▎WuXi AppTec Content Team Report

On December 25, the official website of the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China announced,Novo Nordisk submitted a clinical trial application for the Class 1 new drug etavopivat, which has been accepted. According to public information,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

According toNovo Nordisk Official Website，Etavopivat is being developed for the treatment ofSickle Cell Disease (SCD)。SCD is a chronic, progressive genetic disease related to the health and lifespan of red blood cells. The red blood cells of patients are not healthy disc-shaped but appear crescent-shaped, making them relatively fragile, inelastic, and unable to effectively transport oxygen. This disease causes severe health problems for patients, including anemia, fatigue, episodic pain caused by vaso-occlusive crises, and chronic, progressive end-organ damage. Despite advancements in treatment, most patients still suffer from pain, lifelong disability, reduced quality of life, and decreased life expectancy.

As aAn investigational, once-daily selective PKR agonist,etavopivatBy activating the natural PKR within red blood cells, it can reduce the content of the anaerobic glycolysis product 2,3-DPG in red blood cells, allowing the cells to carry more oxygen, increase adenosine triphosphate (ATP) production, and reduce hemolysis and the formation of sickle cells.。

Previously conducted overseasPhase 1ClinicalIn the trial,Etavopivat hasDemonstrated good tolerability and showed potential in improving red blood cell health, increasing hemoglobin levels, and reducing symptoms in patients.Recurrent 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 U.S. FDA,InAndOrphan Drug Designation granted by the European Commission (EC).

According toClinicalTrials.govWebsite information, currently eTavopivat is being evaluated in a Phase 2 clinical trial for patients with SCD. In addition to SCD, researchers are also assessing it in a Phase 2 clinical trial.etavopivatEfficacy and Safety in Treating Anemia in Patients with Myelodysplastic Syndromes (MDS) and Thalassemia.

References:
[1] Official website of the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China. Retrieved Dec 25, 2023, 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