Johnson & Johnson announced FDA approval of a supplemental new drug application for Caplyta (lumateperone) for the prevention of relapse in schizophrenia, based on compelling long-term clinical data and reported…
Novartis and PTC Therapeutics have advanced their Huntington's disease candidate, votoplam, into Phase 3 development following promising mid-stage results. The decision reflects confidence in the program's potential while signaling caution…
BridgeBio's Attruby (acoramidis) is positioning itself as a formidable competitor in the transthyretin amyloid cardiomyopathy (ATTR-CM) market, with new pivotal trial data and indirect comparisons suggesting potential advantages over Pfizer's…
A significant advancement in antiviral therapy has emerged with the successful development of a CRISPR-Cas13d-based system designed to combat hepatitis E virus (HEV) infections. This innovative approach harnesses the precision…
UniQure has announced plans to submit its groundbreaking Huntington's disease gene therapy, AMT-130, for regulatory approval in the United Kingdom later this year, marking a significant step forward for the…
Exelixis has taken a major step forward in its colorectal cancer development pipeline by securing supplies of Merck's Keytruda for an upcoming phase 3 clinical trial. As reported by Fierce…
Atossa Therapeutics Inc. has achieved a significant regulatory milestone with the U.S. Food and Drug Administration's grant of Rare Pediatric Disease (RPD) designation to (Z)-endoxifen for treating McCune-Albright syndrome (MAS)…
What happens when a pharmaceutical first-mover discovers its historic achievement may not be a blockbuster? Arvinas and Pfizer found an answer this week by handing off their pioneering breast cancer…
Abeona Therapeutics Inc. has announced a significant expansion in access to ZEVASKYN, marking the activation of Children's Hospital of Philadelphia (CHOP) as the sixth Qualified Treatment Center for this groundbreaking…
Systemic lupus erythematosus punishes patients twice. First comes the disease itself, an autoimmune disorder where the body attacks its own tissues, causing debilitating fatigue, joint swelling, fevers, and unpredictable flares.…
Mesenchymal stromal cells (MSCs) have tantalized the medical world for years. These versatile immune-modulating cells offer genuine hope for diseases ranging from epidermolysis bullosa, a genetic disorder where skin tears…
For decades, colorectal cancer treatment has followed the same playbook: surgeons remove the tumor, then patients endure months of chemotherapy hoping to catch any remaining cancer cells. Yet even with…
Enpatoran offers hope for millions suffering from lupus skin manifestations Pharmaceutical company Merck KGaA has announced a significant milestone in lupus treatment research. As reported by Business Wire, the first…
A major breakthrough for the 300 Irish patients living with butterfly skin. For the first time in Ireland, patients battling epidermolysis bullosa, a rare and devastating skin condition, now have…
Labcorp announced the nationwide availability of Agilent Technologies' PD-L1 IHC 22C3 pharmDx, the only FDA-approved companion diagnostic designed to identify patients with platinum-resistant ovarian cancer who may be eligible for…
Johnson & Johnson announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review to IMAAVY® (nipocalimab-aahu), marking a significant milestone as the first therapy to receive this…
AstraZeneca has announced positive results from the pivotal phase III MIRANDA trial of tozorakimab, a groundbreaking first-in-class monoclonal antibody designed to treat Chronic Obstructive Pulmonary Disease (COPD). According to PharmaBiz.com,…
The US Food and Drug Administration (FDA) has approved Idvynso, a groundbreaking two-drug single-tablet regimen combining 100 mg doravirine and 0.25 mg islatravir, for treating HIV-1 infection in adults. As…
Health Canada has officially approved Cysklar® (cysteamine ophthalmic solution) 0.44%, marking a significant breakthrough for Canadians living with cystinosis. According to BusinessWire.com, the approval enables patients to access a topical…
Cancer cachexia represents one of oncology's most overlooked catastrophes. Patients waste away, lose appetite, and deteriorate despite receiving chemotherapy, yet few treatment options exist to address this metabolic collapse. According…
I had learned that the many twists and turns on our rare disease path were similar to the families we met Originally published on May 11, 2022, this article explores celebrates author…
Diabetic macular edema represents a silent threat to working professionals across America. The condition, which causes fluid accumulation in the macula, the eye's central focusing region, ranks as the leading…
The aesthetic skincare market has long overlooked a fundamental principle: skin preparation matters as much as the procedure itself. According to PharmaBiz.com, Galderma's Alastin brand is now challenging that assumption…
Pancreatic cancer remains one of medicine's most formidable challenges, and recent clinical developments suggest a potential turning point. Revolution Medicines presented compelling evidence this week that its experimental drug daraxonrasib…
AskBio Inc., a gene therapy subsidiary of pharmaceutical giant Bayer AG, has taken a significant step forward in advancing treatment options for Parkinson's disease. According to PharmaBiz.com, the company announced…
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