Rolling BLA submission has been agreed to by FDA and is intended to expedite the Agency’s review process
Completion of rolling BLA submission anticipated by end of 2018
RVT-802 has been awarded Breakthrough Therapy and RMAT designations, as well as a pediatric rare disease and orphan designations
Enzyvant to present at Roivant Pipeline Day on July 10
BASEL, Switzerland, July 9, 2018 /PRNewswire/ — Enzyvant, a biopharmaceutical company focused on developing innovative treatments for patients with rare diseases, announced today that it has initiated its rolling submission of a Biologics License Application (BLA) for RVT-802 to the U.S. Food and Drug Administration (FDA). The company expects to complete its BLA submission by the end of 2018.
RVT-802 is an investigational, tissue-based regenerative therapy designed to treat the primary immune deficiency resulting from congenital athymia associated with complete DiGeorge Anomaly (cDGA). RVT-802 is designed to be administered only once over the lifetime of the patient. cDGA is uniformly fatal if untreated, with death typically occurring in the first 24 months of life due to susceptibility to infection.
RVT-802 has been granted Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy (RMAT) designation, rare pediatric disease designation, and orphan drug designation by the FDA. As part of the FDA’s commitment to expedite therapeutics that aim to address high unmet medical needs, the Agency has agreed to a rolling BLA submission process for RVT-802.
“We are very pleased to initiate the RVT-802 BLA submission. This is a milestone not only for Enzyvant, but more importantly for infants with cDGA and their families,” said Dr. Alvin Shih, Chief Executive Officer of Enzyvant. “I am grateful to the entire team who have worked tirelessly to advance this innovative program. We will continue to press forward with the goal of completing the BLA in 2018 to enable a thorough and timely review by the Agency.”
Dr. Shih will be presenting at Roivant Pipeline Day in New York City tomorrow, July 10, at 5:00pm ET.
“I am proud of Enzyvant’s dedication to improving the lives of patients suffering from this fatal condition,” said Vivek Ramaswamy, Founder and Chief Executive Officer of Roivant Sciences. “The ultimate metric of Roivant’s success will be the delivery of truly innovative therapies to patients suffering from serious diseases. Today’s announcement marks an important milestone in our company’s history.”
About Complete DiGeorge Anomaly
Complete DiGeorge Anomaly (cDGA) is a rare disease affecting approximately 1 in 300,000 infants, or roughly 10-20 infants born each year in the United States. Children with cDGA are born without a thymus gland, resulting in severe immunodeficiency due to the inability to produce normally functioning T cells, which defend against infection and regulate essential processes in the immune system. cDGA is uniformly fatal if untreated, with death typically occurring in the first 24 months of life due to susceptibility to infection.
RVT-802 is an investigational tissue-based regenerative therapy designed to treat primary immune deficiency resulting from congenital athymia associated with cDGA. RVT-802 is investigational and not approved for commercial use by the FDA or other health regulators at this time.
Enzyvant is a biopharmaceutical company focused on developing innovative treatments for patients with rare diseases. Enzyvant is collaborating with Duke University to advance the development of RVT-802, an investigational tissue-based therapy for the treatment of primary immunodeficiency associated with complete DiGeorge Anomaly.
Enzyvant is simultaneously preparing to initiate a clinical trial of RVT-801, an investigational enzyme replacement therapy for the treatment of Farber disease. Enzyvant plans to develop treatments for additional rare diseases with high unmet need.
For more information, please visit www.enzyvant.com.
Roivant Sciences is a global biopharmaceutical company focused on reducing the time and cost of the drug development process to improve the lives of patients and their families. Roivant partners with innovative companies and academic institutions to ensure that important medicines are rapidly developed and delivered to patients upon approval.
The Roivant family of companies includes Myovant (women’s health), Axovant (neurology), Urovant (urology), Enzyvant (rare diseases), Dermavant (dermatology), Genevant (RNA therapeutics), Metavant (cardiometabolic diseases), Altavant (next-generation drug development), Datavant (healthcare data), and Arbutus (hepatitis B).
Today there are 25 investigational drugs in 11 therapeutic areas being tested in over 50 clinical trials across the Roivant family of companies.
For more information, please visit www.roivant.com.
About Roivant Pipeline Day
Roivant Pipeline Day will be held on Tuesday, July 10, 2018 in New York City. The event will feature presentations, fireside chats, and Q&A sessions from executives across the Roivant family of companies. The event is scheduled to begin at 2:00 p.m. ET and will continue until approximately 5:30 p.m. ET. Due to limited capacity, attendance is by invitation only but a live webcast will be available to interested parties. To request access to the webcast or to learn more about the event, please email [email protected].