Updated: Dec 6, 2022
"Second Orphan Drug Designation Supports Azafaros’ Strategy to Develop AZ-3102 as a Disease Modifying Treatment in a Range of Severe Rare Inherited Metabolic Disorders"
Read the full article here: Article Link
Announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for AZ-3102
Based on its mode of action, AZ-3102 has broad applicability in addressing these inherited metabolic disorders.
"As we explore the broad potential of AZ-3102 as a promising new treatment option for rare disease patients, we are excited to achieve this further validation from the FDA,” said Stefano Portolano, Chief Executive Officer of Azafaros. “Our orally available azasugar is designed to selectively inhibit two enzymes involved in glycolipid metabolism with the goal of reducing toxic glycolipid accumulation and ameliorate impaired lysosomal function. We appreciate this acknowledgement of our mechanism of action and the urgency to support the rare inherited metabolic disorder patient community with safe and effective therapies.”