FDA Events for Pharming Group (PHAR)
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. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
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:
- 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- Drug:
- Leniolisib
- Announced Date:
- April 6, 2024
- Indication:
- Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Announcement
Pharming Group N.V announces the completion of patient enrollment in 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 that it has completed patient enrollment in its Phase III trial (NCT05438407) for leniolisib. The study focuses on children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency. Leniolisib, an oral, selective PI3Kδ inhibitor, is being evaluated for its safety, tolerability, and effectiveness in reducing lymph node size and increasing the proportion of naïve B cells. The trial enrolled 21 children across sites in the United States, Europe, and Japan. Data from this trial will be used in global regulatory filings starting in 2025 to support the approval of leniolisib for younger pediatric patients with APDS. This study is significant as it aims to intervene early in life to potentially prevent progressive immune-related complications in children with the syndrome.
Read Announcement
Pharming Group FDA Events - Frequently Asked Questions
As of now, Pharming Group (PHAR) has not received any FDA approvals for its therapy in the last two years.
In the past two years, Pharming Group (PHAR) has reported FDA regulatory activity for Leniolisib.
The most recent FDA-related event for Pharming Group occurred on April 23, 2025, involving Leniolisib. The update was categorized as "Guidance," with the company reporting: "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."
Currently, Pharming Group has one therapy (Leniolisib) targeting the following condition: Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS).
More FDA Event Resources from MarketBeat
FDA Event Stage Terminology & Abbreviation Guide
- NDA: New Drug Application
- ANDA: Abbreviated New Drug Application
- sNDA: Supplemental New Drug Application
- BLA: Biologics License Application
- sBLA: Supplemental Biologics License Application
- FDA Approved: Approved by the FDA
- EMA: European Medicines Agency
- CE Mark: European Union Certification
- NMPA: China National Medical Products Administration
- MHLW: Japanese Ministry of Health
- FDA Meeting: Consultation with FDA
- Pre-IND: Pre-Investigational New Drug Meeting
- Breakthrough Therapy: Special FDA designation for promising therapies
- Fast Track: Accelerated FDA approval pathway
- Orphan Drug: Designation for rare disease treatments
- RPD: Rare Pediatric Disease Designation
- RMAT: Regenerative Medicine Advanced Therapy
- DSMB Review: Data Safety Monitoring Board Review
- IDMC Review: Independent Data Monitoring Committee
- MAA: MHRA Marketing Authorization Application
- RTF: Refusal to File (Rejected Application)
- 510(k): FDA Clearance for Medical Devices
- Rolling Submission: Staggered regulatory review process
This page (NASDAQ:PHAR) was last updated on 7/13/2025 by MarketBeat.com Staff
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