Sirtris' resveratrol gets US OD status for MELAS

The USA's Sirtris Pharmaceuticals says that the US Food and Drug Administration has granted the company orphan drug designation for resveratrol in the treatment of MELAS syndrome (mitochondrial myopathy, encephalopathy, lactic acidosis, and stroke-like episodes).

"MELAS can have a devastating effect on the quality of life of patients and their families," said Patrick Chinnery of the UK's Newcastle University, who is leading a MELAS Phase Ib clinical trial with SRT501, Sirtris' proprietary formulation of resveratrol. "Many diseases of aging, such as type 2 diabetes, exhibit impaired mitochondrial function," noted Peter Elliott, Sirtris' senior vice president of development. "As we target SIRT1, a gene tied to the aging process and improved mitochondrial function, we hope to develop new therapies for diseases like MELAS and type 2 diabetes," he added.

MELAS is a progressive and fatal disorder with no known treatments. The earliest symptoms include muscle weakness, fatigue, recurrent headaches and seizures. The reported age of onset varies between three and 40 years, with most patients presenting between the ages of five to 15. The syndrome can manifest as stroke-like episodes in patients under 20 years of age.