This section highlights FDA-related milestones and regulatory updates for drugs developed by Pharming Group (PHAR).
Over the past two years, Pharming Group has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Leniolisib and RUCONEST. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
Leniolisib FDA Regulatory Timeline and Events
Leniolisib is a drug developed by Pharming Group for the following indication: Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Leniolisib
- Announced Date:
- March 27, 2026
- Indication:
- Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Announcement
Pharming Group N.V. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending marketing authorization for Joenja® (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, in adult and pediatric patients 12 years of age and older.
AI Summary
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization for Joenja® (leniolisib) to treat activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, in adult and pediatric patients 12 years and older. The CHMP recommendation advances the drug toward formal approval in the European Union for this specific, serious immune disorder.
A final decision by the European Commission is expected in about two months. If approved under exceptional circumstances, Joenja would be the first authorized treatment for APDS in the EU. The centralized marketing authorization would apply across the 27 EU Member States as well as Norway, Iceland and Liechtenstein. Leniolisib is also under regulatory review in Canada and other countries and is being studied in two Phase II trials for primary immunodeficiencies with immune dysregulation; its safety and efficacy beyond APDS have not been established.
Read Announcement- Drug:
- Leniolisib
- Announced Date:
- March 24, 2026
- Indication:
- Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Announcement
Pharming Group N.V. announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has approved Joenja® (leniolisib), an oral, selective phosphoinositide 3-kinase (PI3K) delta inhibitor, for the treatment of activated PI3K delta syndrome (APDS) in adult and pediatric patients aged 4 years and older.
AI Summary
Pharming Group N.V. announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved Joenja® (leniolisib), an oral, selective phosphoinositide 3-kinase (PI3K) delta inhibitor, for the treatment of activated PI3K delta syndrome (APDS) in adults and pediatric patients aged 4 years and older. The Japanese Pharmaceuticals and Medical Devices Agency (PMDA) evaluated the application under the Priority Review pathway after the MHLW granted Orphan Drug Designation in May 2023.
Leniolisib is also under regulatory review in the European Economic Area, Canada and several other countries. The drug is being studied in two Phase II clinical trials for primary immunodeficiencies with immune dysregulation. Regulators and investigators note that the safety and efficacy of leniolisib have not been established for immune dysregulation conditions beyond APDS, and ongoing studies will help define its broader use.
Read Announcement- Drug:
- Leniolisib
- Announced Date:
- February 1, 2026
- Indication:
- Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Announcement
Pharming Group announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) to its supplemental New Drug Application (sNDA) for Joenja® (leniolisib), an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency.
AI Summary
Pharming Group announced the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) to its supplemental New Drug Application (sNDA) for Joenja (leniolisib), an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency.
The CRL identified concerns related to clinical pharmacology and batch testing methodology. Pharming said it believes these issues can be addressed and plans to work closely with the FDA to meet the agency’s requirements. The company intends to request a Type A meeting with the FDA to discuss next steps and the path for resubmission after resolving the cited issues.
Read Announcement- Drug:
- Leniolisib
- Announced Date:
- October 1, 2025
- Indication:
- Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Announcement
Pharming Group N.V. announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) seeking approval for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency.
AI Summary
Pharming Group N.V. said the U.S. Food and Drug Administration has accepted its supplemental New Drug Application for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor designed to treat children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS). This rare primary immunodeficiency weakens the immune system and often leads to recurrent infections and serious complications.
The FDA granted Priority Review to the application and set a Prescription Drug User Fee Act (PDUFA) target action date of January 31, 2026. The submission is based on positive results from a multinational Phase III study showing that 12 weeks of leniolisib reduced lymphadenopathy and increased naïve B cells, indicating correction of the underlying immune defect. It also included safety data from eight months of treatment, confirming a manageable safety profile.
If approved, leniolisib would become the first and only treatment indicated for children under 12 with APDS in the United States, offering a targeted therapy option for young patients.
Read Announcement- Drug:
- Leniolisib
- Announced Date:
- April 23, 2025
- Indication:
- Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Announcement
Pharming Group N.V. announces that the National Institute for Health and Care Excellence (NICE) has issued positive final guidance recommending Joenja® (leniolisib) for reimbursement and use within the National Health Service (NHS) in England and Wales for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older.
AI Summary
Pharming Group N.V. announced that the National Institute for Health and Care Excellence (NICE) has given positive final guidance recommending Joenja® (leniolisib) for reimbursement and use within the NHS in England and Wales. This decision marks an important milestone because Joenja® is the first and only medication approved specifically to treat activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in patients aged 12 and older. The positive recommendation followed robust Phase III clinical trial data showing that leniolisib, an oral selective PI3Kδ inhibitor, significantly improved immune deficiency and dysregulation compared to placebo. As a result, patients in England can access this breakthrough treatment immediately, while funding is expected for Wales in the coming months. This approval underlines Pharming’s commitment to offering targeted therapies for rare and progressive immunodeficiencies, aiming to enhance patient outcomes and quality of life.
Read Announcement- Drug:
- Leniolisib
- Announced Date:
- March 19, 2025
- Indication:
- Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Announcement
Pharming Group N.V. announces that the first patient has been dosed in a Phase II, proof of concept, clinical trial evaluating leniolisib in common variable immunodeficiency (CVID) patients with immune dysregulation.
AI Summary
Pharming Group N.V. announced that the first patient has been dosed in a Phase II, proof-of-concept clinical trial using leniolisib for common variable immunodeficiency (CVID) patients with immune dysregulation. This open-label, single-arm, dose range-finding study will enroll around 20 patients aged 12 and older who have a CVID diagnosis along with evidence of lymphoproliferation and an additional clinical manifestation, such as autoimmune cytopenias, interstitial lung disease, or enteropathy. The trial aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of leniolisib, and explore its clinical efficacy in this patient population.
Led by Dr. Jocelyn Farmer of Beth Israel Lahey Health, the study will take place at clinical sites in the US, UK, and EU. The results from this trial are expected to guide a future Phase III program, offering a potential new treatment option for CVID patients who currently face significant unmet medical needs.
Read Announcement- Drug:
- Leniolisib
- Announced Date:
- December 11, 2024
- Indication:
- Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Announcement
Pharming Group N.V announces positive topline results of data from its Phase III clinical trial (NCT05438407) evaluating the investigational drug leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, in children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS).
AI Summary
Pharming Group N.V. announced positive topline results from its Phase III clinical trial (NCT05438407) evaluating leniolisib in children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS). Leniolisib is an oral, selective PI3Kδ inhibitor aimed at treating this rare primary immunodeficiency. In the trial, all 21 enrolled children completed the 12-week treatment period and showed improvements in key disease markers, including a reduction in lymph node size and an increase in the fraction of naïve B cells, indicating immunophenotype correction. These findings were consistent across multiple dosing levels and built upon prior data observed in adolescent and adult APDS patients. Pharming plans to include these results in its global regulatory filings for potential approval in pediatric patients starting in 2025.
Read Announcement- Drug:
- Leniolisib
- Announced Date:
- December 11, 2024
- Indication:
- Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Announcement
Pharming Group N.V announced that Global regulatory filings planned to begin in 2025
AI Summary
Pharming Group N.V. announced encouraging Phase III trial results for leniolisib tablets in children aged 4 to 11 with activated phosphoinositide 3-kinase delta syndrome (APDS). The trial data showed improvements in key markers of the disease, such as reduced lymph node size and a healthier immune cell profile, suggesting that leniolisib may address critical aspects of APDS in this younger population.
The company revealed that it plans to use the positive data from this trial as part of its global regulatory filings, which are scheduled to begin in 2025. Pharming’s strategy aims to expand treatment options for pediatric patients with APDS, a rare and serious immunodeficiency disorder, ensuring that children under 12 have access to targeted disease-modifying therapy.
Read Announcement- Drug:
- Leniolisib
- Announced Date:
- October 10, 2024
- Indication:
- Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Announcement
Pharming Group N.V announces the start of a Phase II, proof of concept, clinical trial evaluating leniolisib in primary immunodeficiencies (PIDs) with immune dysregulation linked to altered PI3Kẟ signaling in lymphocytes.
AI Summary
Pharming Group N.V. has announced the start of a Phase II, proof of concept clinical trial studying the use of leniolisib for primary immunodeficiencies (PIDs) with immune dysregulation linked to altered PI3Kδ signaling in lymphocytes. The trial will enroll patients with conditions such as ALPS-FAS, CTLA4 haploinsufficiency, NFKB1 haploinsufficiency, and PTEN deficiency. These conditions share similar immune and clinical features with activated phosphoinositide 3-kinase delta syndrome (APDS), though they have a prevalence about five times higher than APDS.
This open-label, single-arm study aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of leniolisib, while also exploring its clinical efficacy. Conducted at the National Institutes of Health, the trial intends to enroll approximately 12 patients, with results expected to inform a later Phase III study.
Read Announcement- Drug:
- Leniolisib
- Announced Date:
- September 26, 2024
- Indication:
- Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Announcement
Pharming Group N.V. announces that the United Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorization for Joenja® (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older.
AI Summary
Pharming Group N.V. announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorization for its treatment Joenja® (leniolisib). This approval is a significant step for patients with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare and progressive primary immunodeficiency disorder. Joenja® is approved for adult and pediatric patients aged 12 years and older and is the first treatment in the UK specifically indicated for APDS.
Leniolisib is an oral, selective PI3Kδ inhibitor designed to target the underlying immune dysregulation in APDS patients. Until now, treatment for APDS mainly involved managing its various symptoms, and in some cases, patients needed to undergo hematopoietic stem cell transplantation. The MHRA authorization marks an important milestone and offers a new treatment option for those affected by this debilitating disease.
Read Announcement
RUCONEST FDA Regulatory Events
RUCONEST is a drug developed by Pharming Group for the following indication: hereditary angioedema (HAE) treatment.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- RUCONEST
- Announced Date:
- October 20, 2025
- Indication:
- hereditary angioedema (HAE) treatment.
Announcement
Pharming Group N.V. announced that 12 abstracts have been accepted for presentation at the American College of Allergy, Asthma & Immunology (ACAAI) 2025 Annual Scientific Meeting taking place in Orlando, Florida on November 6-10.
AI Summary
Pharming Group N.V. announced that 12 abstracts have been accepted for presentation at the American College of Allergy, Asthma & Immunology (ACAAI) 2025 Annual Scientific Meeting in Orlando, Florida, November 6–10, 2025.
Five posters will present new clinical, economic, and comparative data for RUCONEST® (recombinant C1 esterase inhibitor), highlighting its role in on-demand treatment of hereditary angioedema (HAE). These studies include indirect treatment comparisons, cost-effectiveness analyses, re-analysis of clinical data, patient-reported outcome measures, and clinician perspectives on HAE therapy trials.
Seven posters will showcase real-world evidence for Joenja® (leniolisib) in activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS). They feature results from a Phase III pediatric trial, infection reduction, healthcare utilization, quality of life, caregiver burden, adherence and persistence, and registry data. All ePosters will be available online and on demand for registered attendees beginning November 6.
Read Announcement
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