03.27.23
Pharming Group N.V. has received approval from the U.S. FDA for Joenja (leniolisib), for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older.
Joenjar, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency. The FDA evaluated the Joenja application for APDS under Priority Review, which is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions.
Joenja is expected to launch in the U.S. in early April.
"This FDA approval of Joenja is an important milestone for people living with APDS who will now have access to the first approved treatment option specifically for this debilitating disease,” said Sijmen de Vries, CEO of Pharming. “Until now, management of APDS has relied on the treatment of the diverse symptoms associated with APDS. We are grateful to the patients, caregivers, and physicians who participated in the clinical trials who have made today's approval a reality. I would also like to thank the Pharming and the Novartis teams who have supported the development of Joenja and can, therefore, be justifiably proud of this FDA approval. Today also marks a landmark event for Pharming and demonstrates our commitment to transforming the lives of patients who suffer from rare diseases.”
The approval is based on findings from a multinational, triple-blind, placebo-controlled, randomized Phase II/III clinical trial, which evaluated efficacy and safety in 31 patients diagnosed with APDS aged 12 years and older. Also submitted as part of the application were data from a long-term, open-label extension clinical trial in which 38 patients received Joenja for a median of two years.
Joenjar, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency. The FDA evaluated the Joenja application for APDS under Priority Review, which is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions.
Joenja is expected to launch in the U.S. in early April.
"This FDA approval of Joenja is an important milestone for people living with APDS who will now have access to the first approved treatment option specifically for this debilitating disease,” said Sijmen de Vries, CEO of Pharming. “Until now, management of APDS has relied on the treatment of the diverse symptoms associated with APDS. We are grateful to the patients, caregivers, and physicians who participated in the clinical trials who have made today's approval a reality. I would also like to thank the Pharming and the Novartis teams who have supported the development of Joenja and can, therefore, be justifiably proud of this FDA approval. Today also marks a landmark event for Pharming and demonstrates our commitment to transforming the lives of patients who suffer from rare diseases.”
The approval is based on findings from a multinational, triple-blind, placebo-controlled, randomized Phase II/III clinical trial, which evaluated efficacy and safety in 31 patients diagnosed with APDS aged 12 years and older. Also submitted as part of the application were data from a long-term, open-label extension clinical trial in which 38 patients received Joenja for a median of two years.
