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SICKLE CELL DISEASE | FDA Grants Orphan Drug Designation to AND017

The U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug Designation (ODD) to AND017 for the treatment of sickle cell disease (SCD).

Announced by KIND Pharmaceuticals on October 25, 2024, ODD is granted to a drug or biological product to prevent, diagnose, or treat a rare disease or condition that affects fewer than 200,000 people in the United States. SCD affects nearly 120,000 patients in the US and more than 8 million globally.

“Hydroxyurea and L-glutamine are the limited FDA-approved oral treatments for SCD, AND017 from Kind Pharmaceutical might not only provide a novel oral treatment with a unique mechanism of action but also an obviously better safety and efficacy profile,” said Professor Gang Huang, the Kathryn Mays Johnson Distinguished Chair in Oncology at UT Health San Antonio.

AND017 is an innovative hemoglobin-elevating agent designed to target multiple stages of the red blood cell (RBC) life cycle. It is currently being developed to treat various types of anaemia, including anaemias associated with non-dialysis-dependent chronic kidney disease (NDD-CKD) and dialysis-dependent chronic kidney disease (DD-CKD), as well as cancer-related anaemia, myelodysplastic syndromes (MDS) anaemia, sickle cell disease (SCD), and β-thalassaemia.

The results from AND017’s Phase 1 trials in healthy subjects and Phase 2 clinical trials targeting anaemia in both non-dialysis-dependent chronic kidney disease (NDD-CKD) and dialysis-dependent chronic kidney disease (DD-CKD) were showcased at the 2024 American Society of Nephrology (ASN) Kidney Week. Additionally, preclinical data highlighting the potential of AND017 in treating sickle cell disease (SCD) is scheduled to be presented at an upcoming scientific meeting and published in a scientific journal.

“The FDA’s granting of ODD for AND017 underscores the urgent medical need for new therapies, particularly oral drugs to safely and effectively treat patients with SCD,” said Dong Liu, PhD, Founder, Chairman, and CEO of Kind Pharmaceutical.

Source: PR Newswire

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