At the presentation of the Pharming Group N.V. preliminary (unaudited) financial report for the full year ended 31 December 2021, CEO Sijmen de Vries says:

“2021 marked the beginning of an important period in Pharming’s development. The strength of our commercial business, which we continued to build on during the year through the reimbursement of Ruconest in new markets and expanding the reach of the product through new licensing agreements, has enabled us to increase our investment in long-term growth to achieve a number of additional strategic goals. Also, we were able to make an upfront payment for an investigational gene therapy for the potentially curative treatment of HAE, OTL-105. This product leverages our significant in-house expertise in HAE as we remain focused on driving research and development of specialist products through our existing business and through further in-licensing and acquisition opportunities.”

Operational highlights – Ruconest

  • Reimbursement of Ruconest (recombinant human C1 esterase inhibitor, or “rhC1INH”) agreed with the Spanish Ministry of Health for the treatment of acute HAE attacks in Spain.
  • Exclusive license agreement signed with NewBridge Pharmaceuticals for the distribution of Ruconest in the Middle East and North Africa.
  • Renewed strategic manufacturing partnership with Sanofi. Extended five-year contract, with options for extension, ensures the continuation of the downstream processing in the production of Ruconest.

Earlier stage pipeline – OTL-105

  • Strategic collaboration with Orchard Therapeutics, a global gene therapy leader, to research, develop, manufacture, and commercialize OTL-105, an investigational ex-vivo autologous hematopoietic stem cell (HSC) gene therapy for the treatment of HAE.
    (Source: Pharming)