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02:05

What Is siRNA Therapy for High Cholesterol?

This new type of high cholesterol medication may help when statins aren’t the right fit.

Lauren Smith, MAMera Goodman, MD, FAAP
Written by Lauren Smith, MA | Reviewed by Mera Goodman, MD, FAAP
Updated on September 30, 2024

If you have high cholesterol, your care team may recommend starting a statin (or HMG-CoA reductase inhibitors), which has long been the first-line medication to improve cholesterol levels. When statins aren’t enough, your doctor may recommend other options. One of the newest options to treat high cholesterol is siRNA therapy.

What is siRNA therapy for high cholesterol?

The first siRNA therapy for high cholesterol received FDA approval in December 2021. It stands for small interfering RNA therapy. RNA (ribonucleic acid) is found in all living cells. It gives instructions to other parts of your body.

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These medications work by targeting these “messenger molecules.” They give your body instructions to stop producing PCSK9 proteins. These are proteins produced by your liver. Research has shown that high levels of PCSK9 proteins is linked to higher LDL (“bad”) cholesterol.

By telling your body to make less of this protein, siRNA therapy may help reduce LDL cholesterol

How is siRNA therapy for high cholesterol administered?

While statins are an oral pill that you must take every day, siRNA therapy is currently mainly an injection. An injection may help reduce the “pill burden” if you’re already taking multiple oral pills a day.

The current regiment usually goes like this: After your initial dose, you will return for a second dose after three months. After this, you generally receive two doses a year. 

Can I take siRNA therapy instead of a statin?

In general, siRNA therapy is meant to be used as an add-on treatment to statin therapy. This may help people who have not been able to reach their target cholesterol on statins.

How is siRNA therapy different from PCSK9 inhibitors?

PCSK9 inhibitors are a broad class of medications that lower the production of this protein. They can work in different ways to achieve this goal. Many PCSK9 inhibitors come in injection form, but there are oral options available.

What makes siRNA therapy unique is that it offers a new and novel way of reducing the formation of these proteins. 

What should I do if I’m curious about learning more?

If you’re curious about learning more about this new cholesterol medication, it’s best to ask your cardiologist. They can help go over the risks and benefits of siRNA therapy and help decide if you’d be a good candidate.

Additional Medical Contributors
  • Jennifer Haythe, MDDr. Haythe is a Cardiologist at Columbia University's Irving Medical Center.
    View All References (3)

    Padda, I.S., et al. (2020). Small Interfering RNA (siRNA) Therapy. StatPearls [Internet].

    Pokhrel, B., et al. (2022). PCSK9 inhibitors. StatPearls [Interet].

    U.S. Food & Drug Administration. (2021). FDA approves add-on therapy to lower cholesterol among certain high-risk adults.

    GoodRx Health has strict sourcing policies and relies on primary sources such as medical organizations, governmental agencies, academic institutions, and peer-reviewed scientific journals. Learn more about how we ensure our content is accurate, thorough, and unbiased by reading our editorial guidelines.

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