Recent news highlights regulatory and commercial developments for Pharming Group, including approval of leniolisib in Japan for younger APDS patients and ongoing preparations for quarterly earnings reporting. The company has experienced regulatory setbacks in the U.S. with FDA refusal for expanded pediatric use of Joenja®, but continues to advance patient identification and commercial growth.

• Pharming Group’s leniolisib received approval in Japan to treat APDS patients aged 4 years and older, expanding its geographic and age indication reach [N1].
• The company prepared to report Q4 earnings in early March 2026, indicating ongoing operational transparency and investor communication [N3].
• Pharming projected 27% revenue growth in 2025, driven by RUCONEST® and Joenja®, surpassing prior guidance, reflecting strong commercial execution [N8].
• FDA refused to approve expanded use of Joenja® in children with APDS, issuing a complete response letter, representing a regulatory setback [N5][N6].
• Patient identification efforts continue, with nearly 1,000 diagnosed APDS patients globally by year-end 2025, supporting market expansion potential [N2].

Pharming’s portfolio includes two approved therapies addressing rare diseases with significant unmet needs, supported by growing patient identification and expanding market access. RUCONEST® maintains a differentiated position in the acute HAE market, while Joenja® is the only approved targeted therapy for APDS, with ongoing efforts to broaden its label and geographic reach. The company’s pipeline includes promising late-stage assets such as napazimone for mitochondrial disease and leniolisib for broader immune dysregulation indications, potentially expanding addressable patient populations. Strong revenue growth and positive net income in 2025 reflect operational execution and commercial momentum. Pharming’s solid liquidity and capital structure support continued investment in innovation and market expansion. Recent regulatory approvals, including in Japan for leniolisib, demonstrate progress in global commercialization.

Pharming faces competitive pressures in the HAE market from multiple established and emerging therapies, which may impact RUCONEST® sales. Regulatory setbacks, such as the FDA’s refusal to approve expanded pediatric use of Joenja®, highlight challenges in label expansion and market penetration. The ultra-rare nature of APDS and other target diseases limits patient populations, and underdiagnosis remains a barrier to growth. Manufacturing and supply chain complexities, including reliance on contract manufacturing organizations, pose operational risks. Financially, while recent results show profitability, the company’s history includes prior losses and ongoing investment needs. Market concentration with two major U.S. customers exposes revenue risk. Additionally, evolving healthcare regulations and reimbursement environments may affect commercial performance and access.

Pharming’s competitive advantage lies in its focused rare disease strategy, combining deep scientific expertise with proven capabilities in clinical development, manufacturing, regulatory affairs, and commercialization. Its commercial portfolio includes RUCONEST®, the only recombinant C1 esterase inhibitor for acute HAE attacks, and Joenja®, the first and only approved targeted therapy for APDS, an ultra-rare primary immunodeficiency. The company’s patient-centric approach, established U.S. commercial infrastructure, and ongoing efforts to expand indications and geographic access strengthen its market position. Additionally, Pharming’s pipeline assets address significant unmet needs in rare immunological and mitochondrial diseases, supported by regulatory designations and clinical progress. The company’s manufacturing partnerships and collaborations further support scalability and supply reliability. However, competition in the HAE market is intense, and regulatory and reimbursement challenges remain key considerations.

• Regulatory Risk: Pharming’s therapies require regulatory approvals in multiple jurisdictions. Recent FDA refusal to approve expanded pediatric use of Joenja® illustrates potential challenges in obtaining label expansions and new indications.
• Competitive Risk: The HAE market is competitive with multiple therapies targeting different mechanisms. New entrants or more effective treatments could reduce Pharming’s market share for RUCONEST®.
• Market Access and Patient Identification: Underdiagnosis of rare diseases like APDS limits patient identification and treatment uptake. Delays or difficulties in expanding diagnosis and reimbursement could constrain growth.
• Manufacturing and Supply Chain: Pharming relies on contract manufacturing organizations for production of Joenja® and other products. Disruptions or delays in manufacturing capacity expansion could impact supply and commercialization.
• Financial and Operational Risks: While recent financial results show profitability, Pharming has a history of losses and requires ongoing investment in R&D and commercialization. Revenue concentration with two major customers poses risk to revenue stability.

• Pharming Group N.V. is a global biotechnology company focused on developing and commercializing therapies for rare and ultra-rare diseases, particularly immunological and genetic conditions with significant unmet needs.
• The company was incorporated in the Netherlands in 1988 and is publicly traded on Euronext Amsterdam (PHARM) and Nasdaq (PHAR).
• Pharming commercializes two approved therapies: RUCONEST®, a recombinant C1 esterase inhibitor for acute hereditary angioedema (HAE) attacks, primarily focused on the U.S. market; and Joenja® (leniolisib), an oral selective PI3Kδ inhibitor for activated PI3Kδ syndrome (APDS), approved in the U.S., U.K., Australia, Israel, and Japan for various age groups.
• RUCONEST® is positioned as a differentiated on-demand treatment for acute HAE attacks, addressing the underlying deficiency and is administered intravenously.
• Joenja® is the first and only approved targeted therapy for APDS, an ultra-rare primary immunodeficiency, with ongoing regulatory and clinical efforts to expand its use to broader patient populations and younger age groups.
• Pharming is advancing a focused pipeline including leniolisib for PIDs with immune dysregulation beyond APDS and napazimone (KL1333) for primary mitochondrial disease (mtDNA-driven PMD), with pivotal trials ongoing.
• The company reported 2025 revenues of $376.1 million, up 26.6% from 2024, driven by growth in RUCONEST® and Joenja® sales, with strong U.S. market performance and expanding international sales.
• Pharming reported a net income of $2.54 million for the year ended December 31, 2025, compared to losses in prior years, and positive operating cash flow of $54.7 million.
• As of December 31, 2025, Pharming held $145.3 million in cash and cash equivalents, with a current ratio of 2.59 and a cash ratio of 1.25, indicating solid liquidity.
• The company’s strategic focus includes expanding access to its therapies in key markets, advancing late-stage pipeline assets, and strengthening its commercial infrastructure.
• Pharming faces competition in the HAE market from multiple companies and therapies, but Joenja® currently has no known active clinical competitors in APDS.
• Recent regulatory developments include Japan approval for leniolisib to treat APDS patients aged 4 years and older, while the FDA refused expanded use approval for Joenja® in children with APDS, issuing a complete response letter.
• Patient identification efforts for APDS are ongoing, with nearly 1,000 diagnosed patients globally as of year-end 2025, supporting market expansion potential.
• Pharming’s capital expenditures are modest, primarily for property, plant, and equipment in the Netherlands, with $0.7 million spent in 2025.
• The company maintains manufacturing partnerships for Joenja® drug substance and product with contract manufacturing organizations Ardena and Skyepharma, with pediatric formulations in development.
• Pharming’s 2025 Annual Report and Form 20-F were filed on April 2, 2026, providing detailed disclosures on business, financials, and strategy.
• The company’s revenues are concentrated with two U.S. specialty pharmacy customers representing 77% of net revenues in 2025.
• Pharming’s culture emphasizes patient focus, simplicity, urgency, and disciplined execution to deliver value in rare disease markets.