FDA approves first drug for Alexander disease — ScienceAlert

In the United States, the Food and Drug Administration (FDA) has approved zilganersen, which will be sold under the name Zanvastro, for the treatment of the rare genetic Alexander disease. It is the first drug to directly target the mechanism of this neurological disease, which is often fatal, ScienceAlert reports.

How the drug works

Alexander disease affects fewer than one person per million worldwide. It is associated with a disruption in the gene responsible for producing glial fibrillary acidic protein, GFAP. Abnormal forms of this protein form aggregates known as Rosenthal fibers. They place stress on astrocytes, which is associated with damage to these cells and to myelin in the brain.

Zilganersen reduces GFAP production using antisense oligonucleotides, small fragments of DNA. The drug is administered by healthcare professionals through an injection into the spinal canal once every three months. The approval applies to patients of any age, although signs of the disease most often appear before the age of two.

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Trial results and adverse reactions

The FDA's decision was based in part on a clinical study involving 54 children and adults up to 53 years old. The trial was conducted at 13 sites in eight countries. In younger participants, motor skills stabilized or improved, while in older patients, walking speed remained constant compared with a decline among those who did not receive treatment.

During therapy lasting more than a year, the drug met the FDA's safety requirements. The most common adverse reactions were vomiting, back pain, cough, headache, and pain after the spinal injection. The FDA also warned about cases of aseptic meningitis, an inflammation of the membranes of the brain and spinal cord. The drug does not cure the disease, but it may slow its progression and improve symptoms.