after running out of capital and was further tarnished by the federal arrest of its chief science officer on child‑sex‑trafficking charges. Manhattan...
Biopharma Money Raised: Jan. 1-April 23, 2026
Regeneron’s Otarmeni, a gene‑therapy for congenital hearing loss, earned FDA accelerated approval and will be provided free of charge, marking a rare zero‑cost gene‑therapy launch. At AACR 2026, researchers highlighted breakthroughs in minimal residual disease (MRD) detection that sharpen relapse...
Postmarketing Requirements and Commitments: Reports
The FDA publishes annual reports in the Federal Register detailing the status of post‑marketing requirements (PMRs) and commitments (PMCs) for drugs and biologics, covering both CDER and CBER. These reports summarize data from internal databases and are updated quarterly on...
WHO Prequalifies First-Ever Malaria Treatment for Newborns and Infants, Adds New Diagnostic Tests
The World Health Organization has prequalified the first antimalarial drug formulated specifically for newborns and infants weighing 2‑5 kg—artemether‑lumefantrine—enabling public‑sector procurement for an estimated 30 million babies born each year in malaria‑endemic Africa. The agency also prequalified three rapid diagnostic tests that...
AAN 2026: Tavapadon Post-Hoc Analysis Strengthens Its D1/D5 Agonist Pitch
At the 2026 American Academy of Neurology meeting, AbbVie unveiled post‑hoc results from its Phase III TEMPO‑1 and TEMPO‑2 trials of tavapadon, a once‑daily oral D1/D5 partial agonist for early Parkinson’s disease. The analysis showed statistically significant improvements in seven of...
AI in Oncology Takes Off, Tackling HIV and Liver Disease, Pharma’s Recent Gains
At AACR 2026 in San Diego, AI-powered platforms took center stage, signaling a rapid shift toward data‑driven oncology. Parallel research revealed CRISPR screens that mapped CD4+ T‑cell genes that either promote or block HIV infection, while synthetic‑biology engineers demonstrated implantable...
AAN 2026: Head-to-Head Trial Shows Superiority of Qulipta for Migraine Prevention
AbbVie presented Phase IIIb TEMPLE trial data at AAN 2026, showing its oral gepant Qulipta outperforms topiramate in migraine prevention. Over 24 weeks, Qulipta had a 12.1% discontinuation rate versus 29.6% for topiramate and achieved a 73.7% responder rate (>50% reduction in...
Sanofi MS Drug Rejected in US Gets an Endorsement in Europe
Sanofi’s experimental multiple‑sclerosis drug tolebrutinib, marketed as Cenrifki, received a positive recommendation from the European Medicines Agency for secondary progressive MS, after the U.S. FDA rejected it earlier this year. The EMA’s endorsement paves the way for a final approval...
Drug Digest: Examining the Architecture of Next-Gen Biotherapeutic Modalities
Minaris Advanced Therapies’ chief commercial and technology officer, Dr. Eytan Abraham, discussed how multifunctional biotherapeutics are merging antibodies, ADCs and engineered cells to create more precise, personalized treatments. He highlighted multi‑targeting cell designs that improve specificity and reduce disease escape,...
Biogen Seeks Darzalex Rivalry in China for Multiple Myeloma with Felzartamab Deal
Biogen has secured exclusive rights to TJ Biopharma’s anti‑CD38 antibody felzartamab in Greater China for up to $850 million, including control of a pending biologics licence application for relapsed and refractory multiple myeloma. The drug will be manufactured domestically at TJ...
Novartis Pulls Pluvicto Prostate Cancer Label Expansion Filing in the EU
Novartis has pulled its European Medicines Agency (EMA) application to extend the label of Pluvicto, its lutetium‑177 PSMA‑targeted radioligand therapy, into earlier‑stage prostate cancer. The drug already received approval for this indication in the United States and the United Kingdom,...
Codeine: Why One Person’s Painkiller Can Be Another Person’s Problem
Codeine, a weak opioid commonly sold OTC in the UK, is metabolised into morphine by the liver enzyme CYP2D6, creating wide variability in its effectiveness and safety. Genetic differences mean ultra‑rapid metabolisers (1‑2% of the population) can experience dangerously high...
Exclusive: UC Berkeley Startup Bets on Jumping Genes for GLP-1 Gene Therapy
A UC Berkeley spin‑out is developing a gene‑therapy platform that inserts a GLP‑1‑producing cassette into patients' cells using a jumping‑gene (transposon) system. The approach seeks to turn the body into its own continuous source of the hormone, eliminating the need...
AAN 2026: Long Half-Life and Long-Acting Injectable, What Pharmacists Should Know About RAP-219's Dosing Advantages for Focal Epilepsy
Rapport Therapeutics presented phase 2 data for RAP‑219 at the AAN 2026 meeting, showing 24% of drug‑resistant focal epilepsy patients achieved complete seizure freedom over an eight‑week period. The trial leveraged responsive neurostimulation (RNS) devices to capture objective electrographic seizure data,...
New Antibody Drugs Target Disease From Within
Researchers have used AI to redesign antibody binding fragments, creating more than 600 stable intracellular versions. By adjusting charge distribution, these fragments remain soluble inside cells and retain target specificity, enabling direct binding to disease‑driving proteins such as those implicated...
Regulatory Round-Up: Novartis, Sanofi and Arrowhead Secure CHMP Blessings
The European Committee for Medicinal Products (CHMP) issued positive opinions on three therapies: Sanofi's BTK inhibitor cenrifki for non‑relapsing secondary progressive multiple sclerosis, Arrowhead's siRNA drug redemplo for familial chylomicronemia syndrome, and Novartis' gene‑replacement therapy itvisma for spinal muscular atrophy....
STAT+: Pharmalittle: We’re Reading About a Trump Deal with Regeneron, Reclassifying Medical Marijuana, and More
President Trump sealed a private drug‑pricing pact with Regeneron, securing lower Medicaid prices, a $225 price point for Praluent, and a $27 billion commitment to U.S. drug development. The agreement also coincided with FDA approval of Otarmeni, the first gene therapy...
Precision Delivered: How Radiopharmaceuticals Are Reshaping Cancer Care
Radiopharmaceuticals, especially alpha‑emitting agents, are emerging as precision tools that deliver high‑LET radiation directly to cancer cells while sparing healthy tissue. Building on decades of beta‑emitter use, alpha therapies generate dense DNA damage and are less dependent on tumor oxygenation....
The Road Ahead—What’s Next for Host Cell Protein Analytics?
Advanced LC‑MS strategies are reshaping host cell protein (HCP) analytics, moving beyond the semi‑quantitative limits of traditional ELISA. Data‑independent acquisition (DIA) offers broad relative profiling, while stable‑isotope‑labeled (SIL) peptides provide absolute quantification for high‑risk HCPs. Integrating DIA, SIL, and ELISA...
AJMC® in the Press, April 24, 2026
AJMC highlighted four peer‑reviewed studies that expose cost‑driving inefficiencies in Medicare. One shows generic dispensing rates vary widely, suggesting billions in savings if low‑cost drugs are fully utilized. Another reveals Medicare’s wage‑index formula underpays rural hospitals for device‑intensive surgeries. A...
Bispecific Antibodies Are Reshaping Multiple Myeloma Care: Prerna Mewawalla, MD
Bispecific antibodies are rapidly reshaping multiple myeloma treatment by delivering response rates up to 80% in earlier relapse settings, far surpassing the historic 30% seen with conventional therapies. These agents simultaneously bind a myeloma antigen such as BCMA, GPRC5D or...
Pfizer Presents Auristatin S ADC for GPNMB Tumors
Pfizer has unveiled PF-08046033, an antibody‑drug conjugate that links the potent cytotoxic agent auristatin S to an antibody targeting the transmembrane glycoprotein NMB (GPNMB). The ADC is designed to deliver the payload directly into GPNMB‑expressing tumors, potentially widening the therapeutic...
Pharma Industry Faces Long Haul to Get Return on Investment From AI
The pharmaceutical sector is confronting a prolonged timeline before artificial intelligence delivers a clear return on investment. While AI tools have accelerated early‑stage drug discovery, most companies still grapple with data integration, regulatory compliance, and the high cost of talent....
Modified CRISPR Tool Targets Down Syndrome Mutation
Researchers at Beth Israel Deaconess Medical Center and Harvard Medical School have engineered a modified CRISPR system that inserts the long non‑coding RNA XIST into one copy of chromosome 21, silencing the extra genetic material that causes Down syndrome. The new...
Pfizer Culls Early PD-L1 Asset After Series of Clinical Wins, Deals in Cancer
Pfizer terminated the Phase 1 study of its early‑stage PD‑L1 antibody‑drug conjugate PF‑08046037 after enrolling eight patients, citing strategic business reasons rather than safety or efficacy concerns. At the same time, the company announced a 47% reduction in disease‑progression risk for...
Arrowhead Pharmaceuticals Receives the CHMP’s Positive Opinion for Redemplo (Plozasiran) to Treat Familial Chylomicronemia Syndrome (FCS)
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion on Arrowhead Pharmaceuticals’ RNAi therapy Redemplo (plozasiran) as an adjunct to diet for adults with familial chylomicronemia syndrome (FCS). The recommendation follows the Phase III...
Japanese Pharma Companies Turning to CDMOs Earlier in Product Life Cycle
Japanese pharmaceutical firms are engaging contract development and manufacturing organizations (CDMOs) earlier in the product lifecycle, especially for peptide programs. Over the past 12‑24 months, demand for preclinical and early‑clinical peptide services has risen sharply, driven by venture‑backed biotech spinouts...
Lilly’s CAR-T Devotion; CBER’s Next Top Dog?
Eli Lilly announced the acquisition of Kelonia Therapeutics, an in‑vivo CAR‑T biotech, extending its push into next‑generation cell therapies. The deal follows Lilly’s recent milestone of becoming the first drugmaker to hit a $1 trillion market value, underscoring a diversification strategy that...
The Problem with Psychedelic Research
President Trump signed an executive order to speed the approval of psychedelic drugs for veterans with PTSD and depression, marking the first major regulatory push for these treatments. A new review of 24 studies compared psychedelics to open‑label antidepressants and...
Is Pharma Missing the Boat on Diagnostics?
Pharma giants are accelerating investments in diagnostics to support precision‑medicine strategies, highlighted by Roche's $595 million purchase of SAGA Diagnostics and Abbott's $21 billion acquisition of Exact Sciences. Venture capital for diagnostics lagged behind AI, with only $1.7 billion raised last year versus...
KT-621 Gets FDA Fast Track Designation for Eosinophilic Asthma
Kymera Therapeutics received FDA fast‑track designation for its oral STAT6 degrader KT‑621, targeting moderate‑to‑severe eosinophilic asthma and atopic dermatitis. The first‑in‑class molecule works intracellularly, offering a potential oral alternative to injectable biologics that block the IL‑4/IL‑13 pathway. Phase 2b trials are...
This Artificial Retina Doesn't Just Aim to Restore Sight—It Opens a Hidden Channel of Vision
Researchers at Yonsei University and the Institute for Basic Science have unveiled an implantable artificial retina that detects near‑infrared (NIR) light and converts it into electrical pulses to stimulate surviving retinal ganglion cells. The device combines a phototransistor array with...
Sanofi’s Tolebrutinib Gets CHMP Backing for Certain MS Patients Despite FDA Rejection
Sanofi’s BTK inhibitor tolebrutinib received a positive opinion from the European Medicines Agency’s CHMP for treating relapsing forms of multiple sclerosis, despite a complete response letter from the U.S. FDA in December. The CHMP recommendation paves the way for a...