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Mar 17, 2026
Diagnosing rare diseases can take 3 to 15 years, while about 95% lack approved treatments. Families are advocating for regulatory changes to speed access to therapies as new digital tools aim to improve clinical trials.
Mar 17, 2026
At least seven people in California, Florida, and Texas have been sickened by E. coli linked to cheddar cheese made from raw milk by California-based Raw Farm. The company has declined to voluntarily recall its products.
Mar 17, 2026
Recent studies demonstrate high response rates for antibody-drug conjugate in rare blood cancer, improved outcomes with metastasis-directed radiation in prostate cancer, and a novel drug combination strategy for ovarian cancer resistance.
Mar 16, 2026
Northwestern Medicine scientists discovered that N-acetyl-L-leucine (NALL), an FDA-approved compound, promotes neuroprotective effects in experimental Parkinson's disease models by targeting multiple molecular pathways simultaneously.
Mar 16, 2026
Cogent Biosciences has submitted a New Drug Application for bezuclastinib to treat systemic mastocytosis caused by the KIT D816V mutation, with FDA review underway and strong analyst support.
Mar 16, 2026
Eli Lilly announced positive Phase 3 results for EBGLYSS in pediatric atopic dermatitis, orforglipron in diabetes showing superiority over Rybelsus, and Retevmo in early-stage lung cancer, expanding potential indications across its portfolio.
Mar 16, 2026
BioVersys AG received FDA approval to enroll U.S. patients in a global Phase 3 trial evaluating BV100 for hospital-acquired pneumonia caused by carbapenem-resistant Acinetobacter baumannii, with trial completion expected by end of 2027.
Mar 16, 2026
Telix Pharmaceuticals has submitted regulatory applications for TLX101-Px, a brain cancer imaging agent, to both the European Medicines Agency and the U.S. Food and Drug Administration, seeking approval for glioma diagnosis and treatment monitoring.
Mar 15, 2026
Regulatory incentives for rare disease treatments, including market exclusivity and development subsidies, are spurring pharmaceutical innovation across the US, Europe, Japan, and Australia, with multiple companies advancing therapies for conditions affecting limited patient populations.
Mar 15, 2026
Savara has submitted a Biologics License Application for MOLBREEVI to the FDA with Priority Review status, targeting an August 22, 2026 decision date for treating autoimmune pulmonary alveolar proteinosis.
Mar 15, 2026
CAR T-cell therapy is delivering unprecedented results for multiple myeloma patients, with CARTITUDE-1 trial data showing one-third of patients remaining progression-free for five years or more, raising the possibility of cure for some patients.
Mar 15, 2026
Cogent Biosciences is preparing parallel FDA submissions for bezuclastinib across three indications following positive pivotal trial results, with potential launches beginning in late 2026 and full commercialization expected by early 2027.
Mar 14, 2026
YolTech Therapeutics received FDA approval for its IND application for YOLT-202, an in vivo gene-editing therapy for Alpha-1 Antitrypsin Deficiency. Interim data from an ongoing trial showed rapid increases in AAT levels to normal range with favorable safety.
Mar 14, 2026
Sen. Josh Hawley and Rep. Diana Harshbarger introduced legislation to withdraw FDA approval of mifepristone nationwide, making distribution a federal violation and allowing patients to sue manufacturers.
Mar 14, 2026
AbbVie reported Q4 earnings that beat consensus estimates with $16.62 billion in revenue, while facing competitive setbacks in psoriatic arthritis and Medicare pricing challenges for Botox.
Mar 14, 2026
The ASCENT-04 trial showed sacituzumab govitecan plus pembrolizumab improved progression-free survival in PD-L1-positive TNBC without increased toxicity. Meanwhile, experts debate whether ADCs function as targeted therapy or advanced chemotherapy, with sequencing questions remaining unresolved.
Mar 13, 2026
The FDA has lifted a Complete Response Letter and resumed review of Capricor Therapeutics' Biologics License Application for deramiocel, setting a PDUFA date of August 22nd. The company ended 2025 with $318M in cash.
Mar 14, 2026
The Consolidated Appropriations Act, 2026, enacted February 3, reauthorizes the rare pediatric disease priority review voucher program through 2029, clarifies orphan drug exclusivity scope, and mandates increased transparency in FDA's generic drug determinations.
Mar 13, 2026
Junshi Biosciences reported total revenue of approximately RMB2,498 million in 2025, a 28% increase year-over-year, driven by toripalimab sales growth. Absci Corporation announced it will report fourth quarter and full year 2025 financial results on March 24, 2026.
Mar 14, 2026
The FDA has authorized the first human trial of an allogeneic stem cell therapy for epilepsy, developed by Shanghai-based Unixell Biotechnology. The therapy uses donor-derived stem cells to produce GABA and suppress seizures in drug-resistant patients.
