BASEL, Switzerland, Nov. 1, 2016 /PRNewswire/ -- Enzyvant Sciences, a biopharmaceutical company focused on developing new therapies for patients with rare diseases, has announced the appointment of Dr. Alvin Shih as Chief Executive Officer. Dr. Shih previously served as Executive Vice President and Head of Research and Development at Retrophin, Inc. where he directed the development of therapeutics for a number of rare diseases. Prior to joining Retrophin he was the Chief Operating Officer of the Rare Disease Research Unit at Pfizer Inc. A former healthcare consultant at McKinsey & Company and L.E.K. Consulting, LLC, Dr. Shih holds a BA from Vanderbilt University, an MBA from Northwestern University, and an MD from the University of Alabama School of Medicine. He completed his residency training in internal medicine at the Massachusetts General Hospital. "I look forward to building Enzyvant into the premier developer of novel and transformational treatments for patients with rare diseases," said Dr. Shih. "Enzyvant aspires to make a difference in the lives of patients and families affected by rare diseases that currently lack effective treatment options." Enzyvant was formed earlier this year to advance the development of recombinant human acid ceramidase (rhAC), an enzyme replacement therapy for the treatment of patients with Farber disease. In addition to the rhAC program, Enzyvant is actively pursuing opportunities to augment its pipeline to address additional rare diseases. About Farber diseaseFarber disease is a lysosomal storage disease caused by a mutation in both alleles of the ASAH1 gene, resulting in the deficiency of the lysosomal enzyme acid ceramidase. This leads to the accumulation of the pro-inflammatory sphingolipid ceramide, and a macrophage-driven inflammatory process causing the development of the typical clinical symptoms. Like many other lysosomal storage diseases, Farber disease is a systemic disorder with a broad phenotypic spectrum and is likely underdiagnosed. There are no currently approved therapies for the treatment of Farber disease. Farber patients typically present with the cardinal symptoms of:
Joint contractures or arthritis
Weak or hoarse voice
It may take years for all three cardinal symptoms to appear together, and they may vary greatly in severity. Patients may also present with systemic inflammation (fever), severe pain, peripheral osteolysis, failure to thrive, and developmental delay.