Breakthrough Designation

Seattle's Sound Pharma completes key Phase 3 enrollment for SPI-1005, putting the first-ever approved drug for debilitating Meniere's disease on the horizon.

A surge in biotech innovation is set to transform the high-grade glioma market. With a pipeline of targeted drugs and vaccines, a once-stagnant field is now a beacon of hope.

Kowa's pemafibrate gets FDA Breakthrough status for a rare liver disease. We dissect what this means for patients, the science, and the company's global strategy.

Aidoc's 'First Read' AI has earned a coveted FDA Breakthrough designation, promising to ease radiologist workload. But can an algorithm be trusted with your health?

Praxis Precision Medicines' elsunersen earns Breakthrough Therapy status, signaling a major step toward the first disease-modifying treatment for SCN2A-DEE.

Vanda Pharmaceuticals' rare disease drug gets a key designation in Japan, unlocking major incentives and promising a new treatment for a severe skin disease.

MBX Biosciences prepares for the Jefferies conference, with investors eyeing updates on its late-stage hypoparathyroidism drug and promising obesity portfolio.

A $290M war chest fuels a new public biotech aiming to help millions of migraine patients who don't respond to current blockbuster therapies.

A pivotal trial for a rare muscle disease failed its main goal, but promising data in a specific patient group offers a new, complex path forward.