ALS001 Earns Orphan Drug Designation from the U.S FDA

Release Date: 11-Sep-2021

Coya Therapeutics, Inc. receives the orphan drug designation from US FDA for ALS001, an autologous, expanded Treg cell therapy for the treatment of amyotrophic lateral sclerosis (ALS). ALS is a progressive neurodegenerative disease initially affecting motor neurons in the cortex, brain stem and spinal cord. Its symptoms include loss of mobility, muscle control, speech and finally the ability to breathe.


The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S and it also gives several benefits, like tax credits for qualified clinical trials, potential seven years of market exclusivity after approval and exemption from user fees.

ALS001 has finished an ongoing Phase 2a trial with topline data estimated late summer 2021.


ALS pathogenesis contains of two stages: an early, slow progressive neurodegenerative stage, and further, a rapidly progressing neurotoxic stage associated with anti-inflammatory and pro-inflammatory immune responses. According to the ALS Association, around 5,000 people are diagnosed per year, and the average life expectancy is 2 to 5 years.

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