Pharming Group provides update on ongoing regulatory review of leniolisib for the treatment of APDS in the European Union
Pharming Group has provided an update on the regulatory review of leniolisib for treating APDS in the EU. The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has affirmed the clinical benefit and safety of leniolisib but requested further clarification on manufacturing controls before approval.
Pharming has been granted an extension to January 2026 to fulfill these requirements. Despite the delay, leniolisib has shown positive efficacy and safety in clinical trials, meeting co-primary endpoints and benefiting patients in long-term studies. The drug is already approved in the US since March 2023. Pharming maintains its 2024 revenue guidance.
- CHMP affirmed the positive clinical benefit and safety of leniolisib.
- Leniolisib met co-primary endpoints in Phase II/III trials.
- Over 300 patient-years of treatment demonstrated continued efficacy and safety.
- Leniolisib is already approved by the US FDA since March 2023.
- Pharming maintains its 2024 total revenue guidance.
- CHMP issued a List of Outstanding Issues requiring additional manufacturing controls.
- The regulatory review has been delayed, with an extension to January 2026.
- Approval in the European market has been postponed.
Insights
The update on leniolisib's regulatory review reveals critical insights into its market potential in the European Union. The CHMP affirming the positive clinical benefit and safety of leniolisib aligns with the assessments of the Ad Hoc Expert Group, highlighting the drug's efficacy. Leniolisib's ability to meet the co-primary endpoints in a Phase II/III clinical trial and its safety profile demonstrated over 300 patient-years, underlines its potential impact in treating APDS, a rare and severe condition.
However, the delay due to the outstanding CMC request points to the stringent regulatory landscape in the EU, especially concerning the definition of regulatory starting materials in the manufacturing process. Pharming's prompt action to address these concerns pre-approval indicates their commitment to meet these high standards, though it inevitably postpones market entry. This delay might impact the immediate availability of the treatment to patients in Europe but ensures enhanced quality and regulatory compliance.
For retail investors, understanding the rigorous standards set by the EMA is important as it reflects the company's long-term reliability and dedication to patient safety. This could build confidence in Pharming's portfolio and operational diligence.
The delay in the approval of leniolisib for the European market naturally affects Pharming's short-term revenue projections in this region. However, maintaining its 2024 revenue guidance suggests that the company has accounted for such regulatory hurdles in its financial planning. Investors should note that leniolisib has already secured FDA approval and is available commercially in the United States, providing a revenue stream that can mitigate the impact of the EU delay.
Furthermore, securing an extension until January 2026 to meet the CHMP's CMC request could be seen as a strategic move to ensure a robust market entry once approval is granted. This extension allows Pharming to enhance its manufacturing processes and quality controls, which could lead to a stronger market position upon approval. Investors should watch for updates on the progress of these manufacturing activities, as successful completion could significantly boost Pharming's market share and growth potential in the EU.
Overall, the company's transparency and proactive approach in dealing with regulatory challenges are positive signals of its management's competence and strategic foresight.
Leniolisib addresses an unmet medical need for APDS, a rare disease, thus positioning Pharming in a niche but vital segment of the pharmaceutical market. The positive clinical data and ongoing real-world evidence strengthen leniolisib's case for approval and potential market adoption. The drug's availability in the US and the regulatory journey in the EU highlight the complexities and opportunities in global pharmaceutical markets.
From a market perspective, the delay could give competitors time to advance their treatments, if any, for APDS. However, Pharming's commitment to resolving the CMC issues pre-approval demonstrates a strategic focus on long-term market sustainability and compliance. This approach could enhance Pharming's brand reputation among stakeholders, including patients, healthcare providers and regulatory bodies.
For investors, this update underscores the importance of monitoring regulatory processes and the strategic responses of pharmaceutical companies to such challenges. Understanding the dynamics of drug approval timelines and their impact on market entry can provide deeper insights into the potential risks and rewards associated with investment in biotech firms.
Leiden, the Netherlands, May 30, 2024: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces today an update on the ongoing review of its Marketing Authorisation Application (MAA) for leniolisib for the treatment of adult and pediatric patients 12 years of age and older with activated phosphoinositide 3-kinase delta syndrome (APDS) by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).
Following the May 27-30 CHMP meeting, Pharming received an updated List of Outstanding Issues (LoOI) from the CHMP. The LoOI affirmed the positive clinical benefit and safety of leniolisib, in agreement with the assessment by the Ad Hoc Expert Group (AEG), and included one remaining chemistry, manufacturing and controls (CMC) request.
The CMC request relates to the definition of regulatory starting materials used in the manufacturing process for leniolisib. As Pharming is committed to meeting all of the CHMP’s specific requirements, additional data and quality controls were provided and Pharming proposed implementation of the CMC request post-approval. The CHMP requested that this work be completed pre-approval and has granted Pharming an extension to January 2026 to submit a response. Pharming has already initiated the manufacturing activities requested by the CHMP, which it plans to complete prior to this deadline.
Sijmen de Vries, MD, Chief Executive Officer of Pharming, commented:
“While we are understandably disappointed by the delay to the European license, we are pleased that the CHMP determined the clinical benefit of leniolisib to be positive. The efficacy and safety demonstrated in clinical trials and ongoing real-world experience, with over 300 patient-years of treatment, support that leniolisib fills an unmet medical need. We will continue to work closely with the EMA and CHMP to obtain approval for leniolisib in Europe for people living with APDS. In the meantime, all our clinical development and early access programs will continue.”
The MAA for leniolisib was based on findings from a multinational, triple-blind, placebo-controlled, randomized Phase II/III clinical trial, which met both its co-primary endpoints. The trial evaluated efficacy and safety in 31 patients diagnosed with APDS aged 12 years and older. Also submitted as part of the application were data from a long-term, open-label extension clinical trial in which 37 patients received leniolisib for a median of three years.
Leniolisib is currently available commercially in the United States. The US Food and Drug Administration (FDA) approved leniolisib in March 2023, based on its assessment that leniolisib met clinical and manufacturing standards.
Pharming is maintaining its previously provided 2024 total revenue guidance.
About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)
APDS is a rare primary immunodeficiency that was first characterized in 2013. APDS is caused by variants in either one of two identified genes known as PIK3CD or PIK3R1, which are vital to the development and function of immune cells in the body. Variants of these genes lead to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway, which causes immune cells to fail to mature and function properly, leading to immunodeficiency and dysregulation1,2,3 APDS is characterized by a variety of symptoms, including severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.4,5 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, it has been reported that people with APDS are frequently misdiagnosed and suffer a median 7-year diagnostic delay.6 As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma.4-7 A definitive diagnosis can be made through genetic testing. APDS affects approximately 1 to 2 people per million worldwide.
About leniolisib
Leniolisib is an oral small molecule phosphoinositide 3-kinase delta (PI3Kẟ) inhibitor approved in the US and Israel as the first and only targeted treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Leniolisib inhibits the production of phosphatidylinositol-3-4-5-trisphosphate, which serves as an important cellular messenger and regulates a multitude of cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Results from a randomized, placebo-controlled Phase II/III clinical trial demonstrated clinical efficacy of leniolisib in the coprimary endpoints; demonstrating statistically significant impact on immune dysregulation and normalization of immunophenotype within these patients, and interim open label extension data has supported the safety and tolerability of long-term leniolisib administration.8,9 Leniolisib is currently under regulatory review in the European Economic Area, the U.K., Canada and Australia, with plans to pursue further regulatory approvals in Japan and South Korea. Leniolisib is also being evaluated in two Phase III clinical trials in children with APDS.
About Pharming Group N.V.
Pharming Group N.V. (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines, including small molecules and biologics. Pharming is headquartered in Leiden, the Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific.
For more information, visit www.pharming.com and find us on LinkedIn.
Forward-Looking Statements
This press release may contain forward-looking statements. Forward-looking statements are statements of future expectations that are based on management’s current expectations and assumptions and involve known and unknown risks and uncertainties that could cause actual results, performance, or events to differ materially from those expressed or implied in these statements. These forward-looking statements are identified by their use of terms and phrases such as “aim”, “ambition”, ‘‘anticipate’’, ‘‘believe’’, ‘‘could’’, ‘‘estimate’’, ‘‘expect’’, ‘‘goals’’, ‘‘intend’’, ‘‘may’’, “milestones”, ‘‘objectives’’, ‘‘outlook’’, ‘‘plan’’, ‘‘probably’’, ‘‘project’’, ‘‘risks’’, “schedule”, ‘‘seek’’, ‘‘should’’, ‘‘target’’, ‘‘will’’ and similar terms and phrases. Examples of forward-looking statements may include statements with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, and Pharming's expectations regarding its projected working capital requirements and cash resources, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Pharming's clinical trials and ramifications for the cost thereof; and clinical, scientific, regulatory, commercial, competitive and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming's 2023 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2023, filed with the U.S. Securities and Exchange Commission, the events and circumstances discussed in such forward-looking statements may not occur, and Pharming's actual results could differ materially and adversely from those anticipated or implied thereby. All forward-looking statements contained in this press release are expressly qualified in their entirety by the cautionary statements contained or referred to in this section. Readers should not place undue reliance on forward-looking statements. Any forward-looking statements speak only as of the date of this press release and are based on information available to Pharming as of the date of this release. Pharming does not undertake any obligation to publicly update or revise any.
Inside Information
This press release relates to the disclosure of information that qualifies, or may have qualified, as inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation.
References
- Lucas CL, et al. Nat Immunol. 2014;15(1):88-97.
- Elkaim E, et al. J Allergy Clin Immunol. 2016;138(1):210-218.
- Nunes-Santos C, Uzel G, Rosenzweig SD. J Allergy Clin Immunol. 2019;143(5):1676-1687.
- Coulter TI, et al. J Allergy Clin Immunol. 2017;139(2):597-606.
- Maccari ME, et al. Front Immunol. 2018;9:543.
- Jamee M, et al. Clin Rev Allergy Immunol. 2020 Dec;59(3):323-333.
- Condliffe AM, Chandra A. Front Immunol. 2018;9:338.
- Rao VK, et al Blood. 2023 Mar 2;141(9):971-983.
- Rao VK, et al. J Allergy Clin Immunol 2024;153:265-74.
For further public information, contact:
Pharming Group, Leiden, The Netherlands
Michael Levitan, VP Investor Relations & Corporate Communications
T: +1 (908) 705 1696
E: investor@pharming.com
FTI Consulting, London, UK
Victoria Foster Mitchell/Alex Shaw/Amy Byrne
T: +44 203 727 1000
LifeSpring Life Sciences Communication, Amsterdam, The Netherlands
Leon Melens
T: +31 6 53 81 64 27
E: pharming@lifespring.nl
US PR
Christina Renfroe
E: Christina.Renfroe@precisionvh.com
T: +1 (636) 352-7883
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