Kind Pharmaceutical, encompassing Hangzhou Andao Pharmaceutical Ltd. and Kind Pharmaceuticals LLC, has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for AND017, a drug intended for the treatment of Sickle Cell Disease (SCD). This designation highlights the potential of AND017 to address a critical unmet need in SCD therapy, particularly as an oral treatment option.
Clinical and Preclinical Data
AND017 is currently undergoing Phase 1 and Phase 2 clinical trials for the treatment of anemia in non-dialysis dependent chronic kidney disease (NDD-CKD) and dialysis-dependent chronic kidney disease (DD-CKD). Results from these trials were presented at the American Society of Nephrology (ASN) Kidney Week. Preclinical data supporting the ODD for SCD will be presented at a future scientific meeting and published in a scientific journal.
Mechanism of Action and Therapeutic Potential
AND017 is described as a first-in-class hemoglobin elevating agent (HbEA) designed to target multiple stages of the red blood cell (RBC) life cycle. It is being developed for various anemia indications, including those associated with chronic kidney disease, cancer, myelodysplastic syndromes, sickle cell disease, and β-thalassemia.
Expert Commentary
Prof. Gang Huang at UT Health San Antonio, an expert in hematology and blood diseases, noted the potential of AND017 as a novel oral treatment for SCD with a unique mechanism of action and a potentially better safety and efficacy profile compared to existing treatments like hydroxyurea and L-glutamine. "I am eager to see how a compound with such unbelievable preclinical safety and efficacy data will translate to real world SCD patients," said Prof. Huang.
Orphan Drug Designation Benefits
The FDA grants ODD to drugs or biological products intended to treat rare diseases or conditions affecting fewer than 200,000 people in the United States. This designation provides Kind Pharmaceutical with incentives such as tax credits for qualified clinical trials, exemption from user fees, and potential seven years of market exclusivity upon AND017's approval for SCD treatment.
Sickle Cell Disease Overview
Sickle Cell Disease affects approximately 120,000 patients in the US and over 8 million worldwide. It is a genetic disease characterized by a single amino acid mutation in the β-globin of adult hemoglobin, leading to the sickling of red blood cells, hemolytic anemia, and vaso-occlusive crises, ultimately causing multi-organ failure and premature death.
