The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AND017, a drug developed by KIND Pharmaceutical, for the treatment of sickle cell disease (SCD). This designation, announced on October 25, 2024, underscores the urgent need for new therapies for this rare condition affecting nearly 120,000 patients in the U.S. and over 8 million globally.
Addressing Unmet Needs in SCD Treatment
Currently, only hydroxyurea and L-glutamine are the FDA-approved oral treatments for SCD. According to Professor Gang Huang at UT Health San Antonio, AND017 has the potential to offer a novel oral treatment with a unique mechanism of action and an improved safety and efficacy profile. Black and African American patients are disproportionately affected by SCD, representing more than 98% of the affected population.
In SCD, hemoglobin molecules in red blood cells (RBCs) can sickle, leading to hemolysis and occlusion of capillaries. This results in hemolytic anemia and vaso-occlusive crises (VOCs), which can cause multi-organ failure and premature mortality.
AND017: A Novel Approach to Anemia Treatment
AND017 is described by KIND Pharmaceutical as a first-in-class hemoglobin elevating agent that targets multiple stages of the RBC life cycle. It is under development for treating various anemia indications, including those associated with non-dialysis-dependent chronic kidney disease (NDD-CKD), dialysis-dependent chronic kidney disease (DD-CKD), cancer-related anemia, myelodysplastic syndromes (MDS) anemia, SCD, and β-thalassemia.
Clinical Development and Future Plans
Results from AND017's Phase 1 trial in healthy subjects and Phase 2 clinical trials in treating anemia in NDD-CKD and anemia in DD-CKD are being presented at the 2024 American Society of Nephrology (ASN) Kidney Week. Preclinical data supporting AND017's role in SCD is slated for presentation at a future scientific meeting and publication in a scientific journal.
Regulatory and Market Incentives
The FDA's ODD provides KIND Pharmaceutical with several incentives, including tax credits for qualified clinical trials, exemption from user fees, and potential seven years of market exclusivity upon AND017's approval for SCD treatment. Dong Liu, PhD, founder, chairman, and CEO of Kind Pharmaceutical, emphasized that this designation highlights the critical need for innovative therapies and the company's commitment to addressing this need.
