CAMBRIDGE, Mass. and BASEL, Switzerland, April 3, 2019 /PRNewswire/ — Enzyvant today announced that its investigational regenerative treatment (RVT-802) in late-stage development for congenital athymia will be featured during a platform presentation, at a symposium, and in a poster session at the Clinical Immunology Society (CIS) 2019 annual meeting in Atlanta, GA, April 4-7.
Congenital athymia is a condition where there is a complete lack of a functional thymus, an essential part of the immune system, which leads to severe immunodeficiency. Approximately 20 infants are born with congenital athymia in the U.S. each year. In the absence of treatment, children born with congenital athymia have no ability to fight infections and typically die within the first 24 months of life. Currently, there are no FDA-approved treatments for congenital athymia.
RVT-802, an investigational tissue-based treatment pioneered by Duke University’s M. Louise Markert, M.D., Ph.D., and licensed by Enzyvant in 2016, is designed to regenerate the immune system for the treatment of T cell immunodeficiency resulting from congenital athymia. Dr. Markert’s leading work with RVT-802 has enabled an overall survival of 72 percent among 85 patients with congenital athymia treated at Duke.
“RVT-802 represents a potentially life-saving treatment option for congenital athymia patients,” said Rachelle Jacques, CEO of Enzyvant. “We are proud to have advanced the transformative work of Dr. Markert from an experimental procedure to a potential FDA-approved therapy.”
Dr. Markert will present evidence and insights from her team’s experiences with patients treated with RVT-802 at CIS on Wednesday, April 3 at an Enzyvant-sponsored symposium titled “Thymic Defects: From Biology to Treatment.”
The platform presentation, on Thursday, April 4 by co-author Tyler Yates, M.D., also of Duke University, focuses on the success of RVT-802 in leading to the generation of naïve T cells in athymic children who were first unsuccessfully treated with hematopoietic stem cell transplants.
On Friday, April 5, poster co-author Stephanie E. Gupton, RN and CPNP, a Duke pediatric nurse practitioner, will discuss that with the advent of widespread newborn screening for primary immunodeficiency there has been an increase in the neonatal diagnosis of athymia, escalating the need for “clear, concise guidelines for the care of these patients” receiving treatment with RVT-802.
“We’ve learned a lot from caring for these children, with some of them now living into adolescence and beyond,” said Dr. Markert, Professor of Pediatrics and Immunology at Duke University School of Medicine. “As we potentially move toward an approved treatment that can be more widely available, we want to ensure that physicians have a better understanding of RVT-802 for treatment of patients with congenital athymia.”
Enzyvant Expanding and Enhancing RVT-802 Manufacturing
Enzyvant expects to complete the submission of its rolling Biologics License Application (BLA) for RVT-802 to the FDA in the first half of 2019. As part of this preparation, Enzyvant has partnered with Duke and invested significantly to transition the RVT-802 manufacturing process from a developmental process to a rigorous cGMP program that meets FDA requirements for commercial products. This is a critical component of the BLA submission and required for FDA approval.
More about RVT-802
RVT-802 has been granted Breakthrough Therapy designation, Rare Pediatric Disease designation, Orphan Drug designation, and Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA. RVT-802 was the first program to be granted both Breakthrough Therapy and RMAT designations by the FDA. RMAT was established to expedite the development and FDA review of new cell therapies and tissue engineering products that treat serious and life-threatening conditions. RVT-802 has the potential to become the first FDA-approved therapy with an RMAT designation.
Enzyvant, a wholly-owned subsidiary of Roivant Sciences, is a biopharmaceutical company focused on developing transformative therapies for people with rare diseases. Enzyvant leverages the Roivant platform to develop therapies that address high unmet medical needs while driving greater efficiency in research, clinical development, and commercialization. Enzyvant is currently in the process of completing a rolling Biologics License Application submission with the FDA for RVT-802, an investigational tissue-based therapy for the treatment of primary immunodeficiency associated with congenital athymia. Enzyvant is also preparing to initiate a clinical trial of RVT-801, an investigational enzyme replacement therapy for the treatment of Farber disease. For more information, please visit www.enzyvant.com.
About Roivant Sciences
Roivant aims to improve health by rapidly delivering innovative medicines and technologies to patients. We do this by building Vants – nimble, entrepreneurial biotech and healthcare technology companies with a unique approach to sourcing talent, aligning incentives, and deploying technology to drive greater efficiency in R&D and commercialization.
For more information, please visit www.roivant.com.