Pharming secures novel EU approval for Joenja in activated PI3K delta syndrome

The European Commission (EC) has authorised Joenja (leniolisib) as the first treatment for activated PI3K delta syndrome (APDS) in the EU, following first characterisation of the disease in 2013.

Pharming Group’s oral small molecule phosphoinositide 3-kinase (PI3K) delta inhibitor, is indicated for patients 12 years old and over with the rare primary immunodeficiency.

Leverne Marsh, Chief Commercial Officer of Pharming, commented that the EC’s approval “marks an important milestone for the European APDS community, making Joenja the first therapy in Europe specifically indicated to treat this disease.”

The EC’s approval [makes] Joenja the first therapy in Europe specifically indicated to treat [APDS]”

The EC approval is based on results from a Phase II/III clinical trial, which evaluated leniolisib in 31 patients. Statistically significant improvements in immune dysregulation and immunodeficiency markers were observed, a key consequence of PI3Kδ hyperactivity. Additionally, the data included findings from the long-term, open-label extension study of 37 patients.

Professor Ulrich Baumann, MD, Managing Senior Physician at Hannover Medical School, Germany, said: “The available clinical data on leniolisib consistently demonstrated a significant improvement in key parameters of immune dysregulation in patients with APDS.

”Of particular note is its targeted approach, which directly targets the underlying pathophysiology. Against the background of previously limited therapeutic options, this approval represents an important advancement in the management of this patient population.”

Another notable development from the European Commission includes the expanded approval last month of Sanofi and Regeneron’s biologic Dupixent. According to Dr Alyssa Johnsen, PhD, Global Therapeutic Area Head, Immunology Development at Sanofi, it “provides a first-of-its kind approach to addressing chronic spontaneous urticaria in young children”.

Additionally, earlier this month, the European Parliament and Council agreed to progress the Critical Medicines Act (CMA), a move that is set to help bolster’s Europe supply chain and address shortages of critical medicines across the EU.