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


Oss, the Netherlands, 1 May 2024 — Pharming, a global biopharmaceutical company developing and commercializing innovative protein replacement therapies and precision medicines to serve the unserved rare disease patient, is pleased to announce its recognition as an Industry Innovator by the National Organization for Rare Disorders (NORD®) at the Rare Impact Awards on 8 June 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. 


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.” 


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

You can find the full press release here.


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The development of medicines in Oss began one hundred years ago. It was then that the foundations were laid for what would become a flourishing pharmaceutical sector in the Netherlands, leading to groundbreaking discoveries such as insulin and the anti-conception pill.

Today, the entire chain is still represented in Oss, from early-stage research to the commercial scale production of medicines. Working in Oss, we focus on the health of people all over the world. Employees at dozens of companies large and small put their heart and soul into new medicines, products and services that will improve lives worldwide. Together, our strength lies in our combination of broad knowledge about the development of medicines and a thorough understanding of the requirements of high-quality production.

This shared past and common values stimulate strong bonds among everyone who works in pharmaceuticals in Oss. We build on qualities, expertise and networks that go back decades. And with a wide variety of pharmaceutical companies, suppliers and scientific institutions all located nearby, we offer an attractive business climate and an excellent basis for international collaboration.

We are proud to work together to create a better and healthier future for all, a collaboration that in turn fuels growing employment: today, most pharmaceutical jobs in the Netherlands are in Oss. Here we connect talent and companies and stimulate entrepreneurship, while our expert employees are our most important asset. Their drive ensures that Oss has an influence on health all over the world.

Pharma in Oss offers endless opportunities to startups and talent in a city and region that is a joy to live and work in. We are committed to collaboration and sustainable and successful entrepreneurship. Oss’s pharmaceutical companies are intrinsically competitive while also being committed to quality and safety. This, plus their focus on new health solutions and contemporary technology, makes us a leader in our pursuit of a better life for people worldwide.