AND017 Receives FDA Orphan Drug Designation for Sickle Cell Disease

Credit: US Food and Drug Administration

The US 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).¹

Announced by KIND Pharmaceutical 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.¹

Black and African American patients are disproportionately affected by SCD, comprising more than 98% of the population living with the disease.² Hemoglobin molecules in red blood cells (RBCs) of patients with SCD can sickle in the bloodstream, leading to hemolysis and occlusion of capillaries. Hallmark signs of SCD are hemolytic anemia and vaso-occlusive crises (VOCs), potentially leading to multi-organ failure and premature mortality.

According to KIND Pharmaceutical, AND017 is a first-in-class hemoglobin elevating agent targeting multiple stages of the RBC life cycle.¹ AND017 is under development to treat a variety of anemia indications anemia, including those associated with non-dialysis-dependent chronic kidney disease (NDD-CKD) and dialysis-dependent chronic kidney disease (DD-CKD), as well as cancer-related anemia, myelodysplastic syndromes (MDS) anemia, SCD, and β-thalassemia.

Results from AND017's Phase 1 in healthy subjects and Phase 2 clinical trials in the treatment of anemia in NDD-CKD and anemia in DD-CKD are being presented at the 2024 American Society of Nephrology (ASN) Kidney Week.³ Preclinical data supporting the role of AND017 in SCD is planned to be presented at a future scientific meeting and published in a scientific journal.¹

"I am eager to see how a compound with such unbelievable preclinical safety and efficacy data will translate to real-world SCD patients,” Huang added.¹

The FDA’s ODD qualifies KIND Pharmaceutical for incentives, including tax credits for qualified clinical trials, exemption from user fees, and potential seven years of market exclusivity after AND017's approval for the treatment of SCD.

"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.¹ "The granting of ODD also demonstrates the innovation capability and the vision of Kind Pharmaceutical."

References

1. _{PRNewswire. KIND Announces FDA Granted Orphan Drug Designation (ODD) for AND017 in the Treatment of Sickle Cell Disease (SCD). October 25, 2024. Accessed October 25, 2024. https://prnmedia.prnewswire.com/news-releases/kind-announces-fda-granted-orphan-drug-designation-odd-for-and017-in-the-treatment-of-sickle-cell-disease-scd-302287483.html}
2. _{Mangla A, Ehsan M, Agarwal N, et al. Sickle Cell Anemia. [Updated 2023 Sep 4]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK482164/}
3. _{PRNewswire. Kind announces late-breaking abstract accepted for presentation on and017 clinical trials to treat anemia in chronic kidney disease (CKD) at ASN - Kidney Week 2024. October 17, 2024. Accessed October 25, 2024. https://www.prnewswire.com/news-releases/kind-announces-late-breaking-abstract-accepted-for-presentation-on-and017-clinical-trials-to-treat-anemia-in-chronic-kidney-disease-ckd-at-asn--kidney-week-2024-302279020.html.}