At the presentation of the Pharming Group N.V. preliminary (unaudited) financial report for the full year ended 31 December 2022, CEO Sijmen de Vries comments:

“2022 was a pivotal year for Pharming. It was a year in which we prioritized our efforts in rare diseases and affirmed our commitment to our purpose of serving the unserved rare disease patient. It is this priority and commitment that forms the foundation as we build Pharming into a leading, global rare disease company across multiple geographies, with multiple products, a well-defined pipeline and with the necessary commercial infrastructure in place to enhance our future ambition.

In 2022, we prioritized our efforts on rare diseases and executed on a number of our strategic objectives to help build a sustainable global rare disease business focused on RUCONEST® sales. We significantly expanded our organization and headcount in 2022. These investments are in line with our strategy to become a global, multi-product, rare disease company.

In line with our renewed focus on rare diseases, we are advancing the development of our pipeline through a combination of internal development projects – including OTL-105 as a gene therapy for HAE.

Pharming has an ongoing strategic collaboration with Orchard Therapeutics to research, develop, manufacture and commercialize OTL-105, a newly disclosed investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy for the treatment of HAE. The program has made good progress developing the lentiviral vector to enhance C1-inhibitor expression and is now testing in preclinical HAE disease models. We anticipate providing further updates as we move towards preparing an Investigational New Drug (IND) filing.

While Pharming faces competition within the HAE market, the continued need for effective and reliable treatments for acute attacks, including breakthrough attacks in patients on prophylactic therapy, as well as RUCONEST®’s distinct advantages as the only recombinant treatment that targets the root cause of HAE by replacing missing or dysfunctional C1-INH, allows for sustainability in RUCONEST®’s revenues.”
(Source: Pharming)