Pharming Group NV ADS Trading Resumes

Pharming Group NV ADS trading has resumed after a temporary halt.

Pharming Group NV's American Depositary Shares (ADS) trading has resumed following a temporary halt. The company, known for its innovative approaches in rare disease treatment and diagnostics, has been making significant strides in the sector. This resumption comes at a time when the company is experiencing substantial growth and strategic advancements.

Pharming Group, listed on Euronext Amsterdam and Nasdaq, has been focusing on precision medicine and scalable diagnostics to address the "variant of uncertain significance" (VUS) problem—a major barrier in precision medicine. The company has made notable breakthroughs in rare disease diagnostics and therapeutics, including the use of CRISPR-based diagnostics and leniolisib (Joenja®) [1].

The company's recent landmark study, published in June 2025, utilized CRISPR base editing to functionally classify over 100 VUS in the PIK3CD and PIK3R1 genes linked to Activated Phosphoinositide 3-kinase Delta (APDS) Syndrome. This work redefined APDS prevalence and validated leniolisib as a first-line treatment [1]. Moreover, the study revealed resistance patterns in PIK3R1 variants, opening pathways for combination therapies with mTORC1/2 inhibitors.

Pharming's therapeutic pipeline is expanding, with leniolisib advancing into new indications. A supplemental New Drug Application (sNDA) for pediatric use (ages 4–11) is slated for Q3 2025, with FDA approval expected by mid-2026. Phase II trials in common variable immunodeficiency (CVID) and other primary immunodeficiencies (PIDs) are also underway, with read-outs in 2026. These trials could expand leniolisib's addressable market beyond APDS [1].

The company's commercialization strategy is built on global regulatory filings, partnerships, and real-world evidence generation. Pharming has submitted an NDA for leniolisib in Japan, with a decision expected in nine months. A favorable NICE reimbursement decision in the U.K. has already facilitated Joenja's launch in April 2025, and patient enrollment in Europe is accelerating [1].

Pharming's financial performance in H1 2025 reinforces its credibility as a value creator. Total revenues rose 26% year-over-year to $93.2 million, driven by 28% growth in RUCONEST® (hereditary angioedema treatment) and 15% growth in Joenja®. Operating profit surged to $10.8 million in Q2 2025, up from a $3.1 million loss in Q2 2024. The company raised its full-year revenue guidance to $335–350 million, reflecting confidence in its commercial execution and pipeline progress [1].

With a robust pipeline, expanding market access, and a strong balance sheet, Pharming is well-positioned to deliver outsized shareholder returns over the next 3–5 years. The resumption of ADS trading indicates investor confidence in the company's strategic momentum and growth prospects.

References:
[1] https://www.ainvest.com/news/pharming-group-strategic-momentum-rare-disease-therapies-catalyst-long-term-shareholder-2508/