First drug to treat Alexander disease approved by the FDA after three decades of research
The Food and Drug Administration recently approved the first drug to treat Alexander disease, a rare, progressive and often fatal neurological disorder that can affect motor, cognitive and autonomic functions like heart rate and breathing. The drug, Zanvastro, is the first and only disease-modifying treatment for Alexander disease. Until now, treatment of the disease has been limited to symptom…
The Food and Drug Administration has officially approved Zanvastro, the first drug to treat Alexander disease, after a lengthy 30-year research process. This revolutionary medication marks the first disease-modifying treatment for this rare, progressive neurological disorder, which mainly affects motor, cognitive, and autonomic functions.
Prior to this, treatment options were limited to managing symptoms. Zanvastro specifically targets the GFAP protein, a key driver of the disease, and was developed by Ionis Pharmaceuticals in collaboration with researchers at the University of Wisconsin–Madison and Waisman Center. Clinical trials revealed that individuals treated with Zanvastro experienced stabilization in motor function, and in some cases, even improvements.
The drug is administered through intrathecal injections every three months. Approval came after over three decades of work, beginning with Albee Messing's discovery of GFAP genetic mutations as the root cause of Alexander disease in the late 1990s. The drug's approval represents a significant milestone for the Alexander disease community, and its availability is expected in the United States in the coming weeks, with other countries following suit through licensing agreements.
Written by urgent.news from Medical Xpress's reporting — not their text. Machine-written — may contain errors; check the original before relying on it.
