Search research articles

ABOUT JoVE

Overview Leadership Blog JoVE Help Center

AUTHORS

Publishing Process Editorial Board Scope & Policies Peer Review FAQ Submit

LIBRARIANS

Testimonials Subscriptions Access Resources Library Advisory Board FAQ

RESEARCH

JoVE Journal Methods Collections JoVE Encyclopedia of Experiments Archive

EDUCATION

JoVE Core JoVE Business JoVE Science Education JoVE Lab Manual Faculty Resource Center Faculty Site

Terms & Conditions of Use

Related Concept Videos

Impact of Pharmacokinetic–Pharmacodynamic Models: Regulatory Decisions

Impact of Pharmacokinetic–Pharmacodynamic Models: Regulatory Decisions

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 (CHF).

FDA Approved Drugs: Changes to Approved Drugs

FDA Approved Drugs: Changes to Approved Drugs

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,...

Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption

Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption

Understanding the physiological differences in the pediatric population is crucial for effective pharmacotherapy. Neonates, infants, and children exhibit significant variations in gastric pH, gastric emptying time, intestinal transit time, and biliary function. These variations profoundly affect oral drug absorption, necessitating a nuanced approach to pediatric dosing.Neonates present with a unique physiological profile, having a gastric pH greater than 4 and faster and more irregular gastric...

Dipeptidyl Peptidase 4 Inhibitors

Dipeptidyl Peptidase 4 Inhibitors

Dipeptidyl peptidase 4 (DPP-4) is a serine protease widely distributed in the body. It's involved in the inactivation of GLP-1 and GIP hormones, which are crucial for insulin regulation. DPP-4 inhibitors, such as sitagliptin (Januvia), saxagliptin (Onglyza), linagliptin (Tradjenta), alogliptin (Nesina), and vildagliptin (Galvus), help increase the proportion of active GLP-1, enhancing insulin secretion. These inhibitors work by competitively binding to DPP-4. This binding causes a significant...

Prescription, Nonprescription and Orphan Drugs

Prescription, Nonprescription and Orphan Drugs

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...

Pharmacogenomics: Identification of New Drug Targets

Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...

You might also read

Related Articles

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

Sort by

Same author

The revised remote area nurse model of consultation.

The Australian journal of rural health·2024

Same author

Pediatric Inflammatory Bowel Disease Type Unclassified: A Nationwide Cohort Study in Scotland With up to 20 Years Follow-up Shows Reclassification in the Majority and Mild Course in Those Whose Diagnosis Is Unchanged.

Inflammatory bowel diseases·2024

Same author

Twenty-first Century Trends in the Global Epidemiology of Pediatric-Onset Inflammatory Bowel Disease: Systematic Review.

Gastroenterology·2022

Same author

Understanding and Responding to the Impact of COVID-19 on Paediatric Gastroenterology Training & Practice of Young ESPGHAN Members.

Journal of pediatric gastroenterology and nutrition·2021

Same author

Homecoming-Dignity Through Movement at the End of Life: A Qualitative Interview Study With Healthcare Professionals.

The American journal of hospice & palliative care·2021

Same author

From Paris to Montreal: disease regression is common during long term follow-up of paediatric Crohn's disease.

Scandinavian journal of gastroenterology·2020

Search research articles

Related Experiment Video

Updated: May 12, 2026

Transcatheter Pulmonary Valve Replacement from Autologous Pericardium with a Self-Expandable Nitinol Stent in an Adult Sheep Model

Transcatheter Pulmonary Valve Replacement from Autologous Pericardium with a Self-Expandable Nitinol Stent in an Adult Sheep Model

Published on: June 8, 2022

Mipomersen sodium: first global approval.

Philip Hair¹, Fiona Cameron, Kate McKeage

¹Adis R & D Insight, 41 Centorian Drive, Private Bag 65901, Mairangi Bay, North Shore, 0754 Auckland, New Zealand. dru@adis.com

|April 9, 2013

Summary

Mipomersen is an antisense oligonucleotide approved for homozygous familial hypercholesterolaemia (HoFH). This treatment targets apolipoprotein B-100 to reduce atherogenic lipid particles.

Area of Science:

Pharmacology
Cardiovascular Medicine
Genetics

Background:

Familial hypercholesterolaemia (FH) is a genetic disorder causing high cholesterol.
Homozygous FH (HoFH) is a severe form requiring intensive treatment.
Atherogenic lipid particles, like those containing apolipoprotein B-100, are key contributors to cardiovascular disease.

Purpose of the Study:

To summarize the development of mipomersen sodium (Kynamro™).
To highlight milestones leading to its approval for HoFH treatment.
To review clinical trial data for mipomersen in various FH populations.

Main Methods:

Development of a second-generation antisense oligonucleotide.
Administration via subcutaneous injection.

More Related Videos

Studying Metabolic Brain Connectivity Using 2-Deoxy-2-[18F]Fluoro-D-Glucose Dynamic Positron Emission Tomography at the Single-subject Level

Studying Metabolic Brain Connectivity Using 2-Deoxy-2-[¹⁸F]Fluoro-D-Glucose Dynamic Positron Emission Tomography at the Single-subject Level

Published on: January 24, 2025

Related Experiment Videos

Last Updated: May 12, 2026

Transcatheter Pulmonary Valve Replacement from Autologous Pericardium with a Self-Expandable Nitinol Stent in an Adult Sheep Model

Transcatheter Pulmonary Valve Replacement from Autologous Pericardium with a Self-Expandable Nitinol Stent in an Adult Sheep Model

Published on: June 8, 2022

Studying Metabolic Brain Connectivity Using 2-Deoxy-2-[18F]Fluoro-D-Glucose Dynamic Positron Emission Tomography at the Single-subject Level

Studying Metabolic Brain Connectivity Using 2-Deoxy-2-[¹⁸F]Fluoro-D-Glucose Dynamic Positron Emission Tomography at the Single-subject Level

Published on: January 24, 2025

Multinational phase III clinical trials in HoFH, severe FH, and high-risk patients.

Main Results:

Mipomersen targets apolipoprotein B-100, a component of atherogenic particles.
Achieved regulatory approval in the USA as adjunctive therapy for HoFH.
Undergoing regulatory review in the EU for the same indication.

Conclusions:

Mipomersen represents a novel therapeutic option for HoFH.
The drug's development involved extensive multinational clinical trials.
Ongoing studies are evaluating long-term efficacy and alternative dosing regimens.