RARE Daily

FDA Grants Fast Track Designation for Vigil’s VGL101 for the Treatment of Patients with ALSP

November 2, 2022

Rare Daily Staff

The U.S. Food and Drug Administration has granted Fast Track designation to Vigil Neuroscience’s VGL101 for the treatment of patients with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP).

VGL101, Vigil’s lead product candidate, is a fully human monoclonal antibody targeting human triggering receptor expressed on myeloid cells 2 (TREM2), which is responsible for maintaining microglial cell function. TREM2 deficiency is believed to be a driver of certain neurodegenerative diseases. It is currently being evaluated in a phase 1 trial in healthy volunteers. Vigil is on track to report phase 1 topline data and to initiate a phase 2 proof-of-concept trial for VGL101 in ALSP before the end of 2022.

Fast Track designation is designed to facilitate development and expedite the review of therapies for serious conditions, such as ALSP, and fill an unmet medical need. Programs with Fast Track designation may benefit from early and frequent communications with the FDA as well as the potential for priority review and a rolling submission of the marketing application.

Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) is a rare, progressive neurological disease that causes brain tissue known as white matter to waste away (leukodystrophy), forming lesions in certain brain areas due to disease-causing variants in the CSF1R (colony-stimulating factor-1 receptor) gene. ALSP is one type of leukodystrophy disorder, estimated by some studies to account for 10 to 25 percent of adult-onset leukodystrophies. Lesions of this white matter lead to major changes in personality, cognition, and muscle function, eventually causing people with this disorder to develop dementia and later decline into a vegetative state. Aside from the presence of a specific gene variant, the brains of people with ALSP show characteristic microscopic changes and patterns of atrophy on brain imaging that distinguish ALSP patients from those with other neurological conditions. Symptoms of ALSP overlap with frontotemporal dementia and other disorders associated with dementia such as Alzheimer disease as well as other neurological disorders such as Parkinson’s disease, multiple sclerosis, schizophrenia, and several others, making diagnosis difficult unless genetic testing is done. Symptoms can vary considerably from one person with ALSP to the next (even in the same family).

“ALSP is a devastating disease that affects an estimated 10,000 people in the United States with no approved treatments. We believe this Fast Track designation by the FDA recognizes the significant unmet need of ALSP patients and the therapeutic potential of VGL101,” said Ivana Magovčević-Liebisch, president and CEO of Vigil.

Photo: Ivana Magovčević-Liebisch, president and CEO of Vigil Neuroscience

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