Pharming Eyes $425M Revenue, Bets Big on Rare Disease Pipeline

LEIDEN, The Netherlands – February 03, 2026 – Pharming Group N.V. today outlined an ambitious strategy that balances robust commercial growth with heavy investment in its late-stage rare disease pipeline, projecting total revenues to reach between US$405 million and US$425 million in 2026. During its virtual Investor Day, the biopharmaceutical company detailed plans to leverage its strengthening commercial portfolio to fund the development of two potentially transformative therapies targeting large, underserved patient populations.

The financial guidance, representing an 8% to 13% growth trajectory, comes on the heels of a strong 2025, where preliminary revenues of approximately US$376 million exceeded the company's own upwardly revised forecasts. This performance history lends credibility to a strategy that will see operating expenses rise to between US$330 million and US$335 million in 2026, driven primarily by escalating Research & Development costs for its two key pipeline assets: leniolisib and the newly named napazimone.

Fueling Innovation with Commercial Success

Pharming's strategy hinges on a dual-engine approach: using cash flow from its approved products, RUCONEST® and Joenja® (leniolisib), to power its journey toward becoming a leading global rare disease company. The company’s commercial arm has demonstrated significant momentum. RUCONEST®, a treatment for hereditary angioedema, is expected to deliver steady mid-single-digit growth. However, the star performer is Joenja®, the first and only approved treatment for the rare immunodeficiency Activated PI3K-delta Syndrome (APDS).

Since its U.S. launch in April 2023, Joenja has seen accelerating uptake, a trend Pharming expects to continue. The company's 2026 revenue forecast anticipates significant growth for the drug, with its annual growth rate projected to be about 10 percentage points higher than in 2025. This success provides a solid financial foundation, allowing Pharming to invest confidently in the high-risk, high-reward world of late-stage clinical development.

“Pharming’s vision of developing into a leading global rare disease company is grounded in the strength of our two commercial products and our high-value pipeline,” commented Fabrice Chouraqui, Chief Executive Officer of Pharming. “We expect our commercial portfolio to continue delivering robust growth... We believe both [pipeline] programs offer substantial long‑term value‑creating potential for Pharming.”

Expanding the Potential of Leniolisib

A cornerstone of Pharming’s long-term vision is the expansion of leniolisib beyond its initial APDS indication. The company is exploring the drug’s potential in significantly larger patient populations, specifically primary immunodeficiencies (PIDs) with immune dysregulation and common variable immunodeficiency (CVID) with similar symptoms. Two Phase II proof-of-concept trials are currently underway, with top-line data eagerly awaited in the second half of 2026.

A positive outcome in these trials could be a game-changer. CVID is the most common symptomatic primary immunodeficiency, affecting an estimated 1 in 25,000 to 1 in 50,000 people. While APDS served as a crucial clinical proof-of-concept for the drug’s mechanism, success in the broader CVID population would unlock a market several orders of magnitude larger and address a significant unmet need for patients who suffer from immune system dysfunction beyond just antibody deficiency.

Presenting at the Investor Day, Dr. Jocelyn Farmer of Lahey Hospital & Medical Center, an authority on CVID, provided context on the complex disease biology and the need for targeted therapies that can address the underlying immune dysregulation, a role for which leniolisib may be well-suited.

The path is not without its hurdles. The company recently received a Complete Response Letter from the U.S. FDA for a pediatric version of Joenja for children aged 4 to 11. In a move demonstrating financial prudence, Pharming has conservatively removed potential revenue from this indication from its 2026 guidance until there is more clarity on a revised approval timeline.

Napazimone: A New Hope for Mitochondrial Disease

Pharming also formally introduced napazimone (formerly KL1333) as the compound name for its investigational therapy for mtDNA-driven mitochondrial disease. This program represents a major push into a new therapeutic area characterized by a profound lack of effective treatments. Patients with these rare and debilitating conditions suffer from impaired energy production, leading to severe fatigue, muscle weakness, and reduced life expectancy.

Currently, the standard of care is limited to supportive measures, leaving patients and clinicians with few options. Napazimone has the potential to become the first-ever approved standard of care for this condition. The pivotal FALCON clinical trial is ongoing and remains on track for a data readout in 2027. A successful trial would position Pharming as a pioneer in mitochondrial medicine, a field of immense unmet need.

Dr. Amel Karaa of Massachusetts General Hospital, a leading expert in mitochondrial medicine, highlighted the debilitating nature of these diseases and the urgent need for therapies that can alter their course. The development of napazimone is a direct response to this need, targeting the fundamental energy production deficits that define the condition.

With two commercial products generating substantial revenue and a well-funded, late-stage pipeline featuring assets with blockbuster potential, Pharming is positioning itself as a formidable player in the rare disease space. The company's strategy of reinvesting its commercial success into ambitious R&D reflects a confident, long-term vision aimed at delivering significant value to both patients and investors in the years to come.