BASEL, Switzerland and CAMBRIDGE, Mass., Feb. 4, 2019 /PRNewswire/ — Enzyvant, a biopharmaceutical company focused on developing transformative therapies for patients with rare diseases, today announced that abstracts highlighting the development of its investigational RVT-801 program and the natural history of Farber disease will be presented in Orlando, Florida, by Enzyvant and its collaborators at the 2019 WORLDSymposium™, an annual conference focused on lysosomal diseases.
Poster Presentations, all to be held in Regency Ballroom R:
- RVT-801, a developmental enzyme replacement therapy for Farber disease, ameliorates characteristic features of the disease phenotype in a Farber mouse model,P96, Tuesday, February 5, 4:30 to 6:30 p.m. ET
- The pro-inflammatory immunophenotype of a Farber disease mouse model is ameliorated by repeated dosing with RVT-801, a developmental enzyme replacement therapy for Farber disease,P313, Wednesday, February 6, 4:30 to 6:30 p.m. ET
- Farber disease (acid ceramidase deficiency): Data from an ongoing natural history study, P336, Wednesday, February 6, 4:30 to 6:30 p.m. ET
Enzyvant is also hosting a medical booth (Booth #620) with information on Farber disease as well as a corporate booth (Booth #421).
RVT-801 is a recombinant form of human acid ceramidase (rhAC) that Enzyvant is developing for potential use as an enzyme replacement therapy in acid ceramidase deficiency, manifesting as Farber disease. RVT-801 is currently in the preclinical phase of development. Orphan Drug Designation for RVT-801 has been granted by regulatory agencies in the United States and European Union. RVT-801 is the first and only disease-specific therapy for Farber disease in development.
About Farber Disease
Farber disease is a rare disease and thought to be significantly underdiagnosed, with many cases misdiagnosed as juvenile idiopathic arthritis (JIA). The disease is caused by mutations in the ASAH1 gene, resulting in deficiency of acid ceramidase, an important lysosomal enzyme. This deficiency leads to the accumulation of ceramide within cells, which has pro-inflammatory and pro-apoptotic effects.
Farber disease patients typically present with the cardinal symptoms of joint contractures or arthritis, subcutaneous nodules and/or a weak or hoarse voice. More severe symptoms may also include impaired cognitive development as well as damage to lungs, liver and bones (osteolysis). Disease onset is usually in early childhood but may occur later in life.
There is no disease-specific treatment for Farber disease, although hematopoietic stem cell transplant has been used in some patients. Anti-inflammatory medications may reduce pain and inflammation to some degree but do not prevent disease progression.
Enzyvant is a biopharmaceutical company focused on developing innovative treatments for people with rare diseases. Enzyvant has initiated a rolling Biologics License Application (BLA) submission with the FDA for RVT-802, an investigational tissue-based therapy for the treatment of primary immunodeficiency due to congenital athymia. Enzyvant is simultaneously advancing preclinical development of RVT-801, an investigational enzyme replacement therapy for the treatment of Farber disease.
For more information, please visit www.enzyvant.com