PR97297

LEIDEN, Netherlands, Aug. 2, 2022 /PRNewswire=KYODO JBN/ --

-- 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.