Pharming Group N.V. (PHAR) Business Model Canvas

Pharming Group N.V. (PHAR): Business Model Canvas [Dec-2025 Updated]

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You're digging into Pharming Group N.V.'s mechanics, wanting to see the engine behind their rare disease strategy, especially now that they've added Joenja to the established Ruconest. Honestly, this is a classic, high-touch orphan drug model balancing two commercial assets against significant R&D needs for pipeline drugs like KL1333. With 2025 revenue guidance firmly set between US$365 million and US$375 million, fueled by Ruconest and Joenja-which brought in US$15.1 million in Q3 2025-the revenue streams are clear, but the operational complexity is high. Below, we break down exactly how they structure their key activities, manage their specialized sales force, and control the cost of goods sold for these complex biologics, so you can see the full picture of this specialized biopharma play.

Pharming Group N.V. (PHAR) - Canvas Business Model: Key Partnerships

Novartis: Original licensor of Joenja (leniolisib) for APDS

The relationship centers on the original license for leniolisib, now marketed as Joenja. Pharming Group N.V. reported Joenja revenue of US$15.1 million for the third quarter of 2025. Total revenue guidance for the full year 2025 is set between US$335.0 million and US$350.0 million.

Specialized Genetic Labs: Partnering for APDS diagnostic testing and VUS reclassification

Pharming Group N.V. partners with genetic testing companies to identify APDS patients. As of the January 2025 presentation, Pharming Group N.V. had identified over 1,200 patients in the U.S. with Variants of Uncertain Significance (VUSs). By May 2025, approximately 250 APDS patients were identified in the U.S., with over 160 patients aged 12 or older eligible for Joenja treatment. A published study suggests APDS prevalence may be up to 100-fold higher than previously estimated. The U.S. market contributed 92% of Joenja second quarter 2025 revenues.

Contract Manufacturing Organizations (CMOs): Outsourcing Joenja synthesis and supply chain

Joenja synthesis is outsourced to trusted CMOs that possess rare disease expertise. These CMOs maintain strict quality control protocols to ensure compliance with EMA, FDA, and TGA standards.

Patient Advocacy Groups: Collaborating with HAE International and APDS Alliance

Pharming Group N.V. presents scientific contributions related to Joenja at major medical meetings, such as the American College of Allergy, Asthma & Immunology (ACAAI) 2025 Annual Scientific Meeting. Seven posters highlighted real-world effectiveness of Joenja, including Phase III pediatric trial results.

The operational metrics tied to these partnerships and commercial execution in the first three quarters of 2025 are detailed below:

Metric Category Specific Data Point Value as of Late 2025 (Latest Reported) Citation Reference
Joenja Revenue (H1 2025) Revenue for the first half of 2025 US$23.3 million
Joenja Revenue (Q3 2025) Revenue for the third quarter of 2025 US$15.1 million
Joenja Patient Identification (U.S. VUS) Patients identified with VUS in APDS genes Over 1,200
Joenja Patient Identification (U.S. Eligible) Patients in the U.S. aged 12+ eligible for Joenja (as of May 2025) Over 160
Geographic Expansion Joenja commercial availability start date in the U.K. April 2025
Regulatory Milestone U.S. FDA PDUFA target action date for pediatric sNDA January 31, 2026

The company is preparing to submit a regulatory filing for pediatric label expansion to the U.S. FDA in the third quarter of 2025.

The company is on track to submit a regulatory filing with Japan's PMDA in mid-2025.

The company's overall 2025 total revenue guidance was raised to US$335 million - US$350 million as of the second quarter report.

Cash and marketable securities increased to US$130.8 million at the end of the second quarter of 2025.

Cash generated from operations was US$32.0 million in the third quarter of 2025.

The company targets an annual reduction in G&A expenses of 15% or US$10 million.

Total operating expenses guidance for 2025 is between US$304.0 million and US$308.0 million, assuming constant currency.

The company expects to record one-time restructuring costs of approximately $7 million in the fourth quarter of 2025.

The acquisition of Abliva AB, which impacts expense reporting, was completed in March 2025.

Finance: draft 13-week cash view by Friday.

Pharming Group N.V. (PHAR) - Canvas Business Model: Key Activities

You're looking at the core engine driving Pharming Group N.V.'s performance as of late 2025. The Key Activities section of the Business Model Canvas is where the company executes its strategy to deliver on its value propositions, and right now, that execution is heavily focused on commercial momentum and pipeline advancement.

Commercializing Ruconest and Joenja in the US and Europe remains central. The momentum is clear from the latest figures. For the third quarter of 2025, total revenues hit US$97.3 million. RUCONEST® continues to be the revenue backbone, bringing in US$82.2 million in Q3 2025, marking a 29% increase compared to the third quarter of 2024. Joenja® (leniolisib) is accelerating, with Q3 2025 revenue at US$15.1 million, a 35% year-over-year jump, driven by a 25% increase in patients on paid therapy. The US market is dominant for both products; for instance, in Q1 2025, the US accounted for 97% of RUCONEST® revenue and 90% of Joenja® revenue. The company has raised its full-year 2025 total revenue guidance to a range of US$365 - US$375 million.

The commercial focus is supported by these recent sales figures:

  • RUCONEST® revenue for the first nine months of 2025 reached US$231.2 million.
  • Joenja® Q3 2025 revenue was US$15.1 million, up from US$10.5 million in Q1 2025.
  • The company is actively managing its European and Rest of World access, which contributed 10% of Joenja® revenue in Q1 2025.

Research and Development (R&D) for pipeline assets like KL1333 shows Pharming Group N.V. is moving beyond its current marketed products. The acquisition of Abliva AB brought KL1333, targeting mitochondrial DNA-driven primary mitochondrial diseases, into the late-stage pipeline. While specific 2025 R&D spend isn't detailed here, the action is clear: advancing this asset toward Phase III readiness.

Global regulatory submissions are a critical near-term activity, especially for expanding Joenja®'s reach in the pediatric population. The US Food and Drug Administration (FDA) accepted the supplemental New Drug Application (sNDA) for children aged 4 to 11 years and granted it Priority Review, setting a Prescription Drug User Fee Act (PDUFA) target action date of January 31, 2026. Furthermore, the company was preparing to file its New Drug Application (NDA) for Joenja® in Japan mid-2025, and the European Committee for Medicinal Products for Human Use (CHMP) review was extended to January 2026.

Here's a snapshot of the regulatory progress:

Submission Target Indication/Age Group Key Regulatory Status (Late 2025) Target Date/Status
US FDA sNDA Joenja for APDS (4-11 years) Accepted, Priority Review Decision by January 31, 2026
Japan NDA Joenja for APDS Under Regulatory Review Filing mid-2025
EU CHMP Review Joenja for APDS (Pediatric) Under Review Extended to January 2026

Optimizing G&A expenses is a direct action to improve profitability, especially following the Abliva acquisition costs. Pharming Group N.V. announced a plan to reduce total General and Administrative (G&A) expenses by 15% annually, which translates to an expected saving of US$10 million per year. This is being executed via a restructuring that includes a 20% net reduction in non-commercial and non-medical headcount at the Netherlands headquarters, with one-time restructuring costs of approximately $7 million anticipated in the fourth quarter of 2025. You can see the impact of this focus on operating profit, which reached US$15.8 million in Q3 2025, a 285% increase year-over-year.

Expanding the APDS patient base through diagnostic efforts directly feeds the Joenja® revenue stream. The company is actively working to reclassify Variants of Uncertain Significance (VUSs) as confirmed APDS cases. As of early 2025, Pharming Group N.V. had identified more than 1,200 patients in the US with VUSs who could potentially be eligible for Joenja® treatment upon validation. This diagnostic push is based on new data suggesting APDS prevalence could be up to 100x higher than initially thought, moving the prevalence estimate from an initial ~1.5 patients / million to approximately 40 patients / million. Furthermore, the company is supporting patient access through programs, reporting 187 APDS patients on Expanded Access or Named Patient Programs as of Q1 2025. That's real work happening to find the unserved.

Pharming Group N.V. (PHAR) - Canvas Business Model: Key Resources

You're looking at the core assets Pharming Group N.V. relies on to drive its business forward. These aren't just line items; they're the tangible and intellectual property that generate revenue and fuel the pipeline. Honestly, the financial strength right now is directly tied to the performance of the two commercial products.

Commercial Products Performance Snapshot (Q3 2025 Preliminary Data)

Product Q3 2025 Revenue (US$) Year-over-Year Revenue Growth Key Metric Detail
Ruconest (C1-INH) US$82.2 million 29% increase vs. Q3 2024 Unit sales volume in the U.S. increased by 24% in Q3 2025.
Joenja (leniolisib) US$15.1 million 35% increase vs. Q3 2024 Patients on paid therapy grew by 25% year-over-year in Q3 2025.

The total revenue for the third quarter of 2025 hit US$97.3 million, showing that the core commercial engine is definitely running hot. To be fair, the company made a strategic move to withdraw Ruconest from all non-US markets, which streamlines focus but changes the global footprint resource.

The manufacturing capability for Ruconest remains a critical, though less detailed, resource. This is the recombinant production technology using transgenic rabbits for Ruconest, which provides the supply for this key therapy.

The pipeline asset, KL1333, a first-in-class asset for mitochondrial DNA-driven diseases, is supported by ongoing clinical work. The pivotal FALCON study has treated 180 total patients for 48 weeks, with plans to expand to 40+ total sites. Management anticipates the pivotal study readout in 2027.

The operational backbone includes the global commercial and medical affairs infrastructure. This network was previously noted as spanning across 30+ markets, though the recent strategic withdrawal of Ruconest from non-US markets will necessitate an update on the current active commercial footprint.

Financially, the company holds significant liquidity. The cash and marketable securities position stood at US$130.8 million as of the end of the second quarter of 2025, up from US$108.9 million at the end of the first quarter 2025, primarily from cash generated from operations.

Here are the key product revenue contributions for the first nine months of 2025:

  • RUCONEST® revenue for the first nine months of 2025 was US$241.1 million (Q1: US$68.6M + Q2: US$80.4M + Q3: US$82.2M, using reported Q1, Q2, Q3 figures).
  • Joenja® revenue for the first nine months of 2025 was US$38.4 million (Q1: US$10.5M + Q2: US$12.8M + Q3: US$15.1M, using reported Q1, Q2, Q3 figures).

Finance: draft 13-week cash view by Friday.

Pharming Group N.V. (PHAR) - Canvas Business Model: Value Propositions

You're looking at the core value Pharming Group N.V. delivers to its customers-the patients and prescribers in the ultra-rare disease space. It's all about providing specialized, life-altering treatments where few other options exist. This is where the company truly earns its keep.

On-Demand HAE Treatment: RUCONEST®

For Hereditary Angioedema (HAE) patients needing acute attack treatment, Pharming Group N.V. offers RUCONEST®, a recombinant C1-INH protein replacement therapy. This product is positioned as the on-demand treatment that targets the root cause via IV administration, which means rapid onset. Honestly, the numbers show its continued strength in the U.S. market, even with new oral competition.

Here's a quick look at how RUCONEST® performed in the third quarter of 2025:

Metric Value (Q3 2025) Comparison
Revenue US$82.2 million +29% versus Q3 2024
U.S. Volume Growth +24% In the third quarter of 2025
U.S. Volume Growth (YTD) +28% For the first nine months of 2025
Acute Attack Efficacy 97% Attacks treated with just 1 dose
Sustained Efficacy 93% Acute attacks stopped for at least 3 days

The strategic move to withdraw RUCONEST® from non-US markets underscores the focus on maximizing value where the demand and infrastructure are most robust, which is definitely the U.S. base.

Oral APDS Treatment: Joenja® (Leniolisib)

Joenja® is a major value driver, being the first and only approved oral treatment for Activated PI3K-delta Syndrome (APDS) in the 12 years and older segment. You see the acceleration in uptake reflected clearly in the financials as more patients get diagnosed and transitioned to paid therapy.

The growth story for Joenja® in the third quarter of 2025 is quite compelling:

  • Revenue reached US$15.1 million, a 35% year-over-year increase.
  • Unit sales volume grew by 34% in the third quarter of 2025.
  • The U.S. patient base on paid therapy reached 116 patients, up 25% year-over-year.
  • The company added 13 new APDS patients on therapy in the U.S. during Q3 2025 alone.
  • Joenja® revenue for the first nine months of 2025 was US$38.4 million.

Also, the potential market is expanding; the FDA granted priority review for the pediatric indication (ages 4 to 11), with a decision expected by January 2026.

Pipeline Targeting High Unmet Need

Pharming Group N.V.'s pipeline extends its value proposition into other ultra-rare diseases, leveraging the expertise gained from APDS. This is about future-proofing the revenue base with high-impact science.

For APDS, new research published in Cell suggests that reclassifying patients with a variant of uncertain significance (VUS) could imply up to a 100-fold increase in APDS prevalence, opening up a much larger addressable patient pool. Beyond that, the company has an additional 180 APDS patients in access programs and clinical studies globally.

For mitochondrial disease, the company is actively recruiting for the pivotal FALCON clinical trial studying leniolisib in mitochondrial DNA-driven primary mitochondrial diseases. The current cash position, which stood at US$168.9 million at the end of Q3 2025, is expected to cover these pipeline and pre-launch costs.

Patient-Centric Support

The value proposition is cemented by support programs that ensure continuity of care, which is critical in rare disease management. This is evidenced by the strong adherence rates for Joenja® and the continued growth in U.S. prescribers for RUCONEST®.

The company's overall 2025 total revenue guidance was raised to a range of US$365 million to US$375 million, reinforcing the market's acceptance of the value delivered by these specialized therapies.

Pharming Group N.V. (PHAR) - Canvas Business Model: Customer Relationships

You're looking at how Pharming Group N.V. maintains its connection with the specialized prescribers and the patients relying on its niche, often lifelong, therapies. This isn't about mass-market advertising; it's about deep, focused engagement in the rare disease space.

Dedicated Rare Disease Sales Force: Direct engagement with specialized prescribers.

  • The commercial strategy relies heavily on direct engagement with specialists.
  • The U.S. market is the core focus, contributing 97% of first quarter 2025 revenues.
  • Sustained growth in RUCONEST® reflects a consistent addition of prescribers.
  • Pharming Group N.V. added an average of 22 new prescribers over the past six quarters leading up to the third quarter of 2025.
  • For Joenja®, there was a 25% year-over-year increase in patients on paid therapy in the third quarter of 2025.

The relationship with the prescribing community is clearly driving volume, as seen in the revenue performance for their two key assets:

Metric Time Period Value/Amount
RUCONEST® Revenue Growth (YoY) Q3 2025 29%
Joenja® Revenue Growth (YoY) Q3 2025 35%
RUCONEST® Revenue Growth (YoY) First Nine Months 2025 34%
Joenja® Patients on Paid Therapy Growth (YoY) Q3 2025 25%

High-touch patient support programs (e.g., Ruconest support line).

For on-demand treatments like RUCONEST®, efficacy in the moment is paramount, which necessitates strong post-sale support and clear clinical data to reinforce trust.

  • RUCONEST® is mostly used by patients experiencing more severe/frequent attacks.
  • In a comparative real-world study, 97% of HAE attacks treated with RUCONEST® were resolved after the first dose.
  • Another study indicated 93% of acute attacks were stopped for at least 3 days following treatment.

Educational campaigns for immunologists and healthcare providers (HCPs).

The focus here is on establishing the product's unique value proposition, especially against newer competition, which requires ongoing education.

  • The sustained success of RUCONEST® is attributed to its unique profile and strong differentiation in the on-demand HAE market, even after a new oral acute product launched in July 2025.
  • For Joenja®, the company is preparing to file for U.S. FDA approval for pediatrics in the third quarter of 2025.
  • Pharming Group N.V. continues to work closely with global patient organizations.

Long-term relationships with patients due to chronic, lifelong treatments.

The nature of treating conditions like Hereditary Angioedema (HAE) and APDS (Activated PI3K-delta Syndrome) means the relationship is inherently long-term, often spanning the patient's life.

  • As of June 30, 2025, Pharming Group N.V. had identified 971 diagnosed APDS patients globally.
  • Of those, 257 patients were identified in the U.S..
  • For Joenja® in the U.S., patient numbers on paid therapy grew from 91 at the end of Q2 2024 to 114 at the end of Q2 2025.
  • The company expects to continue to identify and enroll new APDS patients, supported by VUS (Variant of Uncertain Significance) resolution efforts.
Finance: review Q4 2025 patient enrollment projections against the current sales force deployment by next Tuesday.

Pharming Group N.V. (PHAR) - Canvas Business Model: Channels

You're looking at how Pharming Group N.V. gets its therapies, like RUCONEST® and Joenja®, from the lab to the patient, which is a critical part of their rare disease strategy. For orphan drugs, the channel is never just a simple drop-ship; it's highly specialized and relationship-driven.

Specialty Pharmacies and Distributors: Direct distribution of orphan drugs.

Pharming Group N.V. relies on a specialized distribution network, which is typical for high-cost, low-volume orphan drugs. The global specialty drug distribution market was valued at approximately USD 298.48 billion in 2024, indicating the complex environment they operate within. For Pharming Group N.V., the channel focus is heavily skewed geographically, with the U.S. market driving the vast majority of sales. In the second quarter of 2025, the U.S. market accounted for 92% of total revenues, leaving only 8% from the EU and the Rest of World. This concentration means channel management is intensely focused on securing access through key specialty pharmacies and distributors capable of handling these specific therapies, often within limited or exclusive networks.

Direct Sales Teams: Targeting US and European HAE/Immunology specialists.

The sales channel is built around specialists who educate prescribers on Hereditary Angioedema (HAE) and Activated PI3K Delta Syndrome (APDS). The growth in revenue reflects the success of these teams in increasing prescribers. For instance, RUCONEST® second quarter 2025 revenue grew by 28% year-over-year, reflecting strong growth in patients and prescribers. The sales effort is clearly weighted toward the U.S., which is the primary revenue engine. The company is actively managing its cost structure, announcing a plan to reduce general and administrative (G&A) expenses by 15% or US$10 million in 2025 to optimize capital allocation, which would impact the size and efficiency of these teams.

Digital Patient Tools: Joenja patient app for adherence and support.

For Joenja, patient support is integrated directly into the channel to drive adherence and manage the complex journey from diagnosis to paid therapy. While specific patient app usage numbers aren't public, the acceleration in patient uptake is clear. As of June 30, 2025, Pharming Group N.V. had 114 patients on paid Joenja therapy in the U.S., a 25% increase from the prior year's second quarter. The company supports this with the APDS Assist program, which helps enroll patients and navigate insurance requirements. The first half of 2025 saw Joenja patient growth surpass the total increase for all of 2024.

Regulatory Filings: Securing market access via FDA, EMA, NICE, and TGA approvals.

Regulatory success is a prerequisite for channel activation in new markets. Pharming Group N.V. has made significant progress in securing market access throughout 2025:

  • Joenja launched in England and Wales in April 2025 after NICE positive final guidance.
  • TGA approval was secured in Australia in the first quarter of 2025.
  • A supplemental New Drug Application (sNDA) for pediatric Joenja use (ages 4 to 11) was submitted to the U.S. FDA in the third quarter of 2025.
  • The EMA review for APDS patients 12 years and older is on track, with a response due by the January 2026 deadline.
  • A New Drug Application for leniolisib was submitted in Japan.

Here's a quick look at the channel performance metrics as of mid-2025:

Metric Value/Period Source Context
Q3 2025 Total Revenue US$97.3 million Third Quarter 2025 Results
Q3 2025 Joenja Revenue US$15.1 million Third Quarter 2025 Results
US Patients on Paid Joenja Therapy (as of Q2 2025) 114 patients Second Quarter 2025 Results
US Market Revenue Contribution (Q2 2025) 92% Second Quarter 2025 Results
Total Diagnosed APDS Patients Globally (as of Q2 2025) 971 patients Second Quarter 2025 Results
2025 Total Revenue Guidance (Raised July 2025) US$335 million - US$350 million Second Quarter 2025 Results

If onboarding takes 14+ days, churn risk rises, which is why the APDS Assist program is defintely a key part of the channel support structure. Finance: draft 13-week cash view by Friday.

Pharming Group N.V. (PHAR) - Canvas Business Model: Customer Segments

You're looking at the core patient populations Pharming Group N.V. serves as of late 2025, which directly drives the revenue from their two key commercial assets, RUCONEST® and Joenja®. The focus is intensely specialized, targeting rare diseases where they hold or seek leading positions.

Hereditary Angioedema (HAE) patients requiring on-demand treatment

This segment is served by RUCONEST® (C1 esterase inhibitor, lyophilized for intravenous use), which remains the financial bedrock for Pharming Group N.V. The growth in this segment is evident in the top-line numbers. For the third quarter of 2025, RUCONEST® revenue hit US$82.2 million, marking a 29% increase compared to the third quarter of 2024. Looking at the longer trend, revenue for the first nine months of 2025 reached US$231.2 million, a 34% jump year-over-year. This sustained performance is fueled by growth in both the number of patients and the prescribers in the competitive U.S. HAE market. To be fair, this segment is facing new competition, as the company noted the launch of a new oral on-demand therapy in July 2025, but RUCONEST® still showed strong volume growth in the U.S. during that quarter.

Activated PI3K Delta Syndrome (APDS) patients in the US and EU

The customer base for Joenja® (leniolisib) is the population diagnosed with Activated PI3K Delta Syndrome (APDS). As of September 30, 2025, Pharming Group N.V. had identified 990 diagnosed APDS patients globally. The U.S. market is the primary focus for Joenja® uptake, with 270 patients identified there as of the end of Q3 2025. Of those U.S. patients, 175 were 12 years of age or older and eligible for Joenja® treatment. This segment is rapidly expanding its revenue contribution; Q3 2025 Joenja® revenue was US$15.1 million, a 35% increase year-over-year. For the first nine months of 2025, Joenja® generated US$38.4 million in revenue. The company is also actively working to reclassify patients with a Variant of Uncertain Significance (VUS) in the implicated genes, with over 1,400 such U.S. patients identified as of mid-2025.

Here's a quick look at the revenue contribution from these two rare disease segments for the third quarter of 2025:

Indication/Product Q3 2025 Revenue (US$) Year-over-Year Growth
HAE (RUCONEST®) 82.2 million 29%
APDS (Joenja®) 15.1 million 35%
Total Revenue 97.3 million 30%

Immunologists and Hematologists who treat rare immune deficiencies

These specialists are the key channel partners and prescribers. For the HAE segment, Pharming Group N.V. increased its RUCONEST® physician prescriber base by 11% during the full year 2024, adding many previously unknown HAE prescribers. This indicates a continuous effort to map and engage the treating specialists for HAE. For APDS, the growth in Joenja® revenue is driven by a 25% year-over-year increase in patients on paid therapy in the U.S. as of Q3 2025, which requires close collaboration with the specialized Immunologists and Hematologists who diagnose and manage APDS.

Pediatric patients (future segment) following planned Q3 2025 FDA filing for Joenja

This represents a significant near-term expansion opportunity for Joenja®. Pharming Group N.V. planned an FDA filing for children aged 1-11 years in the third quarter of 2025. The U.S. Food and Drug Administration (FDA) granted Priority Review for the supplemental New Drug Application (sNDA) for children aged 4 to 11 years, with a decision date set for January 31, 2026. As of September 30, 2025, there were 54 identified U.S. patients aged 4 to 11 who would become eligible upon regulatory approval. This group is about one-quarter of all APDS patients currently identified in the U.S.. Successful approval could unlock an additional €10-15 million in annual peak sales.

The overall confidence in the commercial trajectory, driven by these segments, led Pharming Group N.V. to raise its total 2025 revenue guidance to between US$365 million and US$375 million as of November 2025.

Pharming Group N.V. (PHAR) - Canvas Business Model: Cost Structure

You're looking at the core expenditures that fuel Pharming Group N.V.'s operations as of late 2025. This structure is heavily weighted toward supporting the commercialization of existing assets and advancing the late-stage pipeline, especially following the Abliva acquisition.

The overall spending envelope for the year is clearly defined. Pharming Group N.V. guided Total Operating Expenses for Fiscal Year 2025 between US$304 million and US$308 million. This guidance already factors in the costs associated with the Abliva acquisition, specifically noting an expected US$10.2 million in non-recurring Abliva-related transaction and integration expenses within that total operating expense figure.

The company is also planning for a one-time hit in the final quarter. Expect non-recurring restructuring costs of approximately $7 million to be recorded in Q4 2025. This is tied to an organizational restructuring that includes a 20% net reduction in non-commercial and non-medical headcount, primarily at the Netherlands headquarters.

Here's a look at the costs associated with producing the company's products, which are complex biologics and small molecules.

Cost Component Q2 2025 Amount (US$m) 1H 2025 Amount (US$m)
Cost of Sales (COGS) 9.0 17.3

The Cost of Goods Sold, or Cost of Sales, for the second quarter of 2025 was US$9.0 million, bringing the total for the first half of 2025 to US$17.3 million. This reflects the costs inherent in manufacturing their specialized products like RUCONEST®, a complex biologic.

Research and Development (R&D) expenses are significant, driven by late-stage clinical work. The pivotal FALCON clinical trial, evaluating KL1333 for primary mitochondrial disease, is a major cost center. The total incremental cost related to KL1333, covering the purchase price, transaction/integration, and development costs, is estimated to be approximately US$133 million ahead of the expected FDA approval in 2028. For the first nine months of 2025, non-recurring Abliva acquisition-related expenses included US$2.1 million in R&D.

Sales, General, and Administrative (SG&A) costs support the global commercial teams for products like RUCONEST® and Joenja®. The company is actively managing these overheads. They remain on track to reduce total G&A expenses by 15% or US$10 million annually as part of the restructuring plan. Non-recurring Abliva acquisition-related expenses impacted SG&A as well, with US$8.0 million recorded in G&A for the first nine months of 2025.

You can see how the Abliva-related costs are distributed across the operating expense categories:

  • Non-recurring Abliva acquisition-related expenses totaled US$10.2 million within the FY 2025 Operating Expenses guidance.
  • For the first nine months of 2025, these non-recurring costs included US$8.0 million in G&A and US$2.1 million in R&D.
  • In Q1 2025 alone, non-recurring Abliva costs included US$5.7 million in G&A and US$2.1 million in R&D.

The company is definitely making structural changes to manage its cost base going forward. Finance: draft 13-week cash view by Friday.

Pharming Group N.V. (PHAR) - Canvas Business Model: Revenue Streams

You're looking at the core income sources for Pharming Group N.V. as of late 2025. The revenue picture is clearly defined by two primary pharmaceutical products, with the full-year outlook recently updated based on strong mid-year performance.

The revenue streams are built around the commercial success of its two main assets. Ruconest, for acute Hereditary Angioedema (HAE) attacks, continues to be a major cash generator, especially in the U.S. market, even with new competition entering the space. Joenja (leniolisib), for the treatment of APDS (Activated PI3K-delta Syndrome), is showing significant growth as patient uptake accelerates.

Here's a breakdown of the key financial figures driving the revenue streams as reported through the third quarter of 2025:

  • - Ruconest product sales for acute HAE attacks.
  • - Joenja (leniolisib) product sales for APDS.
  • - Total 2025 revenue guidance is US$365 million - US$375 million.
  • - Q3 2025 Ruconest revenue was US$82.2 million.
  • - Q3 2025 Joenja revenue was US$15.1 million.

The third quarter of 2025 showed robust performance, leading Pharming Group N.V. to raise its full-year revenue expectations. The total revenue for the third quarter of 2025 hit US$97.3 million, a 30% increase compared to the third quarter of 2024. This momentum is what underpins the revised full-year guidance.

You can see the specific contributions from the two key products in the table below, which also includes the year-to-date performance for the first nine months of 2025.

Revenue Component Q3 2025 Revenue (US$ million) 9M 2025 Revenue (US$ million)
RUCONEST 82.2 231.2
Joenja (leniolisib) 15.1 38.4

The growth in Joenja revenue, which increased by 35% in the third quarter compared to the prior year's third quarter, reflects strong growth in patients on paid therapy. Meanwhile, RUCONEST revenue grew by 29% year-over-year for the quarter, driven by sustained growth in both patients and prescribers in the HAE market. The company noted that RUCONEST is well positioned to provide continued strong cash flows. The revenue from other markets outside of the main focus areas contributed only US$1.1 million, or 1.3% of total RUCONEST revenue in the current quarter, and these markets have not shown financial sustainability.

The overall financial expectation for the year is clear:

  • The 2025 total revenue guidance was raised to US$365 million - US$375 million, up from the prior guidance of US$335 million - US$350 million.
  • The revenue for the first nine months of 2025 reached US$231.2 million from RUCONEST alone.

This revenue base is what management is using to fund pipeline opportunities and cover current operating expenses, which are projected between US$304 million and US$308 million for the full year 2025, before a one-time restructuring cost of approximately $7 million expected in the fourth quarter.


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