BASEL, Switzerland, Nov. 1, 2016 /PRNewswire/ -- Enzyvant Sciences, a biopharmaceutical company focused on developing newtherapies 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
"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:
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.
In addition to Farber disease, a mutation in the ASAH1 gene and the resulting acid ceramidase deficiency may also lead to a severe neurological disorder called spinal muscular atrophy with progressive myoclonic epilepsy (SMA-PME) that causes muscle weakness and wasting (atrophy) and a combination of seizures and uncontrollable muscle jerks (myoclonic epilepsy).
About rhACRecombinant human acid ceramidase (rhAC) is an enzyme replacement therapy (ERT) under development for the treatment of Farber disease. Initially developed by Dr. Edward Schuchman at the Icahn School of Medicine at Mount Sinai, rhAC showed positive results in various preclinical studies. rhAC was granted orphan drug designation by the U.S. Food and Drug Administration. Orphan drug status confers seven years of marketing exclusivity if approved by the FDA for the designated indication. Prior to FDA approval, orphan designation provides incentives for sponsors including tax credits for clinical research expenses, as well as exemption from FDA user fees.
About Enzyvant Sciences Enzyvant Sciences GmbH is a biopharmaceutical company focused on treatments for patients with rare diseases. It is currently conducting the pre-clinical studies for rhAC to enable a clinical trial in patients with Farber disease.
For more information, visit www.enzyvant.com.
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SOURCE Enzyvant Sciences
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