Pharming N : Group reports financial results for the first half of 2022

Pharming Group reports financial results for the first half of 2022

• Total revenues increased 4% in H1 2022 to US$96.8 million, supported by increases in physicians prescribing and number of patients
• Strong balance sheet and stable cash flow from RUCONEST® providing for investments in Pharming's future growth
• Continued investment in launch preparations for leniolisib; post period milestones include US FDA filing and grant of accelerated assessment in EMA
• Reduction in minority stake of BioConnection leads to recognized gain of US$ 12.8 million

Leiden, The Netherlands, August 4, 2022: Pharming Group N.V. ("Pharming" or "the Company")

(Euronext Amsterdam: PHARM/Nasdaq: PHAR) presents its preliminary (unaudited) financial report for the first six months of 2022 ended June 30, 2022.

Chief Executive Officer, Sijmen de Vries, commented:

"Pharming delivered strong half year results with total revenues up 4% compared to the same period last year. RUCONEST® sales growth was supported by increases in physicians prescribing and number of patients. For the second quarter of 2022, sales were US$50.1 million bringing the first half year of 2022 to US$96.8 million.

Concerning pipeline development, we continued to advance leniolisib through the regulatory process as we seek marketing authorization in the US, the UK, and the EEA for the treatment of APDS, a rare, complex and progressive primary immunodeficiency. In the US, we filed a New Drug Application with the FDA for the treatment of APDS in adults and adolescents aged 12 or older at the end of July 2022. On August 2, we announced a new diagnosis code for reporting cases of APDS, which 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, will be effective starting October 1, 2022.

Looking to Europe, the EEA and the UK remain on track for regulatory filings from October this year. On August 1, we announced the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) granted an accelerated assessment for the Marketing Authorisation Application (MAA) for leniolisib in adults and adolescents aged 12 and older.

In our drive to focus on RUCONEST® sales, the launch and commercialization of leniolisib, and the management of our pipeline, we initiated an internal review of our pipeline. As a result of these initial findings, we have taken the strategic decision to discontinue the development of rhC1INH for acute kidney injury and pre-eclampsia. In line with this decision, we will also de-prioritize further

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development in the large-scale production of rhC1INH through the use of our transgenic cattle herd. The herd will be maintained while we consider strategic options for acute kidney injury (AKI) to gain value from the work done to date. We will continue to progress the ongoing Phase IIb clinical trial in AKI until further notice.

With these decisions, we continue to evolve as a company, and continue to focus our efforts and resources on the most value enhancing initiatives for our stakeholders."

Strategic highlights

During the first half of 2022, we continued to execute on our strategic objectives of building a sustainable business by focusing on RUCONEST® sales, the approval, launch and commercialization of leniolisib, and the ongoing development and management of our pipeline.

The development of our pipeline will be through a combination of internal development projects - including the development of additional indications for leniolisib and OTL-105 as a gene therapy for HAE - and the potential acquisition of new, late-stage assets through in-licensing and M&A opportunities.

These potential acquisitions will be financed through a combination of positive cash flow from the RUCONEST® business, anticipated future leniolisib businesses, as well as available cash from our strong balance sheet. If required, Pharming will access additional funding from the capital markets.

Therefore, with the ongoing focus on our strategic objectives we have initiated an internal review of our pipeline. As a result, we are announcing a number of initial changes to the pipeline as detailed below.

Pipeline development

leniolisib

For leniolisib, a three-step approach has been planned for the coming years.

The first step is the anticipated commercial approval and market launch in the US during the first half of 2023, followed by key markets in the European Economic Area (EEA) and the UK in the second half of 2023. The Company will evaluate additional countries and regions and will commercialize the product either directly, or through strategic distribution partnerships.

The second step includes the approval and launch of leniolisib as a treatment of APDS in children as young as one year of age.

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The third step is the continued life cycle management of the leniolisib compound into further indications.

US market

Subject to FDA approval and granting of priority review by the FDA in the US, we remain on track for the commercial approval of leniolisib in the first quarter and market launch early in the second quarter of 2023.

On July 29, 2022, a New Drug Application (NDA) was submitted to the US FDA for leniolisib, for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adults and adolescents aged 12 or older. Aligned with the goals set out in PDUFA VI, Pharming expects a Filing Notification Letter by Day 60, which will include the FDA's determination of priority review. If priority review is granted, it is expected that the FDA review will be completed within six months of the 60-day filing date.

In anticipation of a positive outcome from the FDA, we continue to grow our US field force and leverage our marketing capabilities for the commercialization of leniolisib.

Finally for the US market, on August 2, 2022, Pharming announced that a new diagnosis code for reporting cases of APDS, 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, will be effective starting October 1, 2022, and will help accurately identify, stratify and triage US patients eligible for APDS treatments and research opportunities.

EU and UK markets

In Europe, we remain on track with anticipated filings of the market authorization applications to both regulatory agencies - the European Medical Agency (EMA) and the UK Medicines and Healthcare Products Regulatory Agency (MHRA) - in the second half of 2022. Additionally, the MHRA granted Promising Innovative Medicine (PIM) designation to leniolisib for the treatment of APDS.

Additionally, on August 1, we announced the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) granted an accelerated assessment for the Marketing Authorisation Application (MAA) for leniolisib in adults and adolescents aged 12 and older. Pharming plans to submit the MAA for leniolisib to EMA in October 2022.

Pediatric clinical development

The studies to support the initial applications in the US, EU, and UK have enrolled patients ages 12 years and older. To expand access to leniolisib for the treatment of pediatric APDS, Pharming has developed a clinical plan to include children as young as one year of age. During the first half of the year, positive

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decisions were received from EMA and MHRA on the Pediatric Investigation Plan (PIP) for leniolisib as a treatment for APDS in children.

The leniolisib PIP includes two planned, global clinical trials in pediatric patients with APDS aged 4 to 11 with a second study in patients aged 1 to 6. These two studies will support regulatory filings worldwide. Pharming expects to initiate recruitment for this pediatric program for leniolisib in the second half of 2022.

OTL-105

In July 2021, Pharming announced a strategic collaboration with Orchard Therapeutics to research, develop, manufacture and commercialize OTL-105, a newly disclosed investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy for the treatment of hereditary angioedema (HAE). The program has made significant progress developing the lentiviral vector to enhance C1-inhibitor expression and is now testing in preclinical HAE disease models. We anticipate providing further updates as we move towards preparing an Investigational New Drug (IND) filing.

Acute Kidney Injury (AKI)

Following an internal review of our pipeline, we have taken the strategic decision to discontinue further development of this therapy.

While we consider strategic options to gain value from the work done to date, we will de-prioritize further development and investment in the large-scale production of rhC1INH through the use of our transgenic cattle herd. The herd will continue to be maintained to allow all possible outcomes to be explored.

The ongoing Phase IIb clinical trial will continue as we evaluate these strategic options.

Pre-eclampsia

Following an internal review of our pipeline, a decision has been made to discontinue further development and investment. This was due to the choices we have made to prioritize the investments to support the sales of RUCONEST® and the commercialization of leniolisib.

Pompe

The study into the development of a next-generationalpha-glucosidase therapy for the treatment of Pompe disease is ongoing. We are currently engaged in preclinical studies. If and when results from these preclinical studies become available, we will update the market.

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Operational highlights

RUCONEST®

RUCONEST® sales were up 4% in the first half of 2022 providing stable cash flow for Pharming's future growth. Total revenues were US$96.8 million compared to US$93.2 million in the first half of 2021. RUCONEST® sales growth was supported by increases in physicians prescribing and number of patients.

The US market represented 97% of total revenues for the first half of the year, while Rest of World (RoW) and Europe represented 3%.

With a continued need for safe and reliable acute treatment options for hereditary angioedema (HAE), we remain confident in the ongoing demand for RUCONEST® and continued single digit growth for the 2022 financial year.

Organizational update

Robert Friesen, Chief Scientific Officer and Executive Board member resigned from his role at Pharming Group. Mr Friesen's succession is currently under review.

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