Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Pharmacovigilance01:19

Pharmacovigilance

974
Post-marketing surveillance is a critical component of pharmaceutical regulation, often uncovering unanticipated adverse drug reactions (ADRs) once a drug is widely used over an extended period.
This process, termed pharmacovigilance, aims to detect, evaluate, and minimize harmful effects related to medication use. The data collection for pharmacovigilance depends on spontaneous reporting systems, where healthcare professionals or patients voluntarily report suspected ADRs.
In some cases, there...
974
Cancer Vaccines01:30

Cancer Vaccines

503
Cancer treatment vaccines are a rapidly evolving field that offers a promising approach to immunotherapy. Unlike traditional vaccines that prevent diseases, cancer treatment vaccines are designed to treat existing cancers by stimulating the immune system to recognize and attack cancer cells.
Cancer vaccines come in two categories: preventive (prophylactic) and treatment (active). Preventive vaccines, such as the Human Papillomavirus (HPV) vaccine, protect against viruses that cause certain...
503
Drug Administration and Therapy Phases: Overview01:26

Drug Administration and Therapy Phases: Overview

723
Drugs, the chemical agents used in diagnosing, treating, or preventing diseases, undergo a four-phase process of development: pharmaceutic, pharmacokinetics, pharmacodynamics, and therapeutic.
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
723
Clinical Trials01:16

Clinical Trials

7.2K
Clinical trials are prospective experimental studies conducted on humans to determine the safety and efficacy of treatments, drugs, diet methods, and medical devices. Using statistics in clinical trials enables researchers to derive reasonable and accurate conclusions from the collected data, allowing them to make wise decisions in uncertain situations. In medical research, statistical methods are crucial for preventing errors and bias.
There are four phases in a clinical trial. A phase one...
7.2K
Antiasthma Drugs: Muscarinic Receptor Antagonists01:20

Antiasthma Drugs: Muscarinic Receptor Antagonists

430
Muscarinic receptor antagonists, also known as antimuscarinic agents, are a class of bronchodilators used to treat asthma, although they are more commonly used to treat COPD. They work by inhibiting the action of acetylcholine (ACh), a neurotransmitter, on muscarinic receptors found in the airways.
Antimuscarinic agents compete with ACh for the same binding site on the muscarinic receptors. By binding to these receptors, they inhibit the downstream effects of ACh and block the parasympathetic...
430
Clinical Trials: Overview01:11

Clinical Trials: Overview

3.3K
Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
3.3K

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Icotrokinra: First Approval.

Drugs·2026
Same author

Ferric Maltol: Pediatric First Approval.

Paediatric drugs·2026
Same author

Sevabertinib: First Approval.

Drugs·2026
Same author

Retlirafusp Alfa: First Approval.

Drugs·2026
Same author

Zeprumetostat: First Approval.

Drugs·2025
Same author

Brensocatib: First Approval.

Drugs·2025

Related Experiment Video

Updated: Sep 5, 2025

Optimized LC-MS/MS Method for the High-throughput Analysis of Clinical Samples of Ivacaftor, Its Major Metabolites, and Lumacaftor in Biological Fluids of Cystic Fibrosis Patients
06:14

Optimized LC-MS/MS Method for the High-throughput Analysis of Clinical Samples of Ivacaftor, Its Major Metabolites, and Lumacaftor in Biological Fluids of Cystic Fibrosis Patients

Published on: October 15, 2017

8.4K

Mavacamten: First Approval.

Susan J Keam1

  • 1Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com.

Drugs
|July 8, 2022
PubMed
Summary

Mavacamten is an oral medication approved for adults with symptomatic obstructive hypertrophic cardiomyopathy (HCM). It improves functional capacity and symptoms in patients with New York Heart Association (NYHA) class II-III disease.

Area of Science:

  • Cardiology
  • Pharmacology
  • Drug Development

Background:

  • Hypertrophic cardiomyopathy (HCM) is a genetic heart muscle disease.
  • Obstructive HCM can lead to significant symptoms and functional limitations.
  • Current treatments for HCM have limitations.

Purpose of the Study:

  • To summarize the development milestones of mavacamten.
  • To highlight the regulatory approval of mavacamten for obstructive HCM.

Main Methods:

  • Review of preclinical and clinical development data.
  • Analysis of regulatory submission and approval process.

Main Results:

  • Mavacamten, a cardiac myosin inhibitor, demonstrated efficacy in clinical trials.

More Related Videos

Quail Chorioallantoic Membrane - A Tool for Photodynamic Diagnosis and Therapy
07:43

Quail Chorioallantoic Membrane - A Tool for Photodynamic Diagnosis and Therapy

Published on: April 28, 2022

2.9K
Paramyxoviruses for Tumor-targeted Immunomodulation: Design and Evaluation Ex Vivo
12:42

Paramyxoviruses for Tumor-targeted Immunomodulation: Design and Evaluation Ex Vivo

Published on: January 7, 2019

9.6K

Related Experiment Videos

Last Updated: Sep 5, 2025

Optimized LC-MS/MS Method for the High-throughput Analysis of Clinical Samples of Ivacaftor, Its Major Metabolites, and Lumacaftor in Biological Fluids of Cystic Fibrosis Patients
06:14

Optimized LC-MS/MS Method for the High-throughput Analysis of Clinical Samples of Ivacaftor, Its Major Metabolites, and Lumacaftor in Biological Fluids of Cystic Fibrosis Patients

Published on: October 15, 2017

8.4K
Quail Chorioallantoic Membrane - A Tool for Photodynamic Diagnosis and Therapy
07:43

Quail Chorioallantoic Membrane - A Tool for Photodynamic Diagnosis and Therapy

Published on: April 28, 2022

2.9K
Paramyxoviruses for Tumor-targeted Immunomodulation: Design and Evaluation Ex Vivo
12:42

Paramyxoviruses for Tumor-targeted Immunomodulation: Design and Evaluation Ex Vivo

Published on: January 7, 2019

9.6K
  • The U.S. Food and Drug Administration (FDA) approved mavacamten in April 2022.
  • Approval was for symptomatic New York Heart Association (NYHA) class II-III obstructive HCM.

    • Conclusions:

    • Mavacamten represents a novel therapeutic approach for obstructive HCM.
    • The development and approval of mavacamten offer a new treatment option for patients.
    • Further research may explore mavacamten in other HCM phenotypes.