SEP 28, 2021 9:00 AM PDT

The FDA approves drug to treat a bad cholesterol in children

WRITTEN BY: J. Bryce Ortiz

Familial hypercholesterolemia (FH) is a genetic disorder that afflicts approximately 1 in 250 people. Interestingly, while it is estimated that more than one million people in the United States have FH, less than 10% of these individual know they have it. FH is a disorder in which individuals have significantly high blood levels of low-density lipoprotein (LDL) cholesterol, more commonly known as “bad cholesterol.” When blood levels of this bad cholesterol become high, and remain high for extended periods of time, the cholesterol can accumulate into the walls of blood vessels which raises the chances of serious health problems such as heart attack or stroke. 

For most people, maintaining low levels of LDL cholesterol can be accomplished through lifestyle choices such as choosing a healthy diet, proper exercise, and quitting smoking. However, individuals who are affected by FH have high cholesterol levels early in life. This has many detrimental effects and in these individuals heart disease my start to develop earlier than the normal population, leading to heightened risks for heart attack and stroke at a young age. Because of this, early and frequent diagnostic testing has been encouraged for anyone with a family member with high cholesterol. 

Once an individual is diagnosed with FH, treatment can begin. These treatments include lifestyle changes or improvements in diet and exercise, and drug regimens. While there are many drugs to treat high cholesterol, many of the recently approved medications for FH were not approved for children of all ages. However, last week, the United States Food and Drug Administration (FDA) approved the drug Repatha (Manufactured by Amgen Inc.) for use in children as young as 10-years old. After the approval, David M. Reese, MD, vice president of research and development at Amgen said, “The approval of Repatha for pediatric patients with FH represents a much-needed adjunct treatment option for these children with genetically high cholesterol who are unable to manage their high LDL-C with other lipid-lowering agents alone.”

The FDA based their approval on recent clinical data in children aged 10-17 years old. The participants in the study had FH and received a single injection of Repatha monthly for 6 months. When the blood LDL levels of these patients were compared to patients who were not administered Repatha, the researchers found a 38% reduction in blood LDL levels. The approval of this lifesaving medication is a welcomed relief for pediatric FH patients, their families, and advocates. 

 

Sources: CDCMedlinePlusThe Cleveland ClinicThe FH foundationFDARepathaAmgen

About the Author
  • Science and medical writer | Researcher | Interested in the intersection between translational science, drug development, and policy
You May Also Like
JUL 23, 2021
Cancer
New Drug Eradicates Breast Cancer Tumors in Days
JUL 23, 2021
New Drug Eradicates Breast Cancer Tumors in Days
A new drug can kill between 95 and 100% of cancer cells in mice with human estrogen-receptor-positive breast cancer. The ...
AUG 05, 2021
Cannabis Sciences
Brain's Version of THC Reduces Seizures, Increases Side Effects
AUG 05, 2021
Brain's Version of THC Reduces Seizures, Increases Side Effects
An endocannabinoid similar to tetrahydrocannabinol (THC) that naturally occurs in the brain reduces seizure activity. Ho ...
AUG 12, 2021
Drug Discovery & Development
Postpartum depression drug works through altering brain waves
AUG 12, 2021
Postpartum depression drug works through altering brain waves
     Postpartum depression is a form of depression that occurs after childbirth. This form of depres ...
SEP 09, 2021
Drug Discovery & Development
Can vaccines help prevent and treat opioid addiction?
SEP 09, 2021
Can vaccines help prevent and treat opioid addiction?
According to the United States Centers for Disease Control (CDC), 136 people die from an opioid overdose every ...
SEP 12, 2021
Coronavirus
Real-World, Post-Delta COVID-19 Vaccine Data & Potential Treatment
SEP 12, 2021
Real-World, Post-Delta COVID-19 Vaccine Data & Potential Treatment
New research may have identified a potential treatment or preventive medication for SARS-CoV-2 and other viral infection ...
SEP 14, 2021
Drug Discovery & Development
A novel drug that targets the removal of pathogenic antibodies in myasthenia gravis
SEP 14, 2021
A novel drug that targets the removal of pathogenic antibodies in myasthenia gravis
Myasthenia gravis is a chronic autoimmune disorder characterized by muscle weakness and fatigue. The disorder leads to a ...
Loading Comments...