Pharming获得欧盟支持罕见免疫缺陷疗法

2026-03-28 00:10

Pharming Group (PHAR) announced on Friday that an expert panel of the European Medicines Agency, the EU drug regulator, issued a positive opinion recommending marketing authorization for Joenja, a treatment for a rare form of immunodeficiency.
With its marketing authorization application, the Dutch drugmaker seeks EU approval of Joenja for patients aged 12 years and older with activated phosphoinositide 3-kinase delta syndrome (APDS), which is estimated to affect roughly 1–2 individuals per million globally.
The oral inhibitor of phosphoinositide 3-kinase delta is already available in countries, including the U.S. and U.K.
The European Commission will next review the decision from EMA’s human medicines committee within about two months. If authorized, Joenja will be indicated across 27 EU member states as well as Norway, Iceland, and Liechtenstein as the first approved therapy for APDS.

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