PH-1 (Recombinant Human C1 Esterase Inhibitor)
Primary Mitochondrial Diseases (e.g., Leigh Syndrome, MELAS)
PivotalPivotal-stage clinical program
Key Facts
Indication
Primary Mitochondrial Diseases (e.g., Leigh Syndrome, MELAS)
Phase
Pivotal
Status
Pivotal-stage clinical program
Company
About Pharming Group
Pharming Group is a commercial-stage biopharmaceutical company dedicated to serving the unserved rare disease patient. Its strategy is built on a proven operational framework for rare disease drug development, regulatory strategy, and commercialization, currently anchored by its flagship product, Joenja® (leniolisib), for Activated PI3K Delta Syndrome (APDS). The company is executing on a dual-track strategy of maximizing its existing portfolio while advancing a promising pipeline, including a pivotal-stage program in primary mitochondrial diseases, to drive sustainable long-term growth.
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