Amgen's Repatha gains an edge on rival Praluent with FDA approval to treat children with rare cholesterol condition

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An FDA approval for Amgen's cholesterol-reducing Repatha opens its use to for heterozygous familial hypercholesterolemia, a condition that affects 1 in 250 worldwide but often goes undiagnosed. (Fierce Pharma)

Though it has struggled to live up to early commercial expectations, Amgen’s cholesterol treatment Repatha has largely had the best of its rival in the market, Sanofi and Regeneron’s Praluent.

On Friday, Repatha gained another edge with an FDA approval for pediatric patients 10 years and older with heterozygous familial hypercholesterolemia (HeFH) to reduce bad cholesterol. The nod is for Repatha to be used as an add-on treatment to diet modification and statins.

HeFH is an inherited, genetic condition that affects 1 in 250 people worldwide. The disease is characterized by high levels of LDL-C (bad cholesterol) that start at birth and accelerate the development of cardiovascular disease. Children with the condition can be of normal weight and have good diet and exercise regimens. 

People with HeFH have cholesterol levels generally two to three times higher than normal. Of the approximately 1 million Americans with the disorder, only 10% are diagnosed, says the CDC. Those with HeFH have a 50% chance of passing it on to their children.

“As pediatric FH is an under-recognized condition that can lead to premature coronary artery disease, it’s critically important to have additional treatments that can significantly lower cholesterol,” Katherine Wilemon, the CEO of the FH Foundation said in a statement. 

Repatha, which is self-injected every two weeks or monthly, was already approved for another less-common FH condition—homozygous familial hypercholesterolemia (HoFH)—for those 13 and older. This FDA action expands its usage to those 10 and older with HoFH.

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The FDA based its green light on a study of Repatha’s safety and efficacy in pediatric patients ages 10 to 17 with HeFH. Treatment reduced bad cholesterol by a mean of 38% over 24 weeks compared to placebo, with no new safety risks identified.  

Repatha and Praluent were approved a month apart in 2015. Both target the PCSK9 protein. Repatha generated sales of $887 million last year, a 34% increase from 2019, compared to $359 million for Praluent, which has been approved to treat adults with HoFH and HeFH.

RELATED: Lackluster Amgen, Sanofi PCSK9 drugs could be under major threat from a new rival: analyst

Upon their approval, both drugs were expected to be blockbusters. But with each priced at more than $14,000 for a yearly course of treatment, uptake suffered from payer restrictions. The companies have since reduced their prices.

Looking forward, a new threat to the meds is likely to emerge with the expected approval of Novartis’ inclisiran, which also targets PCSK9 but operates differently. Its primary advantage is that it is administered just twice a year.