World’s first drug to target untreatable and deadly ‘sticky’ cholesterol

Sydney: A new drug offers a breakthrough world first treatment for Lipoprotein(a) — a largely genetic form of ‘sticky’ cholesterol that increases the risk of deadly heart attack and stroke, according to a study.

High levels of Lipoprotein(a), is similar to LDL cholesterol, sometimes called ‘bad cholesterol’, but is more sticky, increasing risk of blockages and blood clots in arteries. It impacts one in five people globally with no approved treatment currently on the market.

The first human trial led by Monash University in Australia demonstrated the success of Muvalaplin.

The first oral drug ever developed to target Lp(a) effectively lowered its levels by up to 65 per cent. It works by disrupting the ability for Lp(a) to form in the body.

The findings were presented at the European Society of Cardiology Congress in Amsterdam and also published in JAMA.

“This drug is a gamechanger in more ways than one. Not only do we have an option for lowering an elusive form of cholesterol, but being able to deliver it in an oral tablet means it will be more accessible for patients,” said study lead Professor Stephen Nicholls, Director of the Monash University’s Victorian Heart Institute and Victorian Heart Hospital.

Common LDL lowering drugs such as statins don’t have the same lowering effect on Lp(a).

Being largely genetic, Lp(a) is also difficult to control through diet, exercise and other lifestyle changes.

Although Lp(a) was discovered nearly 60 years ago there still aren’t any widely accessible treatments available to lower levels and reduce cardiovascular risk.

“When it comes to treating high Lp(a), a known risk factor for cardiovascular disease, our clinicians currently have no effective tools in their kit,” Nicholls said. “Lp(a) is essentially a silent killer with no available treatment, this drug changes that,” he added.

Muvalaplin was not associated with tolerability concerns and lowered lipoprotein(a) (Lp[a]) levels following daily administration for 14 days in the first-in-human phase 1 study involving healthy participants.

Longer and larger trials will be required to further evaluate safety, tolerability, and effect of muvalaplin on Lp(a) levels and cardiovascular outcomes, the researchers said.


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