European Medicines Agency Validates Bristol Myers Squibb’s MAA for Mavacamten in Obstructive Hypertrophic Cardiomyopathy
Oct 14, 2021 01:29
CST Updated
01:29
Bristol-Myers Squibb
Biopharmaceutical and Nutritional Product R&D and Sales
European Medicines Agency
The European Medicines Agency (EMA) is a decentralized agency of the European Union (EU), located in London. It began operations in 1995. The agency is responsible for the scientific evaluation, supervision, and safety monitoring of medicines developed by pharmaceutical companies for use in the EU. By ensuring that all medicines available on the EU market are safe, effective, and of high quality, the EMA protects public and animal health in the 28 EU Member States and countries of the European Economic Area.
Hypertrophic Cardiomyopathy (Image source: urmc.rochester.edu)
October 13, 2021 /BioonBIOON/ -- Bristol-Myers Squibb (BMS) recently announced that the European Medicines Agency (EMA) has acceptedmavacamten's Marketing Authorization Application (MAA), the drug is aNovel, oral cardiac myosin allosteric modulator for the treatment of obstructive hypertrophic cardiomyopathy (oHCM), a highly prevalent chronic heart disease.。
Currently, the New Drug Application (NDA) for mavacamten for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) is also under review by the U.S. FDA, with a Prescription Drug User Fee Act (PDUFA) target date of January 28, 2022. In July 2020, the United StatesFDABreakthrough Therapy Designation was granted to mavacamten for the treatment of oHCM.If approved, mavacamten will become the first cardiac myosin inhibitor for the treatment of oHCM.
Mavacamten is a first-in-class, oral, allosteric inhibitor of cardiac myosin for the treatment of diseases intrinsically caused by excessive cardiac contraction and impaired diastolic filling. Mavacamten is believed to reduce myocardial contractility by inhibiting the formation of excessive myosin-actin cross-bridges. Excessive formation of myosin-actin cross-bridges can lead to myocardial hypercontractility, left ventricular hypertrophy, and reduced compliance. In clinical and preclinical studies, mavacamten has consistently demonstrated a reduction in ventricular wall stress...Biomarker、reduce excessive myocardial contractility, increase diastolic compliance。
Mavacamten was initially developed for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Based on its mechanism of action and evidence of therapeutic activity, mavacamten is also being clinically investigated for the treatment of symptomatic non-obstructive hypertrophic cardiomyopathy (HCM) and heart failure with preserved ejection fraction (HFpEF).
Chemical structure of mavacamten (Image source: chemsrc.com)
The mavacamten NDA and MAA are both based on the results of the pivotal Phase 3 EXPLORER-HCM trial. The trial was conducted in patients with symptomatic oHCM, comparing mavacamten with placebo. Trial results showed that,Mavacamten demonstrated robust therapeutic efficacy, with clinically meaningful improvements in symptoms, functional status, and quality of life, while also demonstrating the ability to relieve left ventricular outflow tract obstruction. In the EXPLORER-HCM study, all primary and secondary endpoints achieved statistical significance.
Roland Chen, MD, Senior Vice President, Cardiovascular Development at Bristol-Myers Squibb, stated: “Despite the global prevalence of oHCM and its debilitating symptoms and cardiac dysfunction, no therapy targeting the underlying cause of this devastating disease has been approved. Currently available prescription medications are largely limited to symptom management. Mavacamten has the potential to provide a treatment option that addresses the unmet medical need within the global oHCM patient population.”
mavacamten (MYK-461) was developed by MyoKardia. On October 5, 2020, Bristol-Myers Squibb announced it would acquire MyoKardia for $13.1 billion in cash, representing a 60% premium. On November 17, 2020, Bristol-Myers Squibb announced that the acquisition of MyoKardia had been successfully completed. This acquisition is Bristol-Myers Squibb's second-largest transaction following its $74 billion acquisition of Celgene in 2019.
It is worth noting that, on August 11, 2020, byInvestment CompanyIncubated by Perceptive AdvisorsLianBio (LianBio)officially established, and on the same day announced two major partnerships: one to introduce BridgeBio Pharma's product pipeline to China, and the other isIntroduce MyoKardia's mavacamten to China.
Bristol-Myers Squibb has placed high expectations on mavacamten. Upon acquiring MyoKardia, the company announced that mavacamten would become a first-in-class drug for the treatment of HCM. The industry is also highly optimistic about the commercial prospects of mavacamten. In December 2020, the pharmaceutical market research firm Evaluate Vantage released *Top 10 New Drugs with the Highest Commercial Potential in 2021*, in which mavacamten ranked third. Evaluate Vantage projected that,Global sales of mavacamten are projected to reach $2 billion in 2026.. (Bioon.com)