NMPA-granted Breakthrough Therapy Designation for the treatment of symptomatic obstructive hypertrophic cardiomyopathy based on results of REDWOOD-HCM and China Phase I study.
Ji Xing Pharmaceuticals Limited (JIXING), a clinical-stage biopharmaceutical company committed to bringing innovative medicines to underserved Chinese patients with serious and life-threatening diseases, announced in February that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of the PRC has granted Breakthrough Therapy Designation for aficamten (CK-3773274) for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in China. Aficamten, discovered by Cytokinetics, Incorporated (Cytokinetics), is a next-generation cardiac myosin inhibitor in development for the potential treatment of HCM. Earlier, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for aficamten to Cytokinetics for the treatment of symptomatic oHCM.
Breakthrough Therapy Designation was granted for aficamten by the NMPA to recognise its potential impact on patients with oHCM and to support the development and review of aficamten in China. Based on clinical trial results for aficamten, especially Cohorts 1 and 2 of REDWOOD-HCM, the global Phase 2 clinical trial, this Breakthrough Therapy Designation by CDE was granted for aficamten for the treatment of symptomatic oHCM, a severe and life-threatening disease. Currently, disease-specific therapies for symptomatic oHCM are limited. Aficamten may be a potential option in light of existing treatments. The CDE will prioritise resources for drugs included in the Breakthrough Therapy Designation programme and provide intensive guidance. A drug that receives Breakthrough Therapy Designation by the CDE is eligible for a priority review in the New Drug Application (NDA) process to further shorten the review time and accelerate approval.
“We are grateful to the NMPA for granting Breakthrough Therapy Designation for aficamten. This important regulatory classification (BTD) allows us to potentially accelerate the clinical development of aficamten which may benefit patients who suffer from oHCM. We look forward to continuing our strong collaboration with the NMPA as we conduct the China cohort of the global Phase 3 clinical trial, SEQUOIA-HCM, under our collaboration with Cytokinetics,” said Joseph Romanelli, CEO of JIXING. “As a company, our goal is to bring disruptive science to help improve upon the standard of care for patients in China, and this Breakthrough Therapy Designation is another great milestone in that quest.”
In December 2021, JIXING announced the completion of its Phase 1 study of aficamten in China. The study was a Phase 1, double-blind, randomised, placebo-controlled study to evaluate the safety, tolerability, and PK profile of aficamten in healthy Chinese participants. A total of 28 subjects were randomised and completed the study. Overall, the results showed a safety and tolerability profile comparable to placebo and dose-proportional pharmacokinetics, similar to the results observed in the Phase 1 study of aficamten in healthy, mostly Caucasian subjects in the US. [APBN]
Source: Ji Xing Pharmaceuticals Limited