Pharming submits a Marketing Authorisation Application to the European Medicines Agency for Leniolisib

Rhea-AI Summary

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.

Positive

• Submission of MAA for leniolisib indicates progress in drug development.
• Accelerated assessment by EMA highlights leniolisib's potential public health impact.
• Positive Phase II/III study data supports the effectiveness and tolerability of leniolisib.

Negative

• None.

Application is based on randomized, controlled and long-term extension data for leniolisib as a treatment for APDS, a rare primary immunodeficiency 

This submission follows the grant of accelerated assessment allowing an expedited review for leniolisib from a standard 210 days to 150 days

LEIDEN, Netherlands, Oct. 11, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that it has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, in adults and adolescents 12 years or older. 

On August 1, 2022, Pharming announced the leniolisib MAA was granted accelerated assessment by EMA's Committee for Medicinal Products for Human Use (CHMP). The accelerated assessment reduces the review timeframe from 210 days to 150 days. Upon request, the EMA will grant an accelerated assessment of an MAA if they decide the product is of major interest for public health, and in particular, from the viewpoint of therapeutic innovation. Marketing authorisation for leniolisib in the EEA is anticipated in H1 2023.

Anurag Relan, Chief Medical Officer of Pharming, commented: 

"This MAA submission, under an accelerated regulatory pathway, is an important step towards approval of our second product in the EEA and highlights Pharming's ongoing commitment to advancing leniolisib as a treatment for patients with APDS. There is a significant unmet need for therapies to improve outcomes for these patients, which, if left untreated, can result in permanent lung damage and lymphoma. Leniolisib has the potential to be the first approved treatment for this rare and orphan-designated disease, and we look forward to continuing our work with key stakeholders to bring this new product to patients."

The MAA is supported by positive data from a Phase II/III study of leniolisib, announced on February 2, 2022, which met its co-primary endpoints of reduction in lymph node size and correction of immunodeficiency in the target population. Furthermore, safety data from the study showed that leniolisib was well tolerated by participants. Also submitted as part of the MAA were data from a long-term, open-label extension clinical trial in patients with APDS treated with leniolisib.

About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)

APDS is a rare primary immunodeficiency that affects approximately 1 to 2 people per million. It is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells. Variants of these genes lead to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway.^1,2 Balanced signaling in the PI3Kδ pathway is essential for physiological immune function. When this pathway is hyperactive, immune cells fail to mature and function properly, leading to immunodeficiency and dysregulation.^1,3 APDS is characterized by 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, 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 The only way to definitively diagnose this condition is through genetic testing.

About Leniolisib

Leniolisib is a small-molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K with immunomodulating and potentially anti-neoplastic activities. Leniolisib inhibits the production of phosphatidylinositol-3-4-5-trisphosphate (PIP3). PIP3 serves as an important cellular messenger activating AKT (via PDK1) and regulates a multitude of cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Unlike PI3Kα and PI3Kβ, which are ubiquitously expressed, PI3Kẟ and PI3Kγ are expressed primarily in cells of hematopoietic origin. The central role of PI3Kẟ in regulating numerous cellular functions of the adaptive immune system (B-cells and, to a lesser extent, T cells) as well as the innate immune system (neutrophils, mast cells, and macrophages) strongly indicates that PI3Kẟ is a valid and potentially effective therapeutic target for several immune diseases. To date, leniolisib has been well tolerated during both the Phase 1 first-in-human trial in healthy subjects and the Phase II/III registration-enabling study.

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, biologics, and gene therapies that are in early to late-stage development. Pharming is headquartered in Leiden, 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 contains forward-looking statements, including with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, Pharming's ability to overcome the challenges posed by the COVID-19 pandemic to the conduct of its business, 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 and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming's 2021 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2021 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. 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.

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

1. Lucas CL, et al. Nat Immunol. 2014;15:88-97.
2. Elkaim E, et al. J Allergy Clin Immunol. 2016;138(1):210-218.
3. Nunes-Santos C, Uzel G, Rosenzweig SD. J Allergy Clin Immunol. 2019;143(5):1676-1687.
4. Coulter TI, et al. J Allergy Clin Immunol. 2017;139(2):597-606.
5. Maccari ME, et al. Front Immunol. 2018;9:543.
6. Jamee M, et al. Clin Rev Allergy Immunol. 2019;May 21.
7. Condliffe AM, Chandra A. Front Immunol. 2018;9:338.

For further public information, contact:

Pharming Group, Leiden, The Netherlands
Heather Robertson, Investor Relations & Corporate Communications Manager
T: +31 71 524 7400
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:
Ethan Metelenis
T: +1 (917) 882 9038
E: Ethan.Metelenis@precisionvh.com

EU PR:
Dan Caley
T: +44 (0) 787 546 8942
E: Dan.caley@aprilsix.com

Logo - https://mma.prnewswire.com/media/1778344/Pharming_Group_NV_Logo.jpg

View original content:https://www.prnewswire.com/news-releases/pharming-submits-a-marketing-authorisation-application-to-the-european-medicines-agency-for-leniolisib-301645354.html

SOURCE Pharming Group N.V.

FAQ

What is the purpose of Pharming's MAA submission for leniolisib?

The MAA submission aims to obtain marketing authorization for leniolisib as a treatment for APDS, addressing a significant medical need.

What is the significance of the EMA's accelerated assessment for leniolisib?

The EMA's accelerated assessment reduces the review period from 210 to 150 days, expediting the potential availability of leniolisib to patients.

What are the anticipated timelines for leniolisib approval?

Marketing authorization for leniolisib in the EEA is anticipated in the first half of 2023.

What were the results of the clinical studies for leniolisib?

The Phase II/III study met its co-primary endpoints, demonstrating a reduction in lymph node size and correction of immunodeficiency, with leniolisib being well tolerated.