Pharming to be honored as Industry Innovator at National Organization for Rare Disorders (NORD) 2024 Rare Impact Awards

Recognition given for achievement in commercializing Joenja (leniolisib), a first-in-class medication brought to market 10 years after disease state, APDS, was first characterized

Leiden, the Netherlands, April 24, 2024: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) is pleased to announce its recognition as an Industry Innovator by the National Organization for Rare Disorders (NORD) at the Rare Impact Awards on June 8, 2024. The Rare Impact Awards honor individuals, groups and organizations who are making exceptional strides to improve the lives of people living with rare diseases.

Pharming will accept the award in recognition of the U.S. Food and Drug Administration (FDA) approval and commercialization of Joenja (leniolisib), an oral, selective PI3Kδ inhibitor. Joenja is the first and only treatment approved in the U.S. for activated phosphoinositide 3-kinase delta syndrome (APDS) in adult and pediatric patients 12 years of age and older. Approximately two weeks after Joenja was approved for use on March 24, 2023, Pharming announced that the first shipments to patients in the U.S. were delivered.

Peter Saltonstall, President and CEO of NORD, commented:
“NORD is very pleased to recognize Pharming for the approval of Joenja in the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS). APDS is a rare inborn error of immunity characterized primarily by frequent infections, lymphoproliferation and autoimmunity for which there was not a targeted therapy until FDA approval of Joenja for both adults and pediatrics 12 years of age and older. On behalf of the approximately 500 individuals in the US with this rare condition, NORD is thrilled to see such innovation taking place at Pharming. With 95% of rare diseases not having therapies available, the approval of Joenja for APDS is worthy of the NORD Industry Innovation Award.  NORD congratulates Pharming for this achievement.”

Stephen Toor, Chief Commercial Officer of Pharming, commented:
“We are honored to be recognized by NORD as an industry leader making a difference in the lives of those living with and caring for someone living with a rare disease. Of the more than 7,000 rare diseases that have been identified, very few have an FDA-approved therapy. Pharming is proud to provide a treatment option for those living with APDS. This is a significant milestone for the scientists who developed Joenja and the patients we serve. We look forward to commemorating this achievement alongside members of the APDS community later this year.”