Pharmaceuticals
In a significant regulatory milestone that could transform treatment for one of medicine's most challenging rare diseases, South Korea-based Hanmi Pharmaceutical announced on February 5, 2026, that the FDA has granted Breakthrough Therapy Designation to efpeglucagon (HM15136) for treating congenital hyperinsulinism (CHI). This designation positions the investigational
Pharmaceuticals
Bristol Myers Squibb’s $850M Janux deal highlights growing pharma interest in tumor-activated immunotherapy. Purple Biotech is an under-the-radar player advancing a conditionally activated, multi-mechanism approach aligned with this emerging shift in solid tumor treatment.
Biotechnology
In a significant regulatory milestone that could transform diagnostic testing for a critical nutritional deficiency, Brooklyn-based Micoy Therapeutics announced on January 12, 2026, that the FDA has granted Breakthrough Device Designation to its NeuroLume™ Test. This innovative point-of-care diagnostic represents the first FDA-recognized test specifically designed
Biotechnology
In a significant regulatory achievement that could transform treatment for millions of Americans living with a debilitating autoimmune skin condition, Biogen announced on January 28, 2026, that the FDA has granted Breakthrough Therapy Designation to litifilimab (BIIB059) for treating cutaneous lupus erythematosus (CLE). This milestone positions the investigational therapy as
Biotechnology
Florida-based Adolore BioTherapeutics announced that the FDA has granted Orphan Drug Designation to its innovative rdHSV-CA8* gene therapy for treating erythromelalgia, positioning the company at the forefront of a revolutionary approach to chronic pain management.
Pharmaceuticals
Pharming Group announced that the FDA issued a Complete Response Letter for its supplemental application to expand Joenja treatment to children aged 4-11 with APDS, citing concerns about dosing precision and manufacturing quality.
Pharmaceuticals
Tenpoint Therapeutics announces FDA approval of YUVEZZI, the first dual-agent prescription eye drop for presbyopia, marking a historic regulatory milestone in treating age-related vision loss affecting 128 million Americans.
Pharmaceuticals
Serina Therapeutics announced FDA clearance of its IND application for SER-252, an innovative apomorphine therapy for advanced Parkinson's disease, marking a significant regulatory victory after successfully addressing previous clinical hold concerns.
Biotechnology
Siren Biotechnology announces FDA clearance for SRN-101, marking the first FDA-cleared AAV-based therapy specifically designed for oncology, advancing into human clinical trials for high-grade glioma treatment.
Biotechnology
Intellia Therapeutics announces FDA lift of clinical hold on MAGNITUDE-2 Phase 3 trial for its CRISPR-based therapy targeting hereditary transthyretin amyloidosis, marking a crucial milestone in navigating safety concerns in gene editing.
Biotechnology
UK-based Scancell Holdings announced FDA clearance for its Phase 3 trial of iSCIB1+, a novel DNA immunotherapy for advanced melanoma, following compelling Phase 2 data showing a 24-percentage-point improvement in progression-free survival.
Pharmaceuticals
COVID-19 vaccines did more than help end a global pandemic. They also introduced the public to a once-fringe idea: RNA itself can be medicine. Silexion Therapeutics is using RNAi to advance a next generation potential therapy for once of the toughest cancers | NASDAQ: SLXN