▎WuXi AppTec Content Team Report

On March 15, the official website of the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China announced,Novo Nordisk's申报的1类新药etavopivat获得临床试验默示许可，拟开发治疗Thalassemia. Publicly available information shows,eTavopivat is a selective pyruvate kinase-R (PKR) agonist.September 2022,Novo Nordisk Acquires for $1.1 BillionForma Therapeutics thereby acquired this product.

Screenshot source:CDE Official Website

ThalassemiaAlso known as thalassemia, it is an autosomal recessive genetic disorder, mainly found in the Mediterranean region.Hai ZhouMore common among residents in border areas.ThisIt is a blood disorder that affects the body's ability to produce normal hemoglobin, specifically due to genetic mutations that hinder the synthesis of hemoglobin molecules, causing abnormal changes in the shape, function, and lifespan of red blood cells.For this disease, there are still challenges in clinical practice.Huge unmet clinical needs.

Etavopivat isAAn investigational, once-daily selective PKR agonist being developed for the treatment ofMyelodysplastic Syndromes (MDS)And thalassemia. ItBy activating red blood cellsThe natural PKR activity to reduce metabolite 2,The level of 3-DPG,Keeps sickle hemoglobin on oxygen longer, thereby reducing polymerization, hemolysis andSickle Change。The ProductPKR-mediated activation can also increase adenosine triphosphate (ATP) levels to improve erythrocyte function, thereby enhancing deformability, membrane repair capacity, erythrocyte health, and lifespan.Overall,These worksCan improve the health of sickle red blood cells and reduce anemia, hemolysis, vascular occlusive crisis, and end-organ damage.

Previously carried out inPhase 1ClinicalIn the trial,Etavopivat hasDemonstrated good tolerability and showedImprove red blood cell health, increase hemoglobin content, and reduce patient sufferingRecurrent Vascular Occlusion Crisis (VOC)Symptoms and the Potential to Improve Patients' Quality of Life。The drug was onceGranted Fast Track Designation, Rare Pediatric Disease Designation, and Orphan Drug Designation by the US FDA,InAndOrphan Drug Designation granted by the European Commission (EC).

According toClinicalTrials.govWebsite information, currently eTavopivat is ongoingPhase 2 Clinical Study for Thalassemia or Sickle Cell Disease.The study will evaluate the product in thalassemia orSickle Cell AnemiaSafety and clinical activity in patients, and testingetavopivatIn terms of reducing the required number of red blood cell transfusions and increasing hemoglobin levels.

References:
[1] Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China. Retrieved Mar 15, 2024, from https://www.cde.org.cn/main/xxgk/listpage/9f9c74c73e0f8f56a8bfbc646055026d

[2] Novo Nordisk to acquire Forma Therapeutics and expand presence in sickle cell disease and rare blood disorders. Retrieved September 1, 2022 from https://www.novonordisk.com/content/nncorp/global/en/news-and-media/news-and-ir-materials/news-details.html?id=132653

[3] Novo Nordisk Official Website. From https://www.novonordisk.com/science-and-technology/r-d-pipeline.html