The recent approval of Joenja® (leniolisib) in the European Union marks another step in that evolution – one from a company long associated with one commercial asset into a broader global rare-disease business. With two commercial products, an expanding international footprint and a pipeline spanning immune dysregulation and primary mitochondrial disease, Pharming is entering a different phase.

Patient purpose as the compass

For CEO Fabrice Chouraqui, the company’s strategy is rooted in the realities faced by people living with rare diseases. Earlier this year, he told shareholders about Victoria, a woman living with hereditary angioedema whose path to diagnosis was “long, frustrating, and often frightening”.

That experience is common in rare disease, where people can spend years searching for the specialist, test or diagnosis that finally changes the course of care. Scientific progress matters, but its real value lies in creating new possibilities for people who have waited too long for answers.

 “For me, Victoria’s story captures why Pharming exists,” Chouraqui said. “We are here to restore possibilities for people affected by serious rare diseases. That patient purpose must remain our compass.”

Activated phosphoinositide 3-kinase delta syndrome (APDS) illustrates the point. Its symptoms can overlap with other immune disorders, and genetic testing and specialist knowledge are often needed to identify the underlying cause.

Recent regulatory progress in Europe is an important step, but approval is only the beginning: diagnosis, reimbursement, physician awareness and patient identification determine how quickly innovation reaches those who may benefit.

A broader center of gravity

“Over the past few years, Pharming has changed significantly,” Chouraqui said. “We have evolved from a company largely defined by one asset into a company with two commercial medicines, a durable revenue base, and a high-value pipeline with the potential to transform our scale and profile.”

 RUCONEST still matters greatly in that equation. It gave Pharming commercial experience, infrastructure and operating cash flow, and remains a durable foundation.

“RUCONEST remains central to the foundation we have built,” Chouraqui said. “But Joenja® is where much of the next phase of growth begins.”

Pharming now occupies an unusual middle ground: it retains the upside and pipeline risk of a focused biotech, but with the revenues, infrastructure and operating discipline of a commercial biopharma organization. In 2025, it reported 27% revenue growth, an operating profit and US$54.7 million in net cash flow from operations. The growth story is no longer only prospective; it is supported by a business already operating at scale.

Joenja at the start of its lifecycle

Joenja is Pharming’s most visible near-term growth driver. The approval in the EU and Japan follows the launch in the US and the UK, while patient-identification and access work continue.

Pharming is also studying leniolisib beyond APDS in broader primary immunodeficiencies with immune dysregulation, conditions with larger patient populations and significant unmet medical needs. Management sees Joenja and the broader leniolisib development program as significant elements of a possible route toward annual revenues in excess of US$1 billion, assuming the programs deliver clinically and commercially.

A pipeline that could change the scale

Napazimone (KL1333), added through the acquisition of Abliva, broadens Pharming’s pipeline into another genetically defined rare disease setting. The late-stage program is being developed for primary mitochondrial disease, where extreme fatigue and muscle weakness can substantially limit daily life and treatment options remain limited.

The addition allows Pharming to apply its rare-disease capabilities to a late-stage opportunity with the potential to contribute meaningfully to future growth.

Execution becomes the differentiator

Pharming’s ambition is clear: to build a leading global rare and ultra-rare disease company with Dutch roots and global reach.

But portfolio ambition only goes so far.

Rare-disease competition increasingly tests the entire system around a medicine: diagnosis, evidence, manufacturing, access, specialist engagement, and patient support. The companies that lead will be those that connect science to the patient journey and execute consistently across markets.

 “The beginning of 2026 has reminded us that biotech is never without challenges and that we need to continue to strengthen our execution capabilities,” Chouraqui said.

The company is no longer a Dutch biotech defined by a single product, but it is not yet the fully diversified global rare-disease company it aims to become.

The components of that next chapter are visible: RUCONEST as the commercial foundation, Joenja as the near- and medium-term growth asset, with its existing license and development in broader primary immunodeficiencies, and napazimone (KL1333) as a late-stage program that could materially broaden its scale.

Pharming has made clear that it will not pursue growth for its own sake: “We will invest where we see the potential to expand and de-risk our pipeline, strengthen our strategic position, and generate attractive returns for shareholders,” Chouraqui said. “Our growth ambition must be matched by a clear path to value creation.”

The story is no longer about one product or one approval. It is about whether a Dutch-founded company can turn rare-disease expertise, financial discipline and a broader pipeline into sustained growth, while creating more possibilities for patients who still have too few.

Note: Some Pharming programs involve investigational product candidates for which safety and efficacy have not been established by regulatory authorities. Other programs relate to approved products being studied for additional indications, age groups, or geographic expansion. There is no guarantee that any investigational program or regulatory submission will receive approval or become commercially available for the uses being investigated.