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FDA expands Juxtapid indication for children with rare cholesterol condition
Juxtapid has been available to treat adults with homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder that causes extremely high levels of LDL cholesterol.

Dhruv S. Kazi, M.D., a cardiologist at Beth Israel Deaconess Medical Center, discusses the research he and his team have done to determine if semaglutide used as secondary prevention of cardiovascular disease is cost-effective.

Researchers say a modest price cut would make semaglutide cost-effective for secondary prevention of cardiovascular disease in the United States.

New research at Mount Sinai explains how influenza infections increase the risk of heart attack through a specific immune pathway.

Rural hospital study found text messaging intervention significantly improved medication adherence for heart failure patients, demonstrating that simple technology can reduce readmissions.

Myqorzo was approved in December to treat adults with obstructive hypertrophic cardiomyopathy. It is available through a REMS program and has a boxed warning about the risk of heart failure.

Elevated levels of lipoprotein(a) is a genetic disease that causes cardiovascular disease. Steven Nissen, M.D., addresses why it’s important to find treatments for this genetic risk factor.

Five experimental drugs show promise in significantly lowering lipoprotein(a). The furthest along is pelacarsen, with phase 3 results expected in the first half of this year, and regulatory filings in the second half of this year.

Transthyretin amyloid cardiomyopathy causes heart failure through protein buildup in heart muscle. Three FDA-approved disease-specific therapies are available, but high costs limit access.

Tryvio, an oral drug for those with uncontrolled hypertension, will be available in the second half of 2024. The list price has not yet to be determined.

The FDA has set an action date of Nov. 29, 2024, to review acoramidis to treat patients with transthyretin amyloid cardiomyopathy.

Inpefa is an inhibitor of both SGLT2 and SGLT1, approved to reduce the risk of cardiovascular death and hospitalization for heart failure in adults. The wholesale acquisition cost is $598 per month.

Alnylam Pharmaceuticals will no longer pursue this indication of Onpattro and will instead on focus on a label expansion for Amvuttra, which is in phase 3 development to treat patients with cardiomyopathy of ATTR amyloidosis.

Even though committee members voted in support of Onpattro for patients with cardiomyopathy related to transthyretin-mediated amyloidosis, there were questions about whether it provided a clinically meaningful benefit. The FDA set an action date of Oct. 8, 2023.

Lodoco targets the inflammation that is an underlying cause of atherosclerotic cardiovascular disease. It will be available in the second half of 2023.

In an analysis, researchers determined that Inpefa is cost-effective at commonly accepted willingness-to-pay thresholds in treating patients with heart failure who have diabetes.

Inpefa (sotagliflozin) is in the SGLT inhibitor class recommended as first-line treatment for heart failure by the American Heart Association and other groups. Jardiance (empaglifozin), a competitor in that class, has a list price of $570.48 for a month's supply.
Evkeeza treats a rare form of high cholesterol. It is now approved for patients as young of 5 years of age.

Alnylam is seeking approval for Onpattro’s use in cardiomyopathy related to transthyretin-mediated (ATTR) amyloidosis. The FDA set an action date of Oct. 8, 2023.

In a real-world example, investigators found that lowered pricing of prescription drugs results in lower patient out-of-pocket costs, particularly for those patients with coinsurance.

The Inflation Reduction Act’s cap on annual out-of-pocket spending and changes to coverage gaps could save Medicare patients more than $1,000 on heart failure medications.

A phase 3 study demonstrated that Onpattro improved functional capacity, health status and quality of life compared with placebo in this patient population.
If approved, Evkeeza would be the first treatment to help children as young as 5 years old control high LDL cholesterol caused by homozygous familial hypercholesterolemia. The FDA target action date is March 30, 2023.

If approved, Xphozah will be the first phosphate absorption inhibitor for adults with chronic kidney disease who are on dialysis. An FDA decision is expected within 30 days.

Furoscix can be administered at home with the use of the On-Body Infusor, which delivers furosemide over five hours. It will be launched in the first quarter of 2023.


































