Pharming Group (NASDAQ:PHAR) reported what management described as a strong finish to 2025, highlighting double-digit revenue growth, a return to operating profitability, and expanding commercial traction for both RUCONEST and Joenja during its fourth-quarter and full-year 2025 earnings call.
2025 financial performance and cash generation
Chief Executive Officer Fabrice Chouraqui said total revenues grew 15% in the fourth quarter of 2025 and 27% for the full year, supported by growth in RUCONEST and Joenja and “disciplined cost management.” The company delivered $26 million of operating profit in 2025 compared with an operating loss in 2024, according to Chouraqui.
Lynard said operating expenses increased to EUR 311.3 million in 2025. Excluding EUR 4.1 million of one-off restructuring costs tied to a G&A reduction program announced in October, operating expenses were EUR 307.2 million, which he said was within the company’s previous guidance range of EUR 304 million to EUR 308 million. He added that excluding EUR 29.7 million of Abliva-related expenses, operating expenses were up only 2% on a like-for-like basis.
On profitability, Lynard said adjusted operating profit (excluding non-recurring Abliva acquisition-related costs and other offsetting items) was EUR 36.4 million in 2025, compared with a loss of EUR 8.6 million in 2024. Operating cash flow improved to EUR 54.7 million versus slightly negative in 2024. Cash and marketable securities rose to EUR 181.1 million at year-end, despite EUR 68 million used for the Abliva acquisition.
Commercial portfolio: RUCONEST and Joenja
Management emphasized that 2025 included significant growth from both commercial products. Chouraqui said RUCONEST rose 26% year-over-year and 9% in the fourth quarter, while Joenja grew 29% for the year and 53% in the fourth quarter, helped by new patients in the U.S. and increased international demand, including the U.K. launch and sales through government-supported access programs.
Chief Commercial Officer LaVerne Marsh said RUCONEST remained resilient in 2025 even as the U.S. hereditary angioedema (HAE) market saw what she described as the first “new wave” of treatment options in almost five years. She said RUCONEST’s strategy continues to focus on high-attack patients who require fast and reliable on-demand treatment.
- RUCONEST: 26% global revenue growth in 2025; 20% U.S. volume growth for the year; 9% global revenue growth and 2% U.S. volume growth in Q4.
- U.S. enrollments: Marsh said Pharming added “over 60 new enrollments” in the U.S. during Q4, slightly above Q3, including new patients and new prescribers.
Marsh said Pharming began to observe competitive impacts in Q4 as patients trialed newly launched therapies, with some returning to RUCONEST. During the Q&A, both Chouraqui and Marsh discussed switches back to RUCONEST, with Marsh characterizing observed switching patterns as early but noting that within about three to six months, some patients who trialed other products may return, particularly among high-attack patients prioritizing reliability and fast relief.
Joenja’s fourth-quarter global revenue increased 53% year-over-year to $19.8 million, Marsh said, and full-year Joenja revenue was $58 million. In the U.S., Marsh said Pharming ended 2025 with 120 patients on paid therapy, up 25% from year-end 2024. She also said Pharming increased the number of identified, diagnosed APDS patients in the U.S. by 40 in 2025, more than double the increase of 18 in 2024.
Internationally, Marsh highlighted “solid first-year uptake” in the U.K. following a launch in April 2025, as well as patients receiving Joenja via government-supported access programs. She added that Pharming had identified 52 eligible U.S. pediatric patients ages four to 11 for a potential launch pending FDA approval, with roughly one-third already receiving therapy through an early access program. Across Europe, Japan, and Canada, Marsh said there were over 80 patients receiving Joenja through early access mechanisms.
Pipeline and regulatory updates: leniolisib expansion and napazimone
Chief Medical Officer Anurag Relan discussed the company’s plans to expand leniolisib (Joenja) beyond APDS into additional primary immunodeficiencies (PIDs). Relan said Pharming has two ongoing phase II proof-of-concept trials in lower-prevalence PIDs and that both studies have completed enrollment, with top-line data expected in the second half of 2026.
Relan also discussed Pharming’s work on variants of uncertain significance (VUS) related to APDS diagnosis. He said discussions with Columbia and genetic testing labs indicated labs require additional evidence to reclassify VUS, and that Pharming is planning new experiments using newer base-editing technology to generate data needed by testing labs. He said it was too soon to provide timelines or the number of patients who could ultimately be reclassified, but the company expects to provide more details “over the coming months” as work gets underway.
On regulatory milestones for APDS, Relan said the company was disappointed by an FDA complete response letter (CRL) in January regarding the pediatric label expansion for Joenja for children ages four to 11. He said Pharming believes it can address issues related to clinical pharmacology and analytical batch testing methodology and noted that a Type A meeting with the FDA is scheduled for later in March to align on the resubmission path.
Internationally, Relan said Pharming filed a marketing authorization application in Europe for leniolisib for patients 12 and older, responded to CHMP questions on manufacturing and quality controls, and expects a CHMP opinion later in March, with potential European Commission approval in the first half of the year. In Japan, he said the Pharmaceutical Affairs and Food Sanitation Council meeting recommended approval for leniolisib for APDS in patients four years and older, and Pharming expects a formal PMDA decision by the end of March.
Relan also provided an update on napazimone (formerly KL1333) for primary mitochondrial diseases. He said a registration-enabling study is underway with endpoints agreed upon with the FDA, and a blinded interim analysis showed both endpoints passed futility. The company expects to complete enrollment in 2026, with data readout in 2027 and potential approval later in 2028.
2026 guidance: revenue growth, spending, and quarterly dynamics
Management reaffirmed 2026 revenue guidance of EUR 405 million to EUR 425 million, implying 8% to 13% growth versus 2025. Lynard said the growth outlook reflects continued U.S. RUCONEST growth partly offset by declining ex-U.S. RUCONEST revenue as Pharming exits those markets, along with accelerated Joenja growth.
For RUCONEST, Lynard cautioned that quarterly revenue fluctuates due to ordering patterns and channel inventory. He said the first quarter is typically the lowest and that in Q1 2026, inventory drawdowns are expected to impact U.S. RUCONEST revenue growth by 7% to 9% year-over-year, which is factored into full-year guidance. He said RUCONEST growth is expected to be mid-single-digit at the midpoint of the range.
For Joenja, Lynard said growth is expected to accelerate in 2026, with annual growth roughly 10 percentage points higher than in 2025. He added that U.S. pediatric revenues are excluded from 2026 guidance pending clarity after the FDA Type A meeting.
Pharming guided 2026 operating expenses of EUR 330 million to EUR 335 million, including more than EUR 60 million of incremental R&D investments. Lynard said the outlook includes a EUR 9 million favorable impact from a 20% G&A headcount reduction program announced in October 2025, while marketing and sales spending is expected to remain stable.
On cost of goods sold, Lynard said Pharming does not assume any additional Joenja commercial milestone payments in 2026, and estimates cost of goods sold at 10% of revenue, corresponding to a gross margin of about 90%.
Q&A: Joenja mix, reimbursement cadence, and M&A posture
During questions, management addressed the expected mix of Joenja growth between the U.S. and international markets. Lynard said approximately 70% to 75% of Joenja’s growth in 2026 is expected to come from the U.S. Chouraqui said international growth is expected to build gradually as approvals are followed by pricing and reimbursement negotiations.
On international reimbursement cadence, Chouraqui said centralized systems can be slower upfront due to reimbursement negotiations but more efficient after reimbursement is secured. Addressing Japan specifically, he said Pharming expects to submit a price shortly after approval and that it typically takes about three months for a price to be granted, with launch timelines planned for the summer.
Asked about pipeline expansion and trial design for leniolisib in more prevalent PIDs, Relan said Pharming expects to discuss the path forward with regulators after phase II data read out, with a base case of a phase III randomized study, while also noting the possibility of alternative regulatory pathways depending on disease context and mechanism.
Regarding business development, Chouraqui said the company continues to look for acquisition and in-licensing opportunities but stressed there is “no urgency” to do a transaction to offset weakness, and that any deal would need to be complementary and value accretive.
About Pharming Group (NASDAQ:PHAR)
Pharming Group N.V. is a clinical-stage biopharmaceutical company headquartered in Leiden, the Netherlands, with a primary focus on developing and commercializing innovative protein replacement therapies for patients living with rare diseases. The company employs a proprietary transgenic technology platform designed to produce recombinant human proteins in the milk of transgenic animals, enabling scalable and cost-efficient manufacturing of complex therapeutic proteins.
The company’s lead product, RUCONEST (recombinant human C1 esterase inhibitor), is approved for the treatment of acute hereditary angioedema (HAE) attacks in multiple markets, including the United States and Europe.
