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

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 November 2024, leniolisib is still only approved in the USA and the UK. Its EMA approval is still pending, leaving APDS patients in Europe with more questions than answers. 

Here's everything you need to know about the current status of leniolisib's approval in Europe. 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 manufacturer, Pharming, has until January 2026 to respond to the additional requests from the CHMP. Meaning that any EMA approval for leniolisib is unlikely to happen before 2026. 

If the CHMP issues a positive recommendation at that point, the medicine's EMA approval should become official within 67 days from that date. In short, European patients should start having access to leniolisib somewhere in the second hald of 2026. 

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 2026, while others may be left waiting for another couple of years.

Leniolisib in the UK: When will it be available? 

In September 2024, leniolisib was approved in the UK. 

Will leniolisib be available on the NHS?

Although leniolisib is now approved by the MHRA, it is not yet available on the NHS. Whether and when this happens will depend on the NICE's review of the medicine. A decision on including leniolisib on the NHS is expected in March 2025 ⁵.  

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

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

How to safely get leniolisib before its EMA approval

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

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.

Buy leniosilib

References:

1. FDA approves first treatment for activated phosphoinositide 3-kinase delta syndrome. FDA, 24 March 2023.
2. EU/3/20/2339 - orphan designation for treatment of activated phosphoinositide 3-kinase delta syndrome | European Medicines Agency. European Medicines Agency, 25 May 2021.
3. Pharming Group provides updates on EMA regulatory review of leniolisib MAA and plans to file for UK regulatory approval. Pharming Group N.V., 10 November 2023.
4. EFPIA Patients W.A.I.T. Indicator 2021 Survey. EFPIA, Accessed 9 April 2024.
5. Leniolisib for activated phosphoinositide 3-kinase delta syndrome in people 12 years and over [ID6130]. NICE, Accessed 09 April 2024.
6. Study of Efficacy of CDZ173 in Patients With APDS/​PASLI. ClinicalTrials.gov, Accessed 09 April 2024.