Inozyme Pharma, Inc. announced that the FDA granted Fast Track designation to INZ-701 for the treatment of ABCC6 Deficiency.
“Through Fast Track designation, the FDA recognizes the potential of INZ-701 in ABCC6 Deficiency. We plan to work closely with the agency to establish an efficient path to approval. Receipt of Fast Track designation underscores our belief that INZ-701 could serve as an important therapy for patients living with ABCC6 Deficiency, notably for pediatric patients in whom this condition increases the risk of major clinical events such as stroke and severe neurological and cardiovascular disease,” said Douglas A. Treco, Ph.D., CEO and Chairman of Inozyme Pharma. “We look forward to presenting our development plans to regulatory agencies and reaching agreement on a pivotal study in pediatric patients with ABCC6 Deficiency by year-end 2024.”
Fast Track is an FDA program designed to facilitate and expedite the development and review of new medicines that are intended to treat or prevent serious conditions and have the potential to address an unmet medical need. The designation has been granted based on nonclinical pharmacology data and preliminary safety and efficacy data from the Company’s ongoing Phase 1/2 trial of INZ-701 in adults with ABCC6 Deficiency. With Fast Track designation, the development of INZ-701 can benefit from more frequent engagement with the FDA and expedited regulatory review.
In April 2024, the Company announced positive topline safety and immunogenicity data from its ongoing Phase 1/2 trial of INZ-701 in adults with ABCC6 Deficiency. Clinical improvements were observed in vascular pathology, visual function, and patient reported outcomes (PROs). The Company also reported initial findings from natural history studies which indicate a substantial disease burden among pediatric patients with ABCC6 Deficiency, manifesting as a high incidence of major clinical events, notably stroke, severe neurological disease, and severe cardiovascular disease, occurring early in life.
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