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Pharming Group Announces European Medicines Agency (EMA) Validates its Marketing Authorization Application under Accelerated Assessment for leniolisib

Marketing authorization in the European Economic Area anticipated in H1 2023

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Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM /Nasdaq: PHAR) announces today that its Marketing Authorisation Application (MAA) for leniolisib has been validated for scientific evaluation under an accelerated assessment by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).

The application, submitted earlier in October 2022, is for the investigational drug, 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 adolescents and adults 12 years or older.

In August 2022, Pharming Group announced the leniolisib MAA was granted accelerated assessment by EMA’s CHMP. The accelerated assessment reduces the review timeframe from 210 days to 150 days. Upon request, 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 authorization for leniolisib in the European Economic Area is anticipated in H1 2023.

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 increase in percentage of naïve B cells in patients with APDS. 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.

Anurag Relan, MD, MPH, Chief Medical Officer of Pharming Group, commented:

“EMA’s validation for review of our MAA under an accelerated assessment pathway highlights Pharming’s ongoing commitment to advance leniolisib as a targeted treatment for adults and adolescents 12 years of age or older with APDS. We anticipate that leniolisib will fill an unmet need for patients with APDS, who currently rely on supportive therapies to treat their primary symptoms. This review constitutes a key milestone in Pharming’s effort to give healthcare providers and their patients global access to leniolisib. We look forward to collaborating with EMA as needed throughout the regulatory process.”

About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS): APDS is a rare primary immunodeficiency that affects approximately 1 to 2 people per million. APDS  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.

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.

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