Enzyvant Receives FDA Rare Pediatric Disease Designation for Investigational Therapy RVT-802
September 5 , 2017 | | Enzyvant
– RVT-802 is an investigational tissue-based therapy for primary immune deficiency associated with complete DiGeorge Syndrome
– RVT-802 has also received Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, and orphan drug designation from FDA
– Enzyvant anticipates potential BLA filing in the first half of 2018
– Morgan Molloy to join Enzyvant as Chief Commercial Officer
BASEL, Switzerland, September 5, 2017 / PRNewswire / — Enzyvant, a biopharmaceutical company focused on developing innovative treatments for patients with rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to RVT-802, Enzyvant’s investigational tissue-based therapy for the treatment of primary immune deficiency resulting from congenital athymia associated with complete DiGeorge Syndrome.
The FDA grants Rare Pediatric Disease designation to therapeutics intended to treat serious or life-threatening rare diseases which primarily affect individuals under the age of 18. With this designation, RVT-802 will be eligible to receive a rare pediatric disease priority review voucher upon BLA approval. RVT-802 has previously received Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, and orphan drug designation from the FDA.
Enzyvant also announced the appointment of Morgan Molloy as the company’s Chief Commercial Officer. Mr. Molloy comes to Enzyvant with more than 25 years of broad commercial leadership experience in the biopharmaceutical industry. Prior to Enzyvant, he served as the Head of U.S. Commercial for Spark Therapeutics and was responsible for the development of commercial strategy and implementation in the U.S. for Spark’s investigational gene therapy programs. Starting in 2007, Mr. Molloy spent seven years at Sanofi-Genzyme and held multiple commercial leadership roles in several of the legacy Genzyme franchises, including the Fabry disease business. Before Sanofi-Genzyme, he spent ten years at Biogen in multiple commercial leadership roles of increasing responsibility. Mr. Molloy started his career at Allergan and holds a B.S. in Business Management from the University of Massachusetts Lowell.
“I am delighted to join a dynamic company with such a promising pipeline of novel therapeutic candidates,” said Mr. Molloy. “Today’s news is further validation of the potential for RVT-802 to be a transformative therapy for children with complete DiGeorge Syndrome.”
“We are pleased to welcome Morgan to our growing team,” said Alvin Shih, MD, Chief Executive Officer of Enzyvant. “Morgan’s extensive experience in rare disease commercialization will help us achieve our mission of delivering therapeutics to rare disease patients. The Rare Pediatric Disease designation underscores the importance of this mission, and we look forward to continuing to work with the FDA to advance the development of RVT-802.”
About Complete DiGeorge Syndrome
Complete DiGeorge Syndrome (cDGS) represents approximately one percent of all patients with DiGeorge Syndrome, a congenital condition. The three main characteristics of cDGS are congenital heart disease, hypoparathyroidism (leading to low calcium levels), and athymia (lack of thymus tissue). Athymia leads to severe immunodeficiency due to the inability to produce normally functioning T cells, which defend against infection and regulate essential processes in the immune system. cDGS is uniformly fatal if untreated, with death typically occurring in the first two years of life due to susceptibility to infection.
RVT-802 is an investigational cell-based therapy using proprietary processes to harvest, culture, and apply allogeneic thymic tissue for the treatment of primary immune deficiency associated with cDGS. In addition to Rare Pediatric Disease designation, RVT-802 has also been granted Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, and orphan drug designation by the FDA. Enzyvant anticipates a potential BLA filing in the first half of 2018. 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. In addition to advancing the development of RVT-802, an investigational tissue-based therapy of the treatment of primary immune deficiency associated with complete DiGeorge Syndrome, Enzyvant is also in the process of initiating a natural history study of patients with Farber disease to better define the natural course of disease and the relationship between specific symptoms, biomarkers, and prognosis. Enzyvant is simultaneously preparing 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.