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. 

For more information about the Rare Impact Awards and to view the full list of honorees, please click here.

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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.”