PHAR | Market Cap: $972.5M (07/13/26)
Industry:
Pharma & Biotech
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DESCRIPTION

Pharming is a Netherlands-based rare disease biotech that develops and sells treatments for rare genetic and immunological conditions. Its two marketed products drive all revenue. RUCONEST is an intravenous, on-demand recombinant C1 esterase inhibitor for acute hereditary angioedema attacks — a rare genetic condition causing unpredictable, potentially life-threatening swelling. RUCONEST is sold exclusively in the U.S. and serves as the core cash engine of the business, generating ~85% of revenue. Joenja (leniolisib) is an oral, once-daily PI3Kδ inhibitor and the only approved targeted treatment for activated PI3Kδ syndrome, an ultra-rare primary immunodeficiency. Joenja is a disease-modifying therapy approved in the U.S., U.K., Australia, Israel, and Japan, and generates the remaining ~15% of revenue. Both products are sold through Pharming's own commercial infrastructure to specialist physicians at academic medical centers. Pharming prices both products at orphan drug premiums, and gross margins are very high, reflecting the lack of competing therapies. RUCONEST generates recurring cash flow, as HAE patients experience attacks throughout their lives, which Pharming uses to fund Joenja's commercialization and a broader pipeline. Growth levers include expanding Joenja geographically, pursuing a pediatric label, reclassifying underdiagnosed patients, and running Phase II studies in larger adjacent immunodeficiency indications. Pharming's pipeline also includes napazimone, a drug in a pivotal trial for primary mitochondrial disease, acquired via its 2025 purchase of Abliva.

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