Joenja

Opinion

EMA has issued an opinion on this medicine

leniolisib

Overview

On 26 March 2026, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation under exceptional circumstances for the medicinal product Joenja, intended 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.

The applicant for this medicinal product is Pharming Technologies B.V.

Joenja will be available as 70 mg film-coated tablets. The active substance of Joenja is leniolisib, an immunostimulant (ATC code: L03AX22). APDS is a complex primary immunodeficiency disease caused by a mutation in either the PIK3CD or PIK3R1 gene. Leniolisib is a PI3Kδ inhibitor targeting the catalytic p110δ subunit of PI3Kδ, thereby specifically inhibiting the hyperactivated PI3K pathway, which underlies APDS. As a result, deficiencies and dysregulation of B and T cell populations normalise.

The benefits of Joenja are an improvement in lymphadenopathy (swollen/enlargement of lymph nodes), and immunophenotype normalization (increase in percentage of naïve B-cells out of total B-cells), as observed in a double-blind, randomised, placebo-controlled study in 31 patients with APDS. Improvements were also observed on spleen parameters (reduced mean sum of product diameter volume of index lesion and mean bi-dimensional size and volume of spleen), but an effect on reducing infections frequency could not be shown.

The most common side effects are headache, vomiting, alopecia, weight increased and neutrophil count decreased.

The full indication is:

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.

Treatment with Joenja should be initiated by physicians experienced in the management of primary immune deficiencies.

Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published on the EMA website in all official European Union languages after the marketing authorisation has been granted by the European Commission.

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Notes:

• In exceptional circumstances, an authorisation may be granted subject to certain specific obligations, to be reviewed annually. This happens when the applicant can show that they are unable to provide comprehensive data on the efficacy and safety of the medicinal product, due to the rarity of the condition it is intended for, limited scientific knowledge in the area concerned, or ethical considerations involved in the collection of such data.

• This product was designated as an orphan medicine during its development. EMA will now review the information available to date to determine if the orphan designation can be maintained

CHMP summary of positive opinion for Joenja

Adopted Reference Number: EMA/CHMP/28350/2026

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
• EMA product number:
  EMEA/H/C/005927
• Orphan:

  This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

• Marketing authorisation applicant:
  Pharming Technologies B.V.
• Opinion adopted:
  26/03/2026
• Opinion status:
  Positive