02/03/2022
Amolyt Pharma Announces European Commission Has Granted Orphan Drug Designation to AZP-3601 for the treatment of Hypoparathyroidism
Top-line data from ongoing clinical trial in patients with hypoparathyroidism expected mid-2022
LYON, France, and Cambridge, MA, February 28, 2022 - Amolyt Pharma, a global company specialized in developing therapeutic peptides for rare endocrine and related diseases, today announced that the European Commission has granted Orphan Drug Designation (ODD) to AZP3601 for the treatment of hypoparathyroidism. Amolyt previously received ODD from the U.S. Food and Drug Administration for AZP-3601 for the same condition in January 2021.
“We are very pleased to have been granted Orphan Drug Designation in both the U.S. and now the E.U.,something we believe reflects the need for new therapiesto treat hypoparathyroidism,” said Thierry Abribat, Ph.D., founder and chief executive officer of Amolyt Pharma. “This highlights the potential significant benefits that AZP-3601 may bring to patients versus currently approved therapies, including sustained serum calcium normalization for a full 24 hours, and reduced risk of hypercalciuria, a key contributing factor to chronic kidney disease.”
“Additionally, we believe AZP-3601’s unique mechanism of action may preserve bone integrity as demonstrated by the bone biomarker data from our Phase 1 clinical trial in healthy volunteers. Together, these clinical benefits are relevant for many patients with hypoparathyroidism, especially the 26% who have established chronic kidney disease and those at risk of developing kidney disease plus the 17% with osteopenia or osteoporosis. We are advancing AZP-3601 through an efficient clinical development plan and we look forward to announcing proof-ofconcept data in patients with hypoparathyroidism mid-year.”
Based on the Committee for Orphan Medicinal Products (COMP) positive opinion, the European Commission grants ODD to potential treatments for diseases that are life-threatening or chronically debilitating and have a prevalence of not more than 5 in 10,000. When granted, ODD carries a range of incentives for sponsors, including protocol assistance, reduced regulatory fees, access to a centralized marketing authorization, and ten years of market exclusivity.
LYON, France, and Cambridge, MA, February 28, 2022 - Amolyt Pharma, a global company specialized in developing therapeutic peptides for rare endocrine and related diseases, today announced that the European Commission has granted Orphan Drug Designation (ODD) to AZP3601 for the treatment of hypoparathyroidism. Amolyt previously received ODD from the U.S. Food and Drug Administration for AZP-3601 for the same condition in January 2021.
“We are very pleased to have been granted Orphan Drug Designation in both the U.S. and now the E.U.,something we believe reflects the need for new therapiesto treat hypoparathyroidism,” said Thierry Abribat, Ph.D., founder and chief executive officer of Amolyt Pharma. “This highlights the potential significant benefits that AZP-3601 may bring to patients versus currently approved therapies, including sustained serum calcium normalization for a full 24 hours, and reduced risk of hypercalciuria, a key contributing factor to chronic kidney disease.”
“Additionally, we believe AZP-3601’s unique mechanism of action may preserve bone integrity as demonstrated by the bone biomarker data from our Phase 1 clinical trial in healthy volunteers. Together, these clinical benefits are relevant for many patients with hypoparathyroidism, especially the 26% who have established chronic kidney disease and those at risk of developing kidney disease plus the 17% with osteopenia or osteoporosis. We are advancing AZP-3601 through an efficient clinical development plan and we look forward to announcing proof-ofconcept data in patients with hypoparathyroidism mid-year.”
Based on the Committee for Orphan Medicinal Products (COMP) positive opinion, the European Commission grants ODD to potential treatments for diseases that are life-threatening or chronically debilitating and have a prevalence of not more than 5 in 10,000. When granted, ODD carries a range of incentives for sponsors, including protocol assistance, reduced regulatory fees, access to a centralized marketing authorization, and ten years of market exclusivity.
