Enzyvant to Develop Novel Biologic Therapy for Complete DiGeorge Syndrome
January 6 , 2017 | | Enzyvant
– RVT-802 is a biologic therapy for complete DiGeorge Syndrome, a fatal pediatric disease affecting roughly 10-20 children per year in the United States
– RVT-802 has been granted orphan drug designation by the FDA
– Enzyvant anticipates a potential BLA filing in 2018
– Enzyvant’s pipeline also includes RVT-801, an investigational enzyme replacement therapy being developed for the treatment of acid ceramidase deficiency
BASEL, Switzerland, January 6, 2017 /PRNewswire/ — Enzyvant, a biopharmaceutical company focused on developing innovative treatments for patients with rare diseases, today announced entering into an exclusive worldwide licensing agreement with Duke University to develop RVT-802, a tissue-based therapy for the treatment of complete DiGeorge Syndrome (cDGS), a rare and fatal congenital immunodeficiency disease. In the absence of treatment, children born with cDGS typically succumb to infections within the first two years of life due to their severe immunodeficiency.
“Patients with complete DiGeorge Syndrome lack effective treatment options, but this novel program offers the hope of a life-saving solution,” said Chief Executive Officer Dr. Alvin Shih. “It is our responsibility to ensure that the children with this disease who need this treatment are able to receive it.”
Under the terms of the agreement, data from Duke’s experience will be used to support submission of a Biologics License Application (BLA) with the Food and Drug Administration (FDA) for approval. RVT-802 expands Enzyvant’s pipeline of potentially transformative biologic therapies for rare genetic diseases with high unmet need.
Dr. M. Louise Markert, Professor of Pediatrics at Duke University, has led research on the treatment of immunodeficiency in patients with cDGS. The findings of Dr. Markert and her research team have been published in the New England Journal of Medicine as well as numerous other peer-reviewed scientific journals and clinical publications. “This is an encouraging development for the patients and parents confronting this disease,” said Dr. Markert. “I look forward to working with Enzyvant to make this life-saving therapy more available to babies who so desperately need it.”
About Complete DiGeorge Syndrome
Complete DiGeorge Syndrome (cDGS) represents approximately one percent of all patients with DiGeorge Syndrome, a congenital immunodeficiency disease. 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 a biologic therapy that was initially developed by Dr. Markert and her colleagues using proprietary processes to harvest, culture, and apply allogeneic thymic tissue for the treatment of primary immune deficiency resulting from cDGS. Preliminary clinical results suggest a survival rate of over 70% for treated patients. RVT-802 has been granted orphan drug designation by the FDA and Enzyvant anticipates a potential BLA filing in 2018.
Enzyvant is a biopharmaceutical company focused on developing innovative treatments for patients with rare diseases. Enzyvant is currently preparing a clinical trial of RVT-801, an investigational enzyme replacement therapy for the treatment of acid ceramidase deficiency (ACD), a rare lysosomal storage disease which manifests as Farber disease. Enzyvant is also planning to advance the development of RVT-802, an investigational biologic therapy for complete DiGeorge Syndrome. Enzyvant plans to develop treatments for additional rare diseases with high unmet need.