Iptacopan's EMA approval is near. When will the medicine be available to you?

Last updated: 02 April 2024

In March 2024, the CHMP made a positive recommendation for granting Fabhalta (iptacopan) EMA approval ¹. When the approval becomes official, this will be the first oral monotherapy available to European patients with paroxysmal nocturnal hemoglobinuria (PNH).

While all of this is good news, what does it mean in terms of timeline?

Here's everything you need to know about iptacopan's availability in Europe and the UK. Including all your options to safely access the medicine before it's available.

What is iptacopan used for?

Fabhalta (iptacopan) is indicated for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH), who have haemolytic anaemia ².

As a complement Factor B inhibitor, iptacopan works to increase hemoglobin levels by acting in the alternative complement pathway. It helps contol the destruction of red blood cells within and outside the blood vessels. In this way, iptacopa aims to reduce anemia and prevent the need for blood transfusions.

Is Fabhalta (iptacopan) EMA approved?

It's likely to be soon. In March 2024, the CHMP made a positive recommendation for the EMA approval of iptacopan. Technically, this isn't yet an official EMA approval, as it can take up to 2 months for the European Commission to make a final decision.

However, CHMP decisions are typically followed, so there is no reason to believe that iptacopan won't get official EMA approval by May 2024.

When will iptacopan be available in Europe?

Unfortunately, getting EMA-approved isn't the same as being available. Before Fabhalta reaches the local markets throughout the EU, each individual member state has to complete their own process of local price negotiation, reimbursement approvals, and decide on lauch dates with the maufacturer.

These local processes can be relatively quick or very long, depending on the country. For example, Germany tends to launch newly approved orphan medicines within an average of 102 days, whereas in Estonia the average time to launch is 1,081 days ³.

With this in mind, it's likely that iptacopan will be available on some EU markets already within 2024, but patients in other EU countries may be facing a few more years of waiting time.

When will iptacopan be available in the UK?

As of March 2024, iptacopan is not yet approved by the MHRA. However, post-Brexit, the MHRA reserves the right to grant "near automatic" approval to medicines after they've been green-lit by the EMA or other trusted regulatory bodies ⁴. This in combination with the fact that iptacopan holds the MHRA's Innovation Passport, make it likely that approval for the medicine will be fast-tracked by the MHRA.

A review by NICE for potential inclusion of iptacopan on the NHS is already underway, with an expected decision date in June 2024 ⁵.

If NICE adopts a positive recommendation for including the medicine on the NHS in June, then iptacopan could be available to patients in the UK around September-October 2024. Of course, all of this assumes a final EMA approval in May 2024 and its near immediate adoption by the MHRA.

How to safely get Fabhalta (iptacopan) before it's available in your country

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 to get Fabhalta before it is available in your country. One way is to enroll in a clinical trial, while the other is to buy iptacopan via the Named Patient Import regulation.

Here's a bit about each option.

Join an iptacopan clinical trial

To get quick access to Fabhalta, you can opt to enroll in an ongoing clinical trial. Although trials aren't always easy to find, if you have the right resources and your doctor's support, it can be done. Keep in mind, though, that a trial doesn't guarantee you'll get iptacopan, as you may be assigned to the placebo group.

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

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 iptacopan, which are recruiting participants in Europe. This list 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 iptacopan as a Named Patient

Not all patients and doctors know that, but you are allowed to buy and import locally unavailable medicines for personal use. Especially if these medicines could improve your quality of life or address life-threatening conditions.

If you want to get iptacopan 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. To qualify for it, you first need to have a prescription from your treating doctor for a medicine that: