Curated news and analysis on clinical trials, drug approvals, and medical research.
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.
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.
The U.S. Department of Health and Human Services formally scrapped its 2025 340B Rebate Model Pilot Program following federal court rulings and issued a new Request for Information on February 13, 2026, seeking stakeholder input on the future of 340B drug pricing.
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.
Aquestive Therapeutics received a Complete Response Letter from the FDA for Anaphylm (dibutepinephrine) sublingual film, citing deficiencies in human factors validation and packaging. The company plans to resubmit as early as Q3 2026.
A class action lawsuit was filed against REGENXBIO covering the period from February 9, 2022 to January 27, 2026, following FDA clinical holds on gene therapies RGX-111 and RGX-121 after a CNS tumor was discovered in a trial participant.
A study of 366 patients found ide-cel CAR-T cell therapy achieved 81.6% overall response rate in patients 70 or older with relapsed/refractory multiple myeloma, with median progression-free survival of 18.9 months versus 5.7 months with standard regimens.
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.
Metabolic pathway scores consistently distinguished patients who developed severe neurologic events after CAR T-cell therapy across six clinical trials, surpassing traditional inflammatory markers in predictive performance.
Researchers have developed an inhalable nanoparticle system for delivering rifampin directly to the lungs, potentially reducing tuberculosis treatment from daily to once-weekly dosing while lowering side effects and improving drug effectiveness.
The FDA has released draft guidance to help drug developers validate new approach methodologies as alternatives to animal testing, marking a milestone in the agency's roadmap to reduce reliance on animal studies in drug development.
Altesa BioSciences closed an oversubscribed $75 million Series B financing led by Forbion with participation from Sanofi to advance vapendavir, a first-in-class oral antiviral targeting rhinovirus infections in COPD patients.
Arvinas announced phase 1 results for ARV-102, a PROTAC degrader that achieved more than 50% LRRK2 degradation in cerebrospinal fluid of Parkinson's patients after 28 days, with no serious adverse events reported.
Insilico Medicine announced a partnership with China Medical System Holdings for AI-powered drug discovery and nominated ISM5059, an NLRP3-targeted drug candidate, for preclinical development following recent FDA IND approval for another AI-created drug.
Maryland lawmakers are advancing legislation to eliminate tax breaks for pharmaceutical company advertising, with 73% of residents supporting the measure. The bill would generate at least $23 million annually for Medicaid and the state health insurance marketplace.
Research into Lynch syndrome is advancing cancer prevention through aspirin therapy, vaccine trials, and a major North American study examining why some patients develop cancer while others remain protected.
Global pharmaceutical R&D deal values surged 49% to $86.7 billion in 2025 as companies concentrate investments in AI-powered drug discovery platforms, with average deal sizes reaching a record $1.16 billion despite fewer total partnerships.
The U.S. FDA has granted Fast Track designation to FluoGuide's FG001 as an intraoperative imaging agent for high-grade glioma surgical resection. The designation follows IND clearance in January and enables accelerated development and regulatory review.
A study of over 83,000 women found that initiating hormone therapy after age 65 significantly increases risks for cancer and cerebrovascular events, reinforcing current guidelines that discourage starting systemic hormone therapy after age 60.
Scientists at Cincinnati Children's have identified a molecular pathway responsible for immune checkpoint inhibitor-induced myocarditis and demonstrated that blocking TNF signaling can prevent this fatal complication without compromising anti-tumor efficacy.
