The U.S. Food and Drug Administration (FDA) has granted accelerated approval of a supplemental New Drug Application (sNDA) for Oxbryta® (voxelotor) tablets for the treatment of Sickle cell disease (SCD) in children aged 4 to less than 12 years, according to Global Blood Therapeutics, Inc. This approval broadens Oxbryta’s previously approved use in the United States to treat SCD in patients aged 12 and up.

Source: Global Blood Therapeutics

Sickle cell disease (SCD) results from homozygous and compound heterozygote inheritance of a mutation in the β-globin gene. When exposed to the deoxygenated environment, red blood cells (RBCs) that contain HbS or HbS in combination with other abnormal β alleles, mnt, undergo polymerization and become rigid. The dense formidable RBC’s lead to vaso-occlusion, tissue ischaemia, infarction as well as hemolysis. The rigid RBC’s are liable to hemolysis and, due to increased density, may affect blood flow and endothelial vessel wall integrity. The dense formidable RBC’s lead to vaso-occlusion, tissue ischaemia, infarction as well as hemolysis. The consequence of hemolysis is a complex cascade of events, including nitric oxide consumption, hemolysis-linked nitric oxide dysregulation, and endothelial dysfunction, which underlie complications such as leg ulceration, stroke, pulmonary hypertension, and priapism.

• The incidence cases of SCD ranges between 27 to 35 cases per 100,000 population in the USA; however, the most common inherited life-threatening disorders most predominantly in the people of African, Indiana, and Arab ancestry.

However, the current Sickle cell disease (SCD) treatment market share, market uptake, attribute analysis concerning the most potential emerging therapies (VIT-2763, SHP655, GMI-1070, FT-4202, GPH101, Mitapivat, 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 Sickle cell disease (SCD) treatment. The key companies in the advanced development stage are Vifor Pharma, Shire, GlycoMimetics Incorporated, Graphite Bio, Inc., Forma Therapeutics, Inc., Agios Pharmaceuticals, Inc., etc.

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook 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 2020 to 2030.

Thelansis specializes in pharmaceutical market research and market Insight Report Company, published reports across the therapeutic area, including rare / ultra-rare and mainstream indication. 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

• Delivery Office:
  B-1030, C Wing Vrindavan tech village, Outer ring road
  Bangalore- 560037
  India+91(124)404-1731
  clientsupport@thelansis.com
• Sales office:
  183 Asylum Street Hartford,
  CT-06103, USA
  Contact no. +1 (302) 380-3552
  m.berg@thelansis.com