- Full year 2025 total revenues increased by 27% to US$376.1 million, driven by continued growth of RUCONEST® and rising demand for Joenja® (leniolisib)
- Fourth quarter 2025 total revenues increased by 15% to US$106.5 million, compared to the fourth quarter 2024
- RUCONEST® full year revenue increased by 26% to US$317.9 million and fourth quarter revenue increased by 9% to US$86.7 million
- Joenja® revenue increased by 29% to US$58.2 million and fourth quarter revenue increased by 53% to US$19.8 million
- Achieved US$25.8 million operating profit in 2025, compared to a loss in 2024
- Achieved US$54.7 million net cash flow from operations in 2025, compared to negative cash flow in the prior year
- 2026 total revenue guidance of US$405.0 – US$425.0 million (8% to 13% growth), driven by significant and accelerating growth for Joenja® and continued growth for RUCONEST®
- Advancing the clinical pipeline with key 2026 milestones including Phase II leniolisib readouts in PIDs with immune dysregulation and pivotal FALCON study enrollment completion for napazimone (KL1333) in primary mitochondrial disease
- Pharming to host a conference call today at 13:30 CET (8:30 am EDT)
Leiden, the Netherlands, March 12, 2026: Pharming Group N.V. (“Pharming” or “the Company”) (NASDAQ:PHAR) presents its preliminary (unaudited) financial report for the three months and full year ended December 31, 2025.
Chief Executive Officer, Fabrice Chouraqui commented:
“2025 was a defining year for Pharming and reflects the focus and discipline our teams have brought to executing our strategy. We outperformed revenue guidance and delivered strong financial performance, with total revenues up 27%, driven by continued RUCONEST® growth and rising demand for Joenja® (leniolisib).
With its efficacy, reliability and rapid onset of action, RUCONEST® remains an established on-demand treatment option for difficult to treat patients. Joenja® performance accelerated in 2025, with growth driven by a 25% increase in patients on paid therapy in the U.S. We have identified approximately 1,000 APDS patients globally, reinforcing the expanding opportunity for Joenja® as diagnosis and patient identification continue to improve.
We also demonstrated disciplined cost management, delivering US$25.8 million in operating profit and US$54.7 million in net cash flow from operations in 2025. This marks an important inflection point and strengthens our ability to fund growth and long-term investment.
Building on this momentum, we plan to further enhance capital allocation to drive growth. We expect 2026 total revenues in the range of US$405 million to US$425 million, representing 8% to 13% growth, driven by both Joenja® and RUCONEST®.
Our pipeline is a significant value driver, and we highlighted its depth and upcoming catalysts at our recent Investor Day.
Entering 2026, our priorities are clear: reinforce RUCONEST® as a cornerstone on-demand therapy for difficult to treat HAE patients, accelerate Joenja®’s expansion, and advance our pipeline to broaden our impact in rare disease. We continue to expect Phase II read-outs for leniolisib in broader primary immunodeficiencies with immune dysregulation in the second half of 2026 and are on track to complete enrollment in the pivotal FALCON study of napazimone (KL1333) in 2026, with data readout anticipated in late 2027.
This momentum reinforces the strength of our business and positions us to advance our high-value pipeline, delivering long-term value for shareholders and meaningful impact for rare disease patients.”
Fourth quarter and full year 2025 highlights
Commercialized products
RUCONEST® marketed for the treatment of acute HAE attacks
RUCONEST® growth continued in the fourth quarter of 2025, with revenue of US$86.7 million, a 9% increase compared to the fourth quarter of 2024. Revenue for the full year 2025 reached a record US$317.9 million, representing 26% year-over-year growth.
In the U.S. market, we continued to expand our patient and prescriber base during the quarter and throughout the year. Revenue growth over the prior year reflects the benefit of a larger patient base, including patients with HAE with normal C1-INH. With its efficacy, reliability and rapid onset of action via IV administration, RUCONEST® remains an established on-demand treatment option for patients experiencing more severe or frequent attacks who have failed other on-demand medications. During the year, we increased the RUCONEST® physician prescriber base by 6%. Unit sales volume in the U.S. increased by 2% in the fourth quarter and 20% for the full year.
Joenja® (leniolisib) marketed for the treatment of APDS
Joenja® revenue increased to US$19.8 million in the fourth quarter of 2025, a 53% increase compared to the fourth quarter of 2024. Revenue for the full year 2025 increased to US$58.2 million, a 29% increase compared to 2024. Revenue growth in the fourth quarter of 2025 was driven by a significant increase in patients on paid therapy in the U.S. and increased demand in international markets, including strong patient uptake in the U.K. following the April 2025 launch and purchases under government-supported access programs.
The U.S. market contributed 86% of 2025 revenues, while the EU and Rest of World contributed 14%.
As of December 31, 2025, 120 patients were on paid therapy in the U.S., representing a 25% increase from the 96 patients at the end of 2024. The number of patients on paid therapy in the U.S. increased by 24 during 2025, compared to an increase of 16 in 2024. The number of U.S. patients diagnosed with APDS that we have identified increased by 40 in 2025 compared to an increase of 18 in 2024.
APDS patient finding
As of December 31, 2025, we have identified 998 diagnosed APDS patients of all ages globally, including 274 patients in the U.S. and 382 in core markets outside of the U.S. Of the identified patients in the U.S., 181 patients are 12 years of age or older and currently eligible for treatment with Joenja®, while 52 are between 4 and 11 years of age.
VUS patient reclassification
We previously announced that genetic testing laboratories were evaluating data from a study published in June 2025 in the journal Cell by researchers at Columbia University to determine the process and potential for reclassifying variants of uncertain significance, or VUS, enabling providers to genetically confirm APDS diagnoses. Based on this evaluation, additional complementary evidence will be required for variant interpretation by genetic testing laboratories. We are planning new experiments to generate the data needed for genetic testing laboratories to evaluate VUSs identified in patients who have undergone genetic testing for APDS or other immunodeficiencies.
There are currently over 1,800 known U.S. patients with a VUS in the PIK3CD and PIK3R1 genes implicated in APDS. We expect to provide an estimate of how many of these patients may be diagnosed with APDS following completion of these experiments.
Joenja® (leniolisib) development
Leniolisib for APDS
As of December 31, 2025, there are 175 APDS patients in either a leniolisib Expanded Access Program (compassionate use), an ongoing clinical study, or a paid access program.
Pediatric label expansion
On January 30, 2026, we received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding our supplemental New Drug Application (sNDA) for Joenja® (leniolisib) for the treatment of APDS in children aged 4 to 11 years. The FDA requested additional pediatric pharmacokinetic (PK) data and clarification related to an analytical batch testing method.
We believe we can address the clinical pharmacology and batch testing methodology issues outlined in the letter, and have requested a Type A meeting with the FDA to discuss the Agency’s feedback and align on the path forward for resubmission. The meeting is expected to occur in March 2026.
European Economic Area (EEA)
We completed the required manufacturing activities and quality controls requested by the European Medicine Agency’s (EMA) Committee for Human Medicinal Products (CHMP) and submitted our comprehensive response, including supporting data, by the January 2026 deadline. We now expect a CHMP opinion on the Marketing Authorisation Application (MAA) for leniolisib in adult and pediatric patients aged 12 years and older with potential EC approval in the first half of 2026.
Japan
A decision by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) is expected by the end of March 2026 on our new drug application (NDA) for leniolisib for the treatment of APDS in adult and pediatric patients 4 years of age and older.
Additional markets
We submitted a response with additional CMC data to Health Canada at the end of January 2026. A decision on the regulatory submission in Canada for APDS patients 12 years of age and older is expected by mid-year.
Leniolisib for additional primary immunodeficiencies (PIDs)
Two Phase II clinical trials are evaluating leniolisib for additional primary immunodeficiencies (PIDs) with immune dysregulation, which represent substantially larger patient populations than APDS. These include (i) genetically identifiable PIDs with immune dysregulation linked to altered PI3Kδ signaling and (ii) common variable immunodeficiency, or CVID, with immune dysregulation identified independently of genetics. We outlined the scientific and clinical rationale supporting the expansion of leniolisib into these patient populations at an Investor Day held on February 3, 2026.
Patient enrollment in both clinical trials is now complete and we anticipate trial read-outs in the second half of 2026.
Napazimone (KL1333) for mitochondrial DNA-driven primary mitochondrial disease
Napazimone (KL1333) is being evaluated in the pivotal FALCON clinical trial in adult patients with mtDNA-driven mitochondrial disease, and has the potential to become the first standard of care in this setting. Pharming management outlined the scientific and clinical rationale for the development of napazimone (KL1333) at the Investor Day held on February 3, 2026.
Over 20 clinical sites in the trial are actively recruiting, and the number of sites is expected to expand during the first half of 2026. The program remains on track to complete enrollment this year, with trial readout anticipated in late 2027 and potential FDA approval by the end of 2028, if successful.
Organizational updates
On September 2, 2025, we announced that Mr. Kenneth Lynard was appointed as Chief Financial Officer, effective October 1, 2025.
On November 6, 2025, we announced that Mrs. Leverne Marsh had been appointed as Chief Commercial Officer effective January 1, 2026, succeeding Mr. Stephen Toor.
Financial Summary
| Consolidated Statement of Income | Q4 2025 | Q4 2024 | 2025 | 2024 |
| Amounts in US$m except per share data | ||||
| Total Revenues | 106.5 | 92.7 | 376.1 | 297.2 |
| Cost of sales | (21.1) | (12.2) | (45.5) | (35.4) |
| Gross profit | 85.4 | 80.5 | 330.6 | 261.8 |
| Other income | 4.2 | 0.1 | 6.5 | 2.2 |
| Research and development | (32.1) | (22.3) | (100.4) | (83.1) |
| General and administrative | (20.0) | (24.7) | (80.0) | (70.7) |
| Marketing and sales | (31.3) | (26.9) | (131.0) | (118.8) |
| Other Operating Costs | (83.4) | (73.9) | (311.3) | (272.6) |
| Operating profit (loss) | 6.2 | 6.7 | 25.8 | (8.6) |
| Finance result (net) and share of result in associates | (1.4) | — | (13.0) | 0.1 |
| Profit (loss) before tax | 4.8 | 6.7 | 12.8 | (8.5) |
| Income tax credit (expense) | 0.5 | (3.8) | (10.3) | (3.3) |
| Profit (loss) for the period | 5.3 | 2.9 | 2.5 | (11.8) |
| Earnings per share | ||||
| Basic, attributable to equity holders of the parent (US$) | 0.008 | 0.004 | 0.004 | (0.018) |
| Diluted, attributable to equity holders of the parent (US$) | 0.007 | 0.004 | 0.004 | (0.018) |
| Segment information – Revenues | Q4 2025 | Q4 2024 | 2025 | 2024 |
| Amounts in US$m | ||||
| Revenue – RUCONEST® (US) | 84.3 | 78.2 | 311.7 | 246.6 |
| Revenue – RUCONEST® (EU and RoW) | 2.4 | 1.4 | 6.2 | 5.6 |
| Total Revenues – RUCONEST® | 86.7 | 79.7 | 317.9 | 252.2 |
| Revenue – Joenja® (US) | 15.4 | 11.8 | 50.1 | 40.5 |
| Revenue – Joenja® (EU and RoW) | 4.4 | 1.3 | 8.1 | 4.5 |
| Total Revenues – Joenja® | 19.8 | 13.0 | 58.2 | 45.0 |
| Total Revenues – US | 99.8 | 90.0 | 361.7 | 287.1 |
| Total Revenues – EU and RoW | 6.8 | 2.7 | 14.4 | 10.1 |
| Total Revenues | 106.5 | 92.7 | 376.1 | 297.2 |
| Consolidated Balance Sheet | December 31, 2025 | December 31, 2024 |
| Amounts in US$m | ||
| Cash and cash equivalents, restricted cash and marketable securities | 181.1 | 169.4 |
| Current assets | 299.5 | 278.4 |
| Total assets | 500.0 | 400.0 |
| Current liabilities | 115.8 | 73.8 |
| Equity | 277.1 | 221.1 |
Underlying figures are unrounded. Therefore, totals may differ slightly from the sum of individual items due to rounding effects in the presentation of this press release.
Financial highlights
Fourth quarter 2025
For the fourth quarter of 2025, total revenues increased by US$13.8 million, or 15%, to US$106.5 million, compared to US$92.7 million in the fourth quarter of 2024. RUCONEST® revenues amounted to US$86.7 million, a 9% increase compared to the fourth quarter of 2024. This increase in RUCONEST® revenues was primarily driven by volume growth and a price increase in the U.S. Joenja® revenues amounted to US$19.8 million in the fourth quarter of 2025, a 53% increase compared to the fourth quarter of 2024. This increase in Joenja® revenues was primarily driven by an increase in volume.
Gross profit increased by US$4.9 million or 6% to US$85.4 million (4Q 2024: US$80.5 million), mainly due to the increase in revenues. This was partially offset by the first sales milestone payment for Joenja® of US$5.0 million included in cost of sales.
Other income increased from US$0.1 million in the fourth quarter of 2024 to US$4.2 million in the fourth quarter of 2025, primarily driven by a US$3.9 million gain on the early termination of the DSP facility lease at Pivot Park in Oss, the Netherlands.
The operating profit decreased 8% and amounted to US$6.2 million compared to US$6.7 million in the fourth quarter of 2024. Adjusted to exclude US$0.1 million of non-recurring Abliva acquisition-related expenses, US$4.1 million in one-off restructuring expenses, and the US$3.9 million gain on the early termination of the DSP facility lease, the operating profit amounted to US$6.5 million. The improved operating result was primarily driven by an increase in revenues, partially offset by higher operating expenses, which include a total of US$9.3 million in Abliva-related expenses, and the first sales milestone for Joenja® of US$5.0 million.
The finance result (net) and share of result in associates amounted to a loss of US$1.4 million compared to US$— million in the fourth quarter of 2024. This was primarily driven by unfavorable EUR/USD exchange rate developments, resulting in a foreign currency loss of US$2.2 million compared to a gain of US$2.6 million in the fourth quarter of 2024, partially offset by higher results from fair value measurements of US$2.5 million and higher share of results in associates of US$1.4 million.
In the fourth quarter of 2025, a net profit of US$5.3 million was realized, compared to a net profit of US$2.9 million in the fourth quarter of 2024. In addition to the aforementioned drivers, the net result was positively impacted by a tax credit of US$0.5 million compared to a tax expense of US$3.8 million in the fourth quarter of 2024.
Cash generated from operations amounted to US$10.7 million, compared to US$9.3 million in the fourth quarter of 2024. Cash and cash equivalents, including restricted cash and marketable securities, increased from US$168.9 million at the end of third quarter of 2025 to US$181.1 million at the end of the fourth quarter of 2025. This increase was primarily driven by the net cash flows generated from …