FDA Grants Orphan Drug Designation to Kind Pharmaceutical's AND017 for Sickle Cell Disease

Kind Pharmaceutical, a clinical-stage biopharmaceutical company focused on developing innovative medicines to treat hematological diseases and cancers, announced that 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). AND017 is being developed to treat various anemia indications associated with non-dialysis dependent chronic kidney disease (NDD-CKD) and dialysis-dependent chronic kidney disease (DD-CKD).

Publish Date: 25-10-2024 Source: Kind Pharmaceutical

Sickle cell disease (SCD) results from homozygous and compound heterozygote inheritance of a mutation in the β-globin gene. This genetic anomaly leads to the formation of dense and rigid red blood cells (RBCs), particularly those containing HbS or HbS in combination with other abnormal β alleles. When exposed to a deoxygenated environment, these RBCs undergo polymerization, becoming rigid and prone to haemolysis. The increased density of these cells can impact blood flow and compromise endothelial vessel wall integrity, causing vaso-occlusion, tissue ischemia, infarction, and further haemolysis. The consequences of haemolysis set off a complex cascade of events, including nitric oxide consumption, intricately linked to nitric oxide dysregulation and endothelial dysfunction. These underlying factors contribute to complications such as leg ulceration, stroke, pulmonary hypertension, and priapism. In a significant development in 2023, the U.S. Food and Drug Administration (FDA) approved two groundbreaking treatments, Casgevy and Lyfgenia, marking the introduction of the first cell-based gene therapies for treating SCD in patients 12 years and older. Notably, Casgevy represents a milestone as the first FDA-approved treatment utilizing a novel genome editing technology. This advancement opens new avenues for managing SCD and offers hope for improved outcomes in affected individuals.

The incidence of sickle cell disease (SCD) ranges between 27 and 35 cases per 100,000 people in the USA.

However, the current Sickle Cell Disease (SCD) treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (Etavopivat, VIT-2763 (Vamifeport), Mitapivat (AG-348), etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Sickle Cell Disease (SCD) treatment. The key companies in the advanced development stage are Forma Therapeutics, Inc., Vifor Pharma, Agios Pharmaceuticals, Inc., etc..

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Sickle Cell Disease (SCD) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2023 to 2033.

About Thelansis:

Thelansis specializes in pharmaceutical market outlook and market forecast reports. We published reports across the therapeutic area, including rare / ultra-rare and mainstream indications. Over the period, we have built a robust repository of 6,000+ Bio-pharma reports that cover Epidemiology studies and Market forecasting based on the KOL opinions.

Competitive intelligence and track of trial results throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the primary focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making.