Joenja
Authorised
This medicine is authorised for use in the European Union
leniolisib
MedicineHumanAuthorised
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- Application under evaluation
- CHMP opinion
- European Commission decision
Overview
Joenja is a medicine used to treat activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents from 12 years of age and weighing 45 kg or more.
APDS is a rare, inherited disease in which the immune system (the body’s natural defences) does not work properly, making patients more susceptible to bacterial and viral infections. The condition can also lead to autoimmune disorders and lymphoma (cancer of lymphocytes, a type of white blood cell).
APDS is rare, and Joenja was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 19 October 2020. Further information on the orphan designation can be found on the EMA website.
Joenja contains the active substance leniolisib.
The medicine can only be obtained with a prescription. Treatment must be started by a doctor with experience in the management of primary immune deficiencies (when the immune system does not function properly).
Joenja is available as tablets to be taken by mouth twice a day, approximately 12 hours apart. Treatment should continue for as long as the patient benefits from it, or until side effects become unacceptable.
For more information about using Joenja, see the package leaflet or contact your doctor or pharmacist.
People with APDS have mutations (changes) in the genes that control the production of a protein called phosphoinositide 3-kinase delta. This protein is essential for the development and function of lymphocytes (B and T cells), which play a key role in the immune system. The mutations make the protein overactive, interfering with the normal development and function of lymphocytes. This leads to increased levels of immature B cells that build up in the lymph nodes (lymphadenopathy) and organs such as the spleen, liver or lungs, as well as reduced levels of naïve B cells (newly formed mature B cells involved in the initial immune response), affecting the patient’s ability to fight infections.
The active substance in Joenja, leniolisib, attaches to phosphoinositide 3-kinase delta and reduces its activity, leading to the normal development and function of B and T cells.
One main study involved 31 people from 12 years of age with APDS, who received either Joenja or placebo (a dummy treatment) along with standard treatment to manage symptoms of the condition. The study looked at the change in the level of lymphadenopathy and in the proportion of naïve B cells after 12 weeks of treatment.
People taking Joenja had a greater reduction in lymphadenopathy than those taking placebo, indicating a reduction in lymphoproliferation (abnormal production of lymphocytes). In addition, there was a greater increase in the proportion of naïve B cells in people taking Joenja than in those given placebo, indicating that B cell development may have normalised.
Studies carried out with Joenja are described in more detail in the medicine’s assessment report.
For the full list of side effects and restrictions with Joenja, see the package leaflet.
The most common side effects with Joenja (which may affect more than 1 in 10 people) include decreased levels of neutrophils (a type of white blood cell), headache, vomiting, increased weight and alopecia (hair loss).
Joenja was shown to be effective at reducing lymphadenopathy and increasing the number of naïve B cells in people with APDS, which is expected to reduce the risk of infection and other complications associated with the condition. The safety profile was considered acceptable, with generally manageable side effects. The European Medicines Agency therefore decided that Joenja’s benefits are greater than its risks and that it can be authorised for use in the EU.
Joenja has been authorised under ‘exceptional circumstances’. This is because it has not been possible to obtain complete information about Joenja due to the rarity of the disease. The company must provide further data on Joenja. It must submit the results from a registry-based study on the long‑term safety and effectiveness of Joenja, and must provide yearly updates on any new information concerning the medicine’s safety and effectiveness. Every year, the Agency will review any new information that becomes available.
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Joenja have been included in the summary of product characteristics and the package leaflet.
As for all medicines, data on the use of Joenja are continuously monitored. Suspected side effects reported with Joenja are carefully evaluated and any necessary action taken to protect patients.
Joenja received a marketing authorisation under exceptional circumstances valid throughout the EU on 21 May 2026.
Product information
This medicine’s product information is available in all official EU languages.
Select 'available languages' to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
Product details
- Name of medicine
- Joenja
- Active substance
- leniolisib phosphate
- International non-proprietary name (INN) or common name
- leniolisib
- Therapeutic area (MeSH)
- Primary Immunodeficiency Diseases
- Anatomical therapeutic chemical (ATC) code
- L03AX22
Pharmacotherapeutic group
Antineoplastic agentsTherapeutic indication
Joenja is indicated for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents 12 years of age and older and weighing 45 kg or more.
News on Joenja
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