Avid Bioservices Selected as Commercial Manufacturer for Critical Enzyme Replacement Therapy by Enzyvant, a Subsidiary of Roivant Sciences - Seite 2

In winning this manufacturing project, Avid Bioservices was selected by Enzyvant from a large field of internationally recognized contract development and manufacturing organizations.

About Farber Disease
Farber disease is a rare lysosomal storage disease caused by mutations in the ASAH1 gene, resulting in deficiency of the lysosomal enzyme acid ceramidase. This deficiency leads to the accumulation of the pro-inflammatory sphingolipid ceramide, and a macrophage-driven inflammatory process causing the development of typical clinical symptoms.

Farber patients typically present with the cardinal symptoms of:

• Joint contractures or arthritis
• Subcutaneous nodules
• Weak or hoarse voice

Patients may also present with systemic inflammation (including fever), severe pain, peripheral osteolysis, failure to thrive, and developmental delay. Like other lysosomal storage diseases, Farber disease has a broad phenotypic spectrum and is likely underdiagnosed.

About RVT-801
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. Enzyvant is currently conducting preclinical studies to enable a clinical trial of rhAC in patients with Farber disease. Orphan drug designation for RVT-801 has been granted by regulatory agencies in the United States and the European Union.

About Enzyvant
Enzyvant is a biopharmaceutical company focused on developing innovative treatments for patients with rare diseases. Enzyvant is conducting 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 is also advancing the development of RVT-802, an investigational tissue-based biologic therapy for the potential treatment of primary immune deficiency associated with complete DiGeorge Syndrome. RVT-802 has been granted orphan drug designation, Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, and pediatric rare disease designation by the U.S. Food and Drug Administration. Enzyvant anticipates a potential BLA filing for RVT-802 later this year. Enzyvant plans to develop treatments for additional rare diseases with high unmet need. For more information, please visit www.enzyvant.com.