Pharming Group N.V. develops and commercializes medicines for rare and ultra-rare diseases, with news focused on RUCONEST®, Joenja® (leniolisib) for activated phosphoinositide 3-kinase delta syndrome, and the company's rare-disease pipeline. Updates commonly cover clinical studies, market authorizations, commercial launches, licensing economics, product revenue trends, and management presentations at healthcare investor conferences.
The company operates as a global biopharmaceutical issuer listed on Euronext Amsterdam and Nasdaq. Recurring coverage also includes financial results, regulatory interactions in major markets, and development programs involving small molecules, biologics, protein replacement therapies, and precision medicines for immunological and genetic conditions.
Pharming Group N.V. announced the publication of positive results from a Phase 3 trial of leniolisib, an innovative treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), in the journal Blood. The trial demonstrated significant improvements in co-primary endpoints, including a reduction in lymph node size and an increase in naïve B cells. Leniolisib was well tolerated with fewer treatment-related adverse events compared to placebo. This publication enhances understanding of APDS, a rare immunodeficiency affecting 1-2 individuals per million, and underscores Pharming's commitment to advancing therapies in this field.
Pharming Group N.V. announced that new clinical data for leniolisib, a PI3Kδ inhibitor, will be presented at the 64th ASH Annual Meeting in New Orleans from December 10-13, 2022. This investigational treatment targets activated PI3Kδ syndrome (APDS), a rare primary immunodeficiency affecting 1-2 people per million. The presentation will include an interim analysis of safety and hematological parameters through December 2021. Leniolisib has been well tolerated in prior trials, indicating its potential as a therapeutic option for APDS.
Pharming Group N.V. (PHAR, PHGUF) announced that the European Medicines Agency (EMA) has validated its Marketing Authorisation Application (MAA) for leniolisib, an oral treatment for activated phosphoinositide 3-kinase delta syndrome (APDS). The MAA, submitted in October 2022, will undergo an accelerated assessment, reducing the review period from 210 to 150 days. Positive Phase II/III study results showed leniolisib effectively reduced lymph node size and increased naïve B cell percentage in patients. Marketing authorisation is expected in H1 2023.
Pharming Group N.V. has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for leniolisib, an oral treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency. The EMA has granted accelerated assessment, shortening the review period from 210 to 150 days. Leniolisib is aimed at treating patients aged 12 and older and has shown positive results in previous studies. Approval is anticipated in H1 2023, addressing a critical need for effective therapies in this patient population.
Pharming Group N.V. announced that the FDA has accepted its New Drug Application (NDA) for leniolisib, a treatment for activated phosphoinositide 3-kinase delta syndrome (APDS). The FDA set a PDUFA goal date of March 29, 2023. The NDA was supported by positive results from a Phase II/III study, showing significant efficacy and safety in patients. Leniolisib is aimed at providing a targeted therapy for a rare disease affecting approximately 1 to 2 individuals per million, currently managed with supportive therapies.
Pharming Group N.V. announced that CEO Sijmen de Vries will present at the H.C. Wainwright 24th Annual Global Investment Conference in New York, scheduled for September 12-14, 2022. De Vries will speak on September 13 at 09:30 ET (15:30 CET). Pharming focuses on developing innovative protein replacement therapies and precision medicines for rare diseases, with a global presence in over 30 markets. Interested parties can contact Pharming's Investor Relations for more information regarding the conference.
Pharming Group N.V. has announced the introduction of a new diagnosis code for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, effective October 1, 2022. This ICD-10-CM code, introduced by the US CDC, will allow better identification and reporting of APDS cases, improving patient care and research opportunities. The condition, affecting 1-2 people per million, is often misdiagnosed, leading to treatment delays. The recognition of APDS is expected to enhance access to meaningful treatments for those affected.
Pharming Group N.V. announced that the European Medicines Agency (EMA) has granted an accelerated assessment for leniolisib, reducing the review period from 210 days to 150 days. This medication targets activated PI3K delta syndrome (APDS), a rare primary immunodeficiency. Pharming plans to submit its Marketing Authorisation Application (MAA) in October 2022. Clinical trials showed leniolisib met its co-primary endpoints and was well-tolerated, indicating potential for significant impact on patient health in Europe.
Pharming Group N.V. has received a positive decision from the UK's MHRA regarding its Paediatric Investigation Plan (PIP) for leniolisib, aimed at treating activated phosphoinositide 3-kinase delta syndrome (APDS) in children aged 1 to under 18. Additionally, leniolisib has been granted Promising Innovative Medicine (PIM) designation, enabling potential access through the MHRA's Early Access to Medicines Scheme. The PIP includes two planned global clinical trials, with recruitment expected to begin in the second half of 2022. Pharming aims to file for global registration and launch leniolisib in the US and Europe in 2023.
Pharming Group N.V. announced positive data from the Phase II/III trial of leniolisib for activated phosphoinositide 3-kinase delta syndrome (APDS) at the Clinical Immunology Society (CIS) 2022 Annual Meeting. The trial met its co-primary endpoints, showing a significant reduction in lymph node size and an increase in naive B cells among treated patients. The study drug was well-tolerated with no treatment-related serious adverse events. Pharming plans to submit global regulatory filings for leniolisib in Q2 2022 and aims for U.S. and European launches in 2023.