Leniolisib's EMA approval: How to prevent delays from delaying your treatment

Last updated: 09 April 2024

For patients with activated PI3K-delta syndrome (APDS) 2023 brought some long-awaited good news. The first treatment for this ultrarare condition, leniolisib, got its FDA approval in March 2023. Considered by the FDA as a first-in-class medication, Joenja (leniolisib) is a beacon of hope to patients everywhere ¹.

However, as of April 2024, leniolisib is still only approved in the USA. Its MHRA and EMA approvals are still pending, leaving APDS patients in Europe and the UK with more questions than answers.

Here's everything you need to know about the current status of leniolisib's approval in Europe and the UK. Including all your options to safely access the medicine on your own timeline.

What is leniolisib used for?

Joenja (leniolisib) is indicated for adults and children with activated PI3K-delta syndrome (APDS). The medicine works by attaching to the phosphoinositide 3-kinase delta enzyme and blocking its activity. By doing so, it aims to support B and T cells in their function of fighting infections, and reduce symptoms ².

What were the results of the Joenja clinical trial?

The phase 2/3 trial which informed the FDA's approval decision for Joenja had a span of 12 weeks. Some of the reported results include:

Joenja performed better than placebo in reducing lymph node size. The adjusted mean change in lymph node size was -0.30 for Joenja, compared to +37.30 for placebo;
Patients treated with Joenja had a 34.76% increase in naive B cells. For placebo patients, the number of naive B cells decreased by 5.37%;
The most common adverse effects related to Joenja were headache, atopic dermatitis, and sinusitis ⁶.

Leniolisib's EMA approval: Delays and timelines

Joenja's marketing authorization application has been under review by the EMA since 27 October 2022. Typically, the EMA's approval process takes 210 days. However, in Joenja's case, it's taken longer, as the regulator asked for additional data in November 2023 ³.

The current indication from the CHMP is that it will make decision on leniolisib's approval in Europe somewhere in the first half of 2024 ³. If the CHMP issues a positive recommendation, the medicine's EMA approval should become official within 67 days from that date.

When will leniolisib be available in Europe?

To keep things interesting, an EMA approval does not mean leniolisib will be immediately available on the local markets. Every EU member state has its own local procedures to complete before that happens - including local price negotiations with the manufacturer and decisions on reimbursement.

How long these processes take varies greatly from one country to another. The average time from approval to availability ranges from 102 days in Germany to 1,081 days in Estonia, with an EU average of 636 days ⁴.

In other words, some patients in Europe may be able to access leniolisib already within 2024, while others may be left waiting for another couple of years.

Leniolisib in the UK: When will it be available?

Pharming, the manufacturer of Joenja, has stated its intention to apply for MHRA approval within the first half of 2024. According to current accelerated procedures, a decision on leniolisib's approval in the UK should be made within 110 days from submission.

Will leniolisib be available on the NHS?

This will depend on the NICE's review of the medicine. A decision on including leniolisib on the NHS is expected in December 2024 ⁵. It is of course dependent on a positive outcome of the application for MHRA approval.

If NICE recommends including Joenja on the NHS, the medicine should become available to patients within 3 months from the NICE's decision.

This takes us to March 2025 as the earliest date when APDS patients in the UK could access leniolisib.

How to safely get leniolisib before its MHRA and EMA approvals

Despite what the timelines above suggest, waiting is not the only option for APDS patients in Europe and the UK.

You could immediately access the medicine by importing it for your personal use. This option is known as Named Patient Import, or expanded access. Here's how it works.

Leniolisib expanded access

Even if a medicine is not yet approved or available in your country, you can legally access it. A regulation known as Named Patient Import allows you to buy and import a medicine, as long as it meets these conditions:

it is approved somewhere;
it is not approved or available in your country (yet);
it has no local alternatives, and
it is for personal use.

This process requires a prescription from your treating doctor. As in all other cases, the doctor assumes responsibility for the treatment. Per country, specific documentation requirements can vary.

Do you want to start your treatment with leniolisib sooner, using the Named Patient Import regulation? You will first need to consult your treating doctor and get a suitable prescription.

Already have a prescription? Share it with our team at Everyone.org. We can support you with buying leniolisib immediately.