Medical Breakthroughs of 2025

From novel treatments of lipid storage diseases, acute pain, and Hemophilia, to marketing authorizations of tests for the diagnosis of several conditions, here are some of the key medical breakthroughs of 2025, so far.

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Approval of the first-ever treatment for non-cystic fibrosis bronchiectasis

In August, the FDA approved Insmed’s BRINSUPRI™ (brensocatib), the first and only treatment for non-cystic fibrosis bronchiectasis (NCFB), a chronic and progressive lung disease that affects about 500,000 people in the U.S. BRINSUPRI is a once-daily oral therapy for adults and adolescents aged 12 and older. The condition causes airway widening, making it harder to clear mucus and bacteria, leading to repeated infections, inflammation, and lung damage.

Approval of first systemic therapy for diffuse midline glioma

In August, the FDA granted accelerated approval to Jazz Pharmaceuticals' Modeyso (dordaviprone), a protease activator, for the treatment of adults and children aged one year and older with diffuse midline glioma harboring an H3 K27M mutation and progressive disease after prior therapy. This marks the first FDA approval of a systemic therapy for this ultra-rare and aggressive brain tumor that affects an estimated 2,000 people in the U.S. each year, primarily children and young adults.

FDA clearance of the first blood test for Alzheimer’s diagnosis