FDA Extends Review Period for Bristol Myers Squibb’s First-in-Class Cardiac Myosin Inhibitor Mavacamten by Three Months

November 20, 2021 /BioValleyBIOON/ --Bristol-Myers Squibb (BMS) recently announced that the U.S. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) of mavacamten by 3 months, with the new Prescription Drug User Fee Act (PDUFA) target date set for April 28, 2022.This drug is a novel, oral, cardiac myosin allosteric modulator for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM)., which is a chronic heart disease with a high incidence rate.

In July 2020, the United StatesFDAMavacamten has been granted Breakthrough Therapy Designation for the treatment of oHCM. If approved, mavacamten will become the first cardiac myosin inhibitor for the treatment of oHCM. Currently, the Marketing Authorization Application (MAA) for mavacamten in the treatment of symptomatic oHCM is also under review by the European Medicines Agency (EMA).

According to the announcement by BMS, on November 18, 2021, the FDA notified the company of an extension to the PDUFA date in order to allow sufficient time to review updated information related to the proposed Risk Evaluation and Mitigation Strategy (REMS). The REMS program was included in the initial application for mavacamten.FDANo additional data or research was required.

Samit Hirawat, M.D., Executive Vice President and Chief Medical Officer of Bristol-Myers Squibb, said: "We are highly confident in mavacamten. In the pivotal EXPLORER-HCM trial, this first-in-class cardiac myosin inhibitor demonstrated clinically meaningful improvements in symptoms, functional status, and quality of life for patients with symptomatic oHCM. We look forward to continuing to collaborate with...FDA"Work closely to bring this important drug to patients."

Mavacamten is a first-in-class, oral, cardiac myosin allosteric inhibitor for the treatment of diseases characterized by excessive cardiac contraction and impaired cardiac diastolic filling.Mavacamten is considered to reduce myocardial contractility by inhibiting the excessive formation of myosin-actin cross-bridges. Excessive formation of myosin-actin cross-bridges can lead to excessive myocardial contraction, left ventricular hypertrophy, and reduced compliance. In clinical and preclinical studies,Mavacamten continues to demonstrate a reduction in cardiac wall stressBiomarker, reduce excessive myocardial contraction, and 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 (Source: chemsrc.com)

The NDA and MAA for mavacamten are both based on the results of the pivotal Phase 3 EXPLORER-HCM trial. This trial was conducted in symptomatic oHCM patients and compared mavacamten with placebo. The trial results showedMavacamten has demonstrated robust therapeutic effects, with clinically meaningful improvements in symptoms, functional status, and quality of life, while also showing the ability to alleviate left ventricular outflow tract obstruction. In the EXPLORER-HCM study, all primary and secondary endpoints achieved statistical significance.

Mavacamten (MYK-461) was developed by MyoKardia. On October 5, 2020, Bristol-Myers Squibb announced a $13.1 billion all-cash acquisition of MyoKardia at a 60% premium. On November 17, 2020, Bristol-Myers Squibb announced the successful completion of the acquisition of MyoKardia. This acquisition is Bristol-Myers Squibb's second-largest deal following the $74 billion acquisition of Celgene in 2019.

Notably, on August 11, 2020, byInvestment CompanyLianBio, incubated by Perceptive Advisors, has officially been established and announced two significant collaborations on the same day: one for introducing BridgeBio Pharma's product pipeline to China, and the other for bringing MyoKardia's mavecamten to China.

Bristol-Myers Squibb has high hopes for mavacamten, stating at the time of acquiring MyoKardia that mavacamten would become a first-in-class drug for the treatment of HCM. The industry is also very optimistic about the commercial prospects of mavacamten. In December 2020, pharmaceutical market research firm Evaluate Vantage released "The Top 10 New Drug Launches with the Most Commercial Potential in 2021," in which mavacamten ranked third. Evaluate Vantage expects,Mavacamten's global sales will reach $2 billion by 2026.. (Bioon.com)

Source of the original text:FDA extends action date for Bristol-Myers Squibb's mavacamten by three months