Everything about eplontersen's EMA approval (and how to get the treatment sooner)

Last updated: 26 March 2024

Wainua (eplontersen) got its FDA approval to treat hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) in 2023. The medicine is the only approved treatment that patients can administer themselves, using an autoinjector. This could make it a more convenient choice for patients whose motor abilities have been impacted by the condition.

Although that's good news, the medicine is currently only accessible in the USA.

When will eplontersen be available to patients in Europe and the UK? Here's everything you need to know about eplontersen's EMA and MHRA approvals. Including timelines and your options to get the treatment faster.

What is eplontersen used for?

Wainua (eplontersen) is a prescription medicine for adult patients with polyneuropathy of hereditary transthyretin-mediated amyloidosis ¹.

Eplontersen is a ligand-conjugated antisense oligonucleotide. It decreases the production of TTR protein. By doing so, the medicine aims to slow the disease progression and improve the patient's quality of life.

Eplontersen EMA approval: What's the status?

The good news is, eplontersen's marketing authorization application is already under review by the EMA ². The less good news is that it can still take months before a decision is made. If the review results in a positive recommendation by the CHMP, it will take another 67 days for eplontersen's EMA approval to become official.

In other words, the earliest timeline for eplontersen to be approved in the EU is late 2024.

When will eplontersen be available in Europe?

Even if Wainua gets EMA approval within this year, that doesn't mean it will be immediately available across Europe. For that to happen, each member state needs to negotiate a local price, reimbursement, and launch dates with the manufacturer.

On average, these processes take from 133 days (in Germany) to 899 days (in Romania)³.

This means that eplontersen may be available in some EU markets in the first half of 2025. Other countries may need to wait a couple of years more.

When will eplontersen be available in the UK?

Although there is no active marketing authorization application for eplontersen in the UK, there is an ongoing review by NICE. NICE's review is essential to determine whether eplontersen will be available on the NHS. The expected decision date is in August 2024. If positive, the medicine should be available on the UK market within 3 months.

With regards to eplontersen's MHRA approval, it's possible that the MHRA is going to follow the EMA's timeline. According to decisions made by the UK government post-Brexit, the MHRA may grant "near automatic" approval to medicines after they've been green-lit by trusted regulatory bodies such as the EMA, FDA, or Japan's PMDA ⁴.

If that's the case, and assuming that the EMA's approval comes within 2024, it's possible that eplontersen becomes available in the UK by late-2024 or early 2025.

How to safely get Wainua before its EMA or MHRA approval

Although approvals are in motion, not all patients can wait for months or years. If that's your case, you'll be happy to learn that you don't have to wait.

There are two main ways for you to quickly and safely access eplontersen before its official EMA or MHRA approvals. One option is to join a clinical trial. The other is to buy eplontersen on the basis of the Named Patient Import regulation.

Here's a bit about each option.

Join an eplontersen clinical trial

To get quick access to Wainua, you can try joining an ongoing clinical trial. With your treating doctor's support, you can find one near you. In order to participate in a clinical trial, you must meet the eligibility criteria. Also important to note is that joining the trial doesn't guarantee you'll get eplontersen. You may be assigned to the placebo group.

Here are some good places to start looking for ongoing eplontersen clinical trials:

ClinicalTrials.gov: This is a database with all clinical trials in the USA. However, some of the trials are also open to international participants. At the moment, there are multiple trials involving eplontersen, which are recruiting participants in Europe. These may be worth keeping an eye on.
EUClinicaltrials.eu: This database contains all clinical trials in the European Union. Currently, it contains limited information on trials launched before 31 January 2022. For those trials, you can refer to the EU Clinical Trials Register.
myTomorrows: This organization supports patients in finding treatment options in clinical trials.

Buy eplontersen as a Named Patient

Patients in most countries are allowed to buy and import locally unavailable medicines for personal use. Especially if these medicines could improve their lives or address life-threatening conditions.

If you want to get eplontersen before its MHRA or EMA approval, this might be the quickest option for you and your doctor.

The regulation that makes this possible is known as the Named Patient Import regulation. It allows for patients to import a medication not yet approved or available in their country if: