Pharming Group N.V.

Pharming Group N.V. is a biopharmaceutical company based in the Netherlands. Through its world-leading research and development, Pharming is dedicated to creating new treatments and therapies that address rare diseases and unmet medical needs in the United States, Europe, and internationally. The company’s lead product, Ruconest, is a recombinant human C1 esterase inhibitor that is used for the treatment of acute hereditary angioedema, a rare and potentially life-threatening condition caused by the sudden swelling of body parts due to a malfunction in the immune system.

Pharming is also involved in the research and development of a number of projects, including rhC1INH, a recombinant human C1 inhibitor, which is being developed for the treatment of pre-eclampsia, acute kidney injury, and Covid-19; leniolisib, a phosphoinositide 3-kinase delta inhibitor (PI3K delta) being developed to treat patients with activated PI3K delta syndrome; and alpha-glucosidase therapy for the treatment of pompe and fabry diseases.

The company has several agreements and collaborations to advance research and development. A development and license agreement was established with Novartis for the development, manufacture, and commercialization of drugs and therapies for rare diseases. Pharming has also formed a strategic partnership with Orchard Therapeutics plc to jointly research, develop, manufacture, and commercialize OTL-105, an investigational ex-vivo autologous hematopoietic stem cell gene therapy for the treatment of hereditary angioedema.

The company has experienced several significant milestones in its history, including the commercialization of Ruconest in 2015, the first European approval of Ruconest in 2016, and U.S. Food and Drug Administration (FDA) approval of Ruconest in 2019. Additionally, Pharming has a number of product candidates in various stages of development, including a recombinant human C1 inhibitor (rhC1INH) for the treatment of acquired angioedema and a recombinant human Antithrombin for the treatment of thrombotic thrombocytopenic purpura.

Pharming continues to have a strong focus on developing novel therapies for rare diseases and unmet medical needs, and seeks to expand its portfolio of products through collaborations and strategic partnerships. The company is committed to providing new treatments and therapies to those affected by rare diseases, and looks forward to continuing to make a positive impact on patient care.