Olezarsen's EMA approval: When is the new medicine for lowering triglycerides coming to you?

Tryngolza (olezarsen) is the first approved treatment for familial chylomicronemia syndrome. And the newest medicine for lowering triglycerides ¹. According to Dr. Brian Bergmark, olezarsen lowers triglycerides to a greater extent than any other currently approved medicine, and is also safe ².

This speaks to the importance of this new medicine in preventing cardiovascular diseases. However, it is currently only available in the USA.

When can patients outside the USA expect to have access to olezarsen? Here's everything you need to know about olezarsen's EMA approval, its availability in the UK, and your options to get the medicine before it's available in your country.

What is olezarsen used for?

Tryngolza (olezarsen) is used to reduce triglycerides in adult patients with familial chylomicronemia syndrome (FCS) ³. The medicine is used in combination with an appropriate diet containing under 20 g of fat per day ¹.

How does olezarsen work for hypertriglyceridimia?

Olezarsen works by targeting a protein called ApoC-III, which normally slows down the breakdown of triglycerides in the blood.

It’s an antisense therapy, meaning it attaches to the genetic instructions (mRNA) that tell cells to make ApoC-III. This triggers the body to destroy those instructions, reducing the amount of ApoC-III made.

With less ApoC-III, an enzyme called lipoprotein lipase (LPL) can work better to break down triglycerides, leading to lower fat levels in the blood. This precise method focuses specifically on ApoC-III, reducing the chances of side effects ⁴.

Can olezarsen lower triglycerides in patients without FCS?

Yes. While olezarsen is the first treatment approved for use in familial chylomicronemia syndrome, it can also lower triglycerides in patients without the condition. In a phase 2 study focusing on non-FCS patients, olezarsen demonstrated a 60% reduction in triglyceride levels compared to placebo ⁵.

Does olezarsen reduce the risk of acute pancreatitis?

Yes. In the Phase 3 Balance study, olezarsen demonstrated a 100% reduction in acute pancreatitis events compared to placebo ⁶. This is an important result, as patients with FCS are at a high risk of potentially fatal acute pancreatitis.

What are olezarsen's clinical trial results?

Tryngolza's FDA approval was largely based on the results of the Phase 3 Balance study. The study compared olezarsen against placebo in patients with FCS. The key reported outcomes were:

• Triglyceride levels were reduced by 44% (in patients taking the 80 mg dose) and 22% (in patients taking the 50 mg dose) vs placebo;
• Triglyceride level reductions were sustained at 6 and 12 months after starting treatment;
• Patients treated with olezarsen reported a meaningful reduction in acute pancreatitis events (1 in the 80 mg group and 1 in the 50 mg group, compared to 11 events in the placebo group);
• There was a substantially greater time to the the first acute pancreatitis event with olezarsen (1 year in the 80 mg group; 102 days in the 50 mg group), compared to placebo (9 days);
• The safety profile of olezarsen was favourable, with no serious adverse events reported ⁷.

When will olezarsen get EMA approval?

Olezarsen has been under review by the EMA since 15 August 2024. Typically, EMA approval decisions take up to 210 days. Meaning that olezarsen could potentially get its approval in Europe in the first half of 2025.

When will olezarsen be available to patients in Europe?

Approval is not the same as availability. Even if olezarsen gets EMA approval in early 2025, that doesn't mean it will be available to patients across the EU right away.

Each member state needs to first go through its own process of price negotiations, local reimbursement decisions, and launch. Depending on where in Europe you're based, this could take on average between 133 days (in Germany) and 899 days (in Romania) ⁸.

In other words, some patients in Europe may be able to start their treatment with olezarsen in the second half of 2025. Others may need to wait another couple of years.

When will olezarsen be approved in the UK?

It's difficult to say. There is currently no active application for olezarsen's approval in the UK. This being said, one is not necessarily required. According to post-Brexit regulations, the MHRA may grant approval to medicines as soon as they have been given the green light by another trusted regulatory body (such as the FDA or EMA). If this is the MHRA's chosen approach, then it could theoretically already approve olezarsen, or approve it as soon as the EMA does (expected in early 2025).

Olezarsen's availability on the NHS

While Tryngolza may be approved in the UK within months, it won't be available on the NHS until NICE has made a decision on the topic. As of January 2025, no NICE assessment of the medicine has been initiated, so it's difficult to provide a potential decision timeline.

Typically, a medicine becomes available on the NHS within 3 months of a positive NICE assessment.

How to get olezarsen before it's available in your country

If you're a patient with FCS or hypertriglyceridimia in Europe, the UK, or elsewhere outside the USA, waiting for olezarsen to become available locally is not your only option.

If your treating doctor believes that you could benefit from treatment with Tryngolza (olezarsen), you can get it right away with a prescription.

Buy olezarsen for personal use

Thanks to a regulation known as Named Patient Import, patients around the world can import a medicine before it's approved or available in their country. In order to qualify for this regulation, the medicine in question needs to:

• be indicated for a life-threatening or debilitating disease;
• be prescribed by the patient's treating doctor;
• have approval somewhere in the world;
• have no alternatives in the patient's country.

Would you like to use the Named Patient Import regulation to start your treatment with olezarsen immediately? First talk to your doctor and get a suitable prescription.

Already have a prescription? Share it with our expert team via the button below. We'll help you buy Tryngolza immediately, before its EMA approval or MHRA approval. 

Request olezarsen

References:

1. TRYNGOLZA (olezarsen) approved in U.S. as first-ever treatment for adults living with familial chylomicronemia syndrome as an adjunct to diet. Ionis Pharmaceuticals, 19 December 2024.
2. Bergmark, Brian. Study: Olezarsen Effective Treatment for High Triglycerides. Mass General Brigham, 9 September 2024.
3. HIGHLIGHTS OF PRESCRIBING INFORMATION . Accessdata.fda.gov, Accessed 09 January 2025.
4. What is Olezarsen used for?. Synapse, 28 June 2024.
5. Rationale and Design of the Balance Study: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of Olezarsen in Patients with Familial Chylomicronemia Syndrome. Journal of Clinical Lipidology, Accessed 09 January 2025.
6. Ionis announces positive olezarsen topline results from Phase 3 study in people with familial chylomicronemia syndrome. Ionis Pharmaceuticals, 26 September 2023.
7. Ionis presents positive results from Phase 3 Balance study of olezarsen for familial chylomicronemia syndrome. Ionis Pharmaceuticals, 7 April 2024.
8. EFPIA Patients W.A.I.T. Indicator 2021 Survey. EFPIA, Accessed 09 January 2025.