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Mar 23, 2026
Dizal's Phase III trial of Zegfrovy for first-line NSCLC with EGFR exon20ins met its primary endpoint, showing improved progression-free survival. Meanwhile, Immutep halted its Phase III eftilagimod alfa trial, and AstraZeneca's LATIFY trial of ceralasertib plus Imfinzi failed to meet its overall survival endpoint.
Mar 23, 2026
The global orphan drugs market is projected to grow from $223.76 billion in 2023 to $486.51 billion by 2032, with North America leading at 38% market share. Over 500 orphan drugs are approved with more than 800 candidates in clinical trials, driven by regulatory incentives and major pharmaceutical company investments in rare disease therapies.
Mar 23, 2026
GSK's antibody-drug conjugate risvutatug rezetecan has received Orphan Drug Designation in Japan for small-cell lung cancer, marking its sixth global regulatory designation. The decision was based on phase I trial data showing durable responses in extensive-stage SCLC patients. The drug is currently in phase III development for relapsed ES-SCLC.
Mar 23, 2026
Sarepta Therapeutics received FDA feedback supporting supplemental applications for AMONDYS 45 and VYONDYS 53, while New Zealand approved a Phase 1 trial for Huntington's Disease candidate SRP-1005. The company faces significant share price declines despite these developments, with analysts showing wide disagreement on valuation targets ranging from $5 to $80.
Mar 23, 2026
The global generic drugs market reached $389 billion in 2024 and is projected to grow to $675 billion by 2033. In the U.S., generics account for 91% of prescriptions but only 18% of drug spending, saving over $373 billion annually. Biosimilar adoption remains below 20% despite some products reaching over 60% market share within three years.
Mar 11, 2026
The FDA has approved leucovorin for cerebral folate deficiency with FOLR1 gene variant, not for autism broadly. Agency officials cited insufficient data for broader autism efficacy despite previous hype. Prescriptions for the drug surged 71% among children following earlier promotional statements.
Mar 19, 2026
Bayer's Phase III FIND-CKD trial shows finerenone significantly slows kidney function decline in non-diabetic chronic kidney disease patients. The drug met its primary endpoint with improved eGFR slope over 32 months, marking its fifth consecutive positive Phase III trial. Bayer plans to submit the data to the FDA for label expansion into non-diabetic CKD.
Mar 22, 2026
Prothena reported Q4 and full year 2025 financial results with $308.4 million in cash as partners Roche and Novo Nordisk initiated Phase 3 trials for prasinezumab and coramitug, both expected to complete in 2029.
Mar 21, 2026
uniQure disclosed the FDA will not accept Phase I/II data alone for AMT-130 approval, causing shares to drop 49%. The company held regulatory meetings and reported $622.5 million in cash while investors face an April 13 class action deadline.
Mar 21, 2026
The FDA has issued draft guidance creating a "Plausible Mechanism Framework" to accelerate approval of individualized therapies for ultra-rare diseases where traditional clinical trials are not feasible. The guidance focuses on gene editing and RNA-based treatments targeting specific genetic abnormalities.
Mar 21, 2026
Moderna reported Q4 revenues of $678 million, down 29.8% year-over-year but beating analyst expectations. The FDA reversed course and will review the company's mRNA flu vaccine application.
Mar 21, 2026
Deupirfenidone, a deuterated form of pirfenidone, is set to be tested in a phase 3 trial this year after successful phase 2b results. The FDA and European Commission have granted orphan drug designation to support its late-stage development.
Mar 20, 2026
Novo Nordisk received FDA approval for a 7.2-mg semaglutide dose marketed as Wegovy HD for adult patients with obesity who have tolerated the standard 2.4-mg dose. The approval came through the FDA's new Commissioner's National Priority Voucher pilot program.
Mar 20, 2026
The FDA has released draft guidance to help drug developers validate New Approach Methodologies as alternatives to animal testing, establishing four validation pillars and signaling a shift toward human-centric safety data in drug development.
Mar 20, 2026
Biogen announced that litifilimab received FDA Breakthrough Therapy Designation for cutaneous lupus erythematosus and will present Phase 2 AMETHYST study results at the 2026 AAD Annual Meeting, with Phase 3 data expected in 2027.
Mar 20, 2026
The FDA announced major policy changes including requiring only one study for drug approvals instead of two, and offering performance bonuses to reviewers who complete work ahead of schedule while maintaining thoroughness.
Mar 20, 2026
DelveInsight pipeline reports reveal robust clinical development across three cancer indications, with 45+ companies advancing cutaneous squamous cell carcinoma therapies, 70+ companies developing liver cancer treatments, and multiple hepatocellular carcinoma candidates receiving FDA designations in 2025-2026.
Mar 20, 2026
The FDA accepted Takeda's new drug application for oveporexton, an investigational orexin 2 receptor agonist for narcolepsy type 1, and granted priority review with a PDUFA action date in Q3 2026.
Mar 20, 2026
Northwestern University researchers found that levetiracetam, an FDA-approved anti-seizure medication, prevents formation of toxic amyloid-beta 42 peptides in the brain, offering a potential strategy for early Alzheimer's prevention.
Mar 20, 2026
Krystal Biotech posted Q4 2025 net revenue of $107.1 million from Vyjuvek sales, with full-year revenue reaching $389.1 million. The company expanded to over 90 patients across Germany, France, and Japan while advancing its pipeline with new FDA designations.
