Complement Therapeutics has announced that CTx001, its gene therapy treatment for geographic atrophy (GA) secondary to age-related macular degeneration (AMD), has received Fast Track designation from the US Food and Drug Administration (FDA).
The designation follows the FDA’s clearance of an Investigational New Drug (IND) application for CTx001.
GA is an advanced form of dry AMD, caused by the progressive degeneration of photoreceptors, retinal pigment epithelium and choriocapillaris. Over time, GA causes irreversible loss of vision. It is a leading cause of blindness in elderly populations, affecting more than five million people worldwide. There is a serious unmet medical need for treatments for this condition, with few treatment options available.
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The efficacy of CTx001, a novel adeno-associated virus (AAV)-based gene therapy, in GA will be researched in the phase 1/2 Opti-GAIN clinical trial. Initial patient dosing in the trial is expected to begin in the US in 2026.
Fast Track designation is designed to speed up the FDA review process for drugs that may address unmet medical needs for serious diseases.
“FDA Fast Track designation for CTx001 is an important regulatory milestone recognising the seriousness of GA, the unmet medical need faced by patients, as well as the innovative potential for CTx001 to meaningfully address this need,” said Dr Rafiq Hasan, CEO of Complement. “Together with the recent IND clearance, this designation supports our efforts to efficiently advance CTx001 into clinical development as we work to address a leading cause of irreversible vision loss.”
Complement is a clinical-stage biotechnology company specialising in treatments for complement-mediated diseases.
