-- Implemented by the Centers for Disease Control and Prevention, the diagnosis 
code will accurately identify US patients with APDS, supporting care and 
research efforts

Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: 
PHARM/Nasdaq: PHAR) announces that a new diagnosis code for reporting cases of 
activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary 
immunodeficiency, will be added to the International Classification of 
Diseases, 10th Revision, Clinical Modification (ICD-10-CM) by the US Centers 
for Disease Control and Prevention (CDC). The diagnosis code, D81.82 - 
Activated Phosphoinositide 3-kinase Delta Syndrome (APDS), will be effective 
starting October 1, 2022.

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Anurag Relan, Chief Medical Officer of Pharming, commented:

"By assigning this ICD-10-CM code, the CDC is formally recognizing APDS as a 
discrete immunological disease, and that will make a life-altering difference 
for people affected by the condition. By using the unique diagnostic code to 
identify both established and new patients with APDS, physicians will increase 
care options for affected individuals while helping to boost the world's 
understanding of the prevalence, mechanisms, and outcomes of this progressively 
debilitating disease. For healthcare practitioners, this milestone marks an 
opportunity to make a big difference by taking a simple action."

The assignment of the ICD-10-CM code will, for the first time, enable 
physicians and payors in the US to add a diagnosis of APDS to patients' health 
records, which will help connect these individuals with researchers studying 
the prevalence and course of the disease. In addition, by allocating a specific 
diagnosis, the new ICD-10-CM code may help confirm medical necessity in 
individual patients, thus improving their access to relevant care options 
through US health insurance plans.

Caused by genetic variants affecting approximately one to two people per 
million, APDS causes significant lymphoproliferation and immune dysfunction, as 
well as an increased risk of lymphoma. There is no approved therapy for the 
disease and treatment is generally limited to supportive care, such as 
antibiotics and immunoglobulin replacement therapy. Physician and patient 
advocacy groups specializing in immunodeficiency disorders, along with 
Pharming, expect the decision to raise awareness about this rare disease.

Vicki and Fred Modell, co-founders of the Jeffrey Modell Foundation, commented:

"We are excited that US regulatory authorities have assigned APDS an ICD-10-CM 
code. As a foundation dedicated to early diagnosis, meaningful treatments, and 
cures for primary immunodeficiency, we are aware of the physical and emotional 
challenges people with APDS face due to misdiagnosis of their disease. By 
increasing recognition of the condition, we expect the new diagnostic code to 
help ensure that every patient is included when it comes to the delivery of 
appropriate and meaningful treatments for APDS."

About Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)

APDS is a rare primary immunodeficiency that affects approximately one to two 
people per million. Also known as PASLI, 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 PI3K delta 
(phosphoinositide 3-kinase delta) pathway.(1,2) Balanced signaling in the PI3K 
delta 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 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.

About the Jeffrey Modell Foundation

Vicki and Fred Modell established the Jeffrey Modell Foundation (JMF) in 1987, 
in memory of their son Jeffrey, who died at the age of 15, from complications 
of Primary Immunodeficiency (PI) — a genetic condition that is chronic, 
serious, and often fatal. JMF is a global nonprofit organization dedicated to 
early diagnosis, meaningful treatments and, ultimately, cures through research, 
physician education, public awareness, advocacy, patient support, newborn 
screening, and genetic sequencing. For more information, visit 
https://www.info4pi.org/.

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 US 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.

SOURCE Pharming Group N.V.