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

FDA Approved Drugs: Changes to Approved Drugs01:26

FDA Approved Drugs: Changes to Approved Drugs

330
Post-approval, manufacturers may modify an approved new or generic drug product. Such modifications can encompass alterations in the Active Pharmaceutical Ingredient (API), manufacturing process, formulation, batch size, manufacturing site, and container closure system (FDA Guidance for Industry, April 2004). Often, a drug product may undergo multiple changes.These modifications require careful evaluation to determine their potential impact on the drug product's identity, strength, quality,...
330
Drug Administration and Therapy Phases: Overview01:26

Drug Administration and Therapy Phases: Overview

1.7K
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...
1.7K
Drug Regulation01:25

Drug Regulation

3.3K
Drug regulation encompasses the management of drug usage by evaluating its safety and efficacy through assessments conducted by regulatory authorities. Regrettably, the history of drug regulation is marred by several catastrophic events. One such incident is the Elixir Sulfanilamide tragedy, in which the toxic compound diethyl glycol was included in a sweet-tasting medication, leading to numerous fatalities. This event prompted the enactment of the Food, Drug, and Cosmetic Act in 1938. Under...
3.3K
Prescription, Nonprescription and Orphan Drugs01:02

Prescription, Nonprescription and Orphan Drugs

1.4K
Prescription drugs require a prescription from a medical practitioner and can only be obtained from a pharmacy. They have many applications, including treating pain, anxiety, and hypertension.
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
1.4K
Impact of Pharmacokinetic–Pharmacodynamic Models: Regulatory Decisions01:15

Impact of Pharmacokinetic–Pharmacodynamic Models: Regulatory Decisions

72
PK–PD modeling has significantly influenced FDA regulatory decisions, particularly drug approval, dosage optimization, and labeling. These models integrate pharmacokinetics (PK) and pharmacodynamics (PD) to predict drug behavior and effects, aiding in optimizing dosing regimens and enhancing the probability of clinical trial success.One notable example is Nesiritide (Natrecor®), a recombinant human brain natriuretic peptide for treating acute decompensated congestive heart failure...
72
Pharmacovigilance01:19

Pharmacovigilance

2.0K
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...
2.0K

You might also read

Related Articles

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

Sort by
Same author

Tividenofusp Alfa: First Approval.

Molecular diagnosis & therapy·2026
Same author

Milsaperidone: First Approval.

Clinical drug investigation·2026
Same author

Narsoplimab: First Approval.

Drugs·2026
Same journal

Biologics and Small Molecule Inhibitors: Novel Therapeutic Strategies for Cutaneous Adverse Drug Reactions.

Drugs·2026
Same journal

Use of Sedative-Hypnotic Drugs and the Risk of Developing Alzheimer's Disease: A Systematic Review, Meta-Analysis and Meta-Regression.

Drugs·2026
Same journal

Relacorilant: First Approval.

Drugs·2026
Same journal

Developmental Progress and Future Potential for Inhaled Biologics in the Treatment of Respiratory Diseases.

Drugs·2026
Same journal

Linerixibat: First Approval.

Drugs·2026
Same journal

Recaticimab: The First Fc-Engineered PCSK9 Monoclonal Antibody for More Long-Lasting Effect in Lipid Lowering.

Drugs·2026
See all related articles

Related Experiment Video

Updated: Apr 7, 2026

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

8.9K

Aficamten: First Approval.

Matt Shirley1

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

Drugs
|April 6, 2026
PubMed
Summary
This summary is machine-generated.

Aficamten is a new cardiac myosin inhibitor approved for symptomatic obstructive hypertrophic cardiomyopathy (HCM). This medication improves function and symptoms in adults with obstructive HCM, marking a significant advancement in cardiovascular treatment.

More Related Videos

An Automated Radiosynthesis of [68Ga]Ga-FAPI-46 for Routine Clinical Use
10:33

An Automated Radiosynthesis of [68Ga]Ga-FAPI-46 for Routine Clinical Use

Published on: May 24, 2024

1.8K
Author Spotlight: Advancements in CAR-T Cell Manufacturing and Gene Therapy Production
06:18

Author Spotlight: Advancements in CAR-T Cell Manufacturing and Gene Therapy Production

Published on: August 18, 2023

3.9K

Related Experiment Videos

Last Updated: Apr 7, 2026

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

8.9K
An Automated Radiosynthesis of [68Ga]Ga-FAPI-46 for Routine Clinical Use
10:33

An Automated Radiosynthesis of [68Ga]Ga-FAPI-46 for Routine Clinical Use

Published on: May 24, 2024

1.8K
Author Spotlight: Advancements in CAR-T Cell Manufacturing and Gene Therapy Production
06:18

Author Spotlight: Advancements in CAR-T Cell Manufacturing and Gene Therapy Production

Published on: August 18, 2023

3.9K

Area of Science:

  • Cardiology
  • Pharmacology
  • Clinical Trials

Background:

  • Hypertrophic cardiomyopathy (HCM) is a genetic heart muscle disease.
  • Obstructive HCM (oHCM) significantly impacts patient quality of life.
  • Limited treatment options exist for symptomatic oHCM.

Purpose of the Study:

  • To summarize the development milestones of aficamten.
  • To highlight the regulatory approvals for aficamten in oHCM.
  • To review the clinical trial data supporting aficamten's efficacy.

Main Methods:

  • Phase III, randomized, double-blind, placebo-controlled SEQUOIA-HCM trial.
  • Evaluation of aficamten's effect on functional capacity and symptoms.
  • Assessment of safety and tolerability profiles.

Main Results:

  • Aficamten demonstrated significant improvements in functional capacity and symptoms in adults with oHCM.
  • Regulatory approvals obtained in China, USA, and EU based on trial findings.
  • Ongoing Phase III evaluation for non-obstructive HCM (nHCM).

Conclusions:

  • Aficamten represents a novel therapeutic option for symptomatic obstructive HCM.
  • The drug's development highlights successful progression from clinical trials to market approval.
  • Further research is underway to expand its therapeutic applications.