RELEASE: Pharming Announces First Commercial Shipments of Joenja® (leniolisib) to Patients in the US(2)

LEIDEN, The Netherlands, April 11, 2023 /PRNewswire/ -- Pharming Group N.

RELEASE: Pharming Announces First Commercial Shipments of Joenja® (leniolisib) to Patients in the US(2)

LEIDEN, The Netherlands, April 11, 2023 /PRNewswire/ -- Pharming Group N.V. ("Pharming") (Euronext Amsterdam: PHARM) (Nasdaq: PHAR) announces the first commercial shipments of Joenja® (leniolisib) to patients in the United States. Joenja®, a selective oral PI3Kδ inhibitor, is the first and only US-approved treatment for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare and progressive primary immunodeficiency, in adult patients and pediatrics 12 years of age and older.

Under the terms of Pharming's 2019 exclusive license agreement with Novartis for leniolisib, the first corresponding commercial sale of Joenja® triggers a landmark $10 million payment from Pharming to Novartis.

Stephen Toor, Pharming's commercial director, commented:

"We are pleased to announce that the first shipments of Joenja® to patients, with payer reimbursement, were delivered approximately two weeks after FDA approval, marking an important milestone for APDS patients. We look forward to Joenja® widely available in the US as the first and only approved treatment for patients with APDS."

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 one of two identified genes known as PIK3CD or PIK3R1, which are vital for 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, causing immune cells to fail to mature and function properly, leading to immunodeficiency and dysregulation.1,2,3 APDS is characterized by a variety of symptoms, including severe and recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.4,5 Because these symptoms may be associated with a variety of conditions, including other primary immunodeficiencies, it has been reported that people with APDS have They are often misdiagnosed and suffer a median diagnostic delay of 7 years.6 As APDS is a progressive disease, this delay can lead to an accumulation of damage over time, including permanent lung damage and lymphoma.4-7 It can be done a definitive diagnosis through genetic testing. APDS affects approximately 1 to 2 people per million worldwide.

Acerca de Joenja® (leniolisib)

Joenja® (leniolisib) is a small molecule oral phosphoinositide 3-kinase delta (PI3Kẟ) inhibitor approved in the US 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. Joenja® inhibits the production of phosphatidylinositol-3-4-5-triphosphate, which serves as an important cellular messenger and regulates a multitude of cellular functions including proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Results of a randomized, placebo-controlled phase II/III clinical trial demonstrated clinical efficacy of Joenja® on co-primary endpoints; demonstrating a statistically significant impact on immune dysregulation and normalization of the immunophenotype in these patients, and interim open-label extension data have supported the safety and tolerability of long-term administration of Joenja®.8 Leniolisib is currently under regulatory review by part of the European Medicines Agency, with plans to seek further regulatory approvals in the UK, Canada, Australia and Japan. Leniolisib is also being evaluated in a Phase III clinical trial in children 4 to 11 years of age with APDS, with an additional trial planned in children 1 to 6 years of age with APDS. For information on Joenja®, visit:

Acerca de 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 precision medicines and protein replacement therapies, including small molecules, biologics and gene therapies that are in early or late stages of development. Pharming is headquartered in Leiden, The Netherlands, with employees around the world caring for patients in more than 30 markets in North America, Europe, the Middle East, Africa and Asia-Pacific.

For more information, visit 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 the use of terms and phrases such as "target", "ambition", "anticipate", "believe", "could", "estimate", "expect", ''goals'', '' aim'', ''can'', ''milestones'', ''objectives'', ''outlook'', ''plan'', ''probably'', ''project'', ''risks'', "program," "seek," "should," "target," "will," and similar terms and phrases. Examples of forward-looking statements may include statements regarding the 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, the statements of which 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 their cost ramifications; and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties described in Pharming's 2021 Annual Report and Annual Report on Form 20-F for the year ended December 31, 2021, filed with the Commission on In the US Securities and Exchange, 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. 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. All 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 press release. Pharming undertakes no obligation to publicly update or revise any.

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1. Lucas CL, et al. Nat Immunol. 2014;15(1):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. 8. RAO VK, et al Blood. 2023 Mar 2;141(9):971-983.

For more information, contact:

Pharming Group, Leiden, The Netherlands Michael Levitan, Vice President Investor Relations and Corporate Communications T: 1 (908) 705 1696

Heather Robertson, Director of Investor Relations and Corporate Communications E:

FTI Consulting, Londres, Reino UnidoVictoria Foster Mitchell/Alex Shaw/Amy ByrneT: 44 203 727 1000

LifeSpring Life Sciences Communication, Ámsterdam, Países BajosLeon MelensT: 31 6 53 81 64 27E:

RR PP USAEthan MetelenisE: Ethan.Metelenis@precisionvh.comT:1(917)8829038

RR PP UEClaire DobbsE: T: 44 7864 640093


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