Pharming Group NV: Human protein replacement therapies, initially in rare/ultra-rare diseases, plus new small molecule program for APDS. RUCONEST (rhC1INH), the only recombinant enzyme replacement therapy marketed for acute Hereditary Angioedema (HAE) attacks and being developed for large unmet indications. Profitable and cash flow positive – revenue from product sales €134M (2018) and €123M (1st 9 months 2019). Growth (9 mo. YOY) in revenues (+26%), operating profit (+38%) and net profit (+73%). Development pipeline includes RUCONEST in new versions (LCM), new license from Novartis of late stage drug Leniolisib for APDS (ultra-rare immunodeficiency condition), rhC1INH for new larger indications including contract-induced nephropathy, pre-eclampsia and AKI, recombinant alpha-Glucosidase for Pompe disease
Based in...
Clinical Stage
Disease Space
Anti-inflammatory, Autoimmune, Rare Disease
Profitable, Revenues
Europe, Public
Market Cap
500MM - 1B
Therapeutic Modalities
Recombinant DNA
Darwinweg 24
Leiden, 2333 CR

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