More people started taking both drugs, and the company finally turned profitable.
From the Netherlands, a biopharmaceutical company focused on rare and often invisible diseases has posted a quarter that speaks to something more than commercial momentum — it speaks to the slow, deliberate work of reaching patients who have long had few options. Pharming Group's 30 percent revenue growth in Q3 2025, driven by treatments for hereditary angioedema and a rare immunodeficiency disorder, reflects a company crossing the threshold from promise into sustainable purpose. The FDA's priority review of a pediatric formulation for children as young as four signals that the circle of care may soon widen further, arriving at a decision point in January 2026 that could reshape the lives of some of medicine's most overlooked patients.
- A competitor's new oral HAE therapy entered the U.S. market in July, yet RUCONEST held its ground — unit sales climbed 24 percent, suggesting patient loyalty and clinical confidence are not easily displaced.
- Operating profit nearly quadrupled year-over-year to $15.8 million, and the company swung from $11 million in cash burn to $44 million generated — a signal that growth and financial discipline are finally moving in the same direction.
- The FDA's priority review for leniolisib in children aged 4 to 11 with APDS introduces a January 2026 decision date that could unlock 54 identified pediatric patients immediately, with a broader market potentially doubling if genetic reclassifications proceed.
- A 20 percent cut in non-commercial headcount and withdrawal from low-yield international RUCONEST markets reflect a deliberate narrowing of focus — trading breadth for depth in the pursuit of long-term profitability.
- Full-year revenue guidance was raised to $365–375 million, new commercial leadership is being installed, and the company's index promotion from small-cap to mid-cap marks a quiet but meaningful shift in how the market now sees its scale.
Pharming Group, the Dutch biopharmaceutical company listed on both Euronext Amsterdam and Nasdaq, reported third-quarter revenues of $97.3 million — a 30 percent increase over the same period last year. The growth was carried by its two commercial products: RUCONEST, an intravenous treatment for hereditary angioedema attacks, contributed $82.2 million, while Joenja, an oral therapy for the rare immunodeficiency disorder APDS, added $15.1 million. Both grew by roughly 30 percent year-over-year.
What made the quarter notable was not just the revenue line but what sat beneath it. In the U.S., RUCONEST continued gaining patients and prescribers even after a rival oral therapy entered the market in July. For Joenja, 116 patients were on paid therapy by September's end, up from 93 a year earlier, with the recently launched U.K. market adding to international momentum. Operating profit reached $15.8 million — nearly four times the $4.1 million recorded a year ago — and the company generated $44 million in operating cash year-to-date, reversing an $11 million burn from the prior year's first nine months.
The regulatory pipeline offered its own signal. The FDA accepted and granted priority review to a supplemental application for leniolisib in children aged 4 to 11 with APDS, with a decision expected by January 31, 2026. The company has identified 54 U.S. children in that age group who would immediately become eligible for treatment. More broadly, researchers estimate that a significant share of over 1,400 patients carrying uncertain genetic variants could eventually be reclassified as having APDS — a development that could substantially expand the addressable market.
CEO Fabrice Chouraqui raised full-year revenue guidance to $365–375 million, up from a prior range of $335–350 million. Alongside the stronger outlook, the company announced a 20 percent reduction in non-commercial headcount — primarily at its Netherlands headquarters — targeting roughly $10 million in annual savings. RUCONEST will also be withdrawn from non-U.S. international markets, where it generated only $1.1 million in the quarter, freeing resources for higher-potential opportunities. New commercial leadership is being brought in, and Pharming's recent promotion from a small-cap to a mid-cap index on Euronext quietly confirmed what the numbers were already saying: this is a company that has grown into a different category of itself.
Pharming Group, the Dutch biopharmaceutical company trading on Euronext Amsterdam and Nasdaq, reported third-quarter revenues of $97.3 million on Thursday, a jump of 30 percent from the same period last year. The company's two main commercial products drove the growth: RUCONEST, an intravenous treatment for acute hereditary angioedema attacks, brought in $82.2 million in the quarter, up 29 percent year-over-year, while Joenja, an oral therapy for a rare immunodeficiency disorder called APDS, generated $15.1 million, up 35 percent.
The financial performance reflected something deeper than raw sales numbers. In the U.S. market for RUCONEST, the company continued adding both patients and prescribers even after a competitor launched a new oral on-demand HAE therapy in July. The company attributed this sustained growth to the drug's proven efficacy and rapid intravenous delivery. Unit sales volume in the U.S. climbed 24 percent in the quarter. For Joenja, the momentum came from a 25 percent year-over-year increase in patients actually taking the drug—116 patients on paid therapy by the end of September, up from 93 a year earlier. The U.K. market, which launched Joenja in April, contributed meaningfully to the international revenue growth.
Operating profit nearly quadrupled, reaching $15.8 million in the third quarter compared to $4.1 million in the prior year—a 285 percent increase. The company generated $32 million in cash from operations during the quarter and $44 million year-to-date, a stark reversal from the $11.1 million cash burn in the first nine months of 2024. These numbers mattered because they signaled the company was moving toward sustainable profitability in its core business rather than simply growing revenue while bleeding cash.
The regulatory calendar offered another catalyst. In October, the FDA accepted and granted priority review to a supplemental application for leniolisib in children aged 4 to 11 years with APDS, with a decision expected by January 31, 2026. This pediatric expansion could unlock a significant new patient population. The company has identified 54 U.S. children in that age group with diagnosed APDS who would become eligible for treatment upon approval. Beyond that immediate opportunity, the company noted that over 1,400 U.S. patients carry genetic variants of uncertain significance in the genes implicated in APDS, and researchers estimate that 20 percent of those patients could ultimately be reclassified as having APDS, potentially doubling the addressable market.
CEO Fabrice Chouraqui raised the company's full-year revenue guidance to $365 million to $375 million, up from the prior range of $335 million to $350 million. He attributed the confidence to sustained momentum in both commercial products and the pipeline. The company also announced a strategic decision to withdraw RUCONEST from non-U.S. markets, which contributed only $1.1 million, or 1.3 percent of quarterly RUCONEST revenue. That move freed up resources to focus on higher-potential opportunities.
In a move to optimize the balance sheet, Pharming announced a 20 percent reduction in non-commercial and non-medical headcount, primarily at its Netherlands headquarters, targeting a 15 percent reduction in general and administrative expenses—roughly $10 million annually. The company expects one-time restructuring costs of approximately $7 million in the fourth quarter. Separately, the company appointed Kenneth Lynard as Chief Financial Officer effective October 1, and announced that Leverne Marsh would become Chief Commercial Officer on January 1, 2026, succeeding Stephen Toor, who will remain as an advisor. Marsh comes from Dexcom, where she led marketing, and previously held senior commercial roles at Novartis.
The company also noted that Pharming was promoted from the Euronext AScX small-cap index to the AMX mid-cap index effective September 22, reflecting its growing market capitalization and trading activity. Looking ahead, the company said it does not expect material impact from recently announced U.S. tariffs, though some uncertainties remain around potential exclusions. The pipeline continues advancing: two Phase II trials in primary immunodeficiencies are underway to expand leniolisib's potential, and a pivotal study for KL1333 in primary mitochondrial disease is ongoing.
Notable Quotes
We delivered another strong quarter, with significant growth in revenue and profitability, reinforcing our confidence in the business.— CEO Fabrice Chouraqui
Leverne brings a strong track record of high impact launches and deep experience across the commercial spectrum, which will be instrumental as we continue executing our strategy to become a leading global rare disease company.— CEO Fabrice Chouraqui, on new Chief Commercial Officer Leverne Marsh
The Hearth Conversation Another angle on the story
What made the difference between this quarter and last year? The numbers jumped significantly.
Two things converged. First, the patient base actually grew—more people started taking both drugs, and more doctors prescribed them. Second, the company finally turned profitable at the operating level. They're not just selling more; they're keeping more of what they earn.
But didn't a competitor launch a new HAE drug in July? How did RUCONEST keep growing?
That's the interesting part. The new competitor's drug is oral, which sounds convenient, but RUCONEST is intravenous and works faster. Some patients prefer speed and reliability over convenience. The company kept adding patients even in a crowded market.
What about the pediatric approval they're waiting for?
That's the next inflection point. They've identified 54 children in the U.S. who could be treated if the FDA approves the pediatric formulation by January. But the bigger opportunity is the genetic reclassification—over 1,400 patients carry uncertain variants that might actually be APDS. If 20 percent of those get reclassified, the patient population doubles.
They're cutting headcount while raising guidance. Isn't that a contradiction?
Not really. They're cutting administrative overhead—the stuff that doesn't directly make drugs or sell them. They're redeploying that capital to commercial execution and pipeline development. It's disciplined capital allocation.
Who's the new commercial officer, and why does it matter?
Leverne Marsh, coming from Dexcom. She's spent her career launching specialty products and managing commercial strategy. At a rare disease company trying to expand globally, that experience in scaling fast-growing markets is exactly what they need.
What's the real risk here?
Execution. They've raised guidance, they've made leadership changes, they're cutting costs. All of that only works if the pipeline delivers and the market keeps absorbing the drugs at the pace they're projecting. One clinical setback or a slower-than-expected uptake in the pediatric population could reset expectations quickly.