In a groundbreaking development for rare disease therapeutics, Switzerland-based biotech startup Enzyvant has secured unprecedented dual fast-track designations from the FDA for its investigational therapy RVT-802, targeting complete DiGeorge Syndrome (cDGS).
The company has made history by becoming the first to receive simultaneous Breakthrough Therapy designation and Regenerative Medicine Advanced Therapy (RMAT) designation, marking a significant milestone in the advancement of tissue-based therapies.
Novel Therapeutic Approach and Clinical Promise
RVT-802 represents an innovative biologic therapy that utilizes proprietary processes to harvest, culture, and apply allogeneic thymic tissue for treating primary immune deficiency resulting from complete DiGeorge Syndrome. The therapy, invented by Dr. M. Louise Markert, Professor of Pediatrics at Duke University, has demonstrated remarkable preliminary clinical results, achieving a survival rate exceeding 70% in treated infants.
Regulatory Fast-Track Significance
The dual designation provides Enzyvant with enhanced access to the FDA's Office of Tissues and Advanced Therapies, facilitating development guidance and expedited review. The RMAT designation, established under the 21st Century Cures Act in December 2016, specifically targets promising cell and tissue engineered products addressing serious conditions with unmet medical needs.
Development Timeline and Corporate Background
Enzyvant anticipates submitting a Biologics License Application (BLA) in the first half of 2018. This rapid progress is particularly noteworthy given the company's recent establishment under the Roivant Sciences umbrella, led by Vivek Ramaswamy.
"This is an important moment for our team, and most significantly for families confronting this terrible disease," stated Ramaswamy, highlighting the potential impact of RVT-802 on patient communities.
Dr. Alvin Shih, Enzyvant's CEO, emphasized their commitment to patient access: "We look forward to working very closely with the FDA to make our therapeutic candidate, RVT-802, available to cDGS patients as swiftly and responsibly as possible."
Pipeline Expansion
Beyond RVT-802, Enzyvant is advancing RVT-801, an enzyme replacement therapy for acid ceramidase deficiency (ACD), which manifests as Farber disease. This development further demonstrates the company's dedication to addressing rare diseases with significant unmet medical needs.
