Biotechnology
On September 17, 2026, the FDA approved Fayuvi, the first treatment for Sanfilippo syndrome Type A—at a price of $3.95 million. What does this approval mean for rare disease medicine, access, and the future of gene therapy?
Pharmaceuticals
The FDA's approval of Kerendia for type 1 diabetes-associated kidney disease marks the first new treatment option in over 30 years for this underserved patient population.
Biotechnology
Vera Therapeutics' ORIGIN 3 Phase 3 trial demonstrates that TRUTAKNA met all prespecified endpoints, achieving eGFR stabilization and a 76% reduction in kidney disease progression risk in IgA nephropathy patients.
Policy
The FDA launched its Expedited IND Pilot Program on September 15, 2026, as part of Operation TrialBlazer. The ambition is real, but the delays driving research offshore run deeper than the mechanism being deployed to address them.
Pharmaceuticals
Novo Nordisk has rebranded as simply 'Novo.' The name change is the most visible signal yet that the company understands the urgency of its competitive challenge against Eli Lilly in the GLP-1 obesity drug market.
Biotechnology
GSK's Jideytro demonstrates a 94% response rate in first-line ROS1-positive NSCLC, with a remarkable 15% complete response rate and 100% intracranial response rate in patients with brain metastases.
Biotechnology
Novartis's remibrutinib has achieved what the BTK inhibitor class could not: a Phase 3 win in multiple sclerosis with no liver safety signal. Here's why this matters for the future of MS treatment.
Biotechnology
Amgen's Phase 3 DeLLphi-305 trial delivered a landmark overall survival win for tarlatamab in first-line extensive-stage small cell lung cancer, marking the first time a bispecific T-cell engager has demonstrated this benefit in this setting.
Biotechnology
Novartis's $12 billion acquisition of Avidity Biosciences and the subsequent failure of del-desiran in Phase 3 trials reveals critical lessons about the limits of acquisition-driven drug development in rare disease.
Biotechnology
uniQure has filed the first-ever gene therapy application for Huntington's disease. The science is compelling, the regulatory backstory is dramatic, and the stakes for 30,000 Americans and 200,000 at-risk individuals could not be higher.
Pharmaceuticals
AstraZeneca's Etcamah became the first FDA-approved cancer therapy triggered by a blood test detecting resistance before imaging shows progression. The approval rewrites how oncologists think about treatment switching in HR-positive breast cancer.
Biotechnology
The FDA's approval of Zanvastro marks the first treatment ever approved for Alexander disease, signaling a broader shift in how RNA-targeted therapies can address ultra-rare neurological conditions.