Pharming’s Leniolisib gains FDA approval in ultra-rare disease

Pharming’s Leniolisib gains FDA approval in ultra-rare disease

Published: Mar 27, 2023

By Tristan Manalac

BioSpace

The FDA approved Pharming’s leniolisib, now to be marketed as Joenja, for the treatment of the ultra-rare disease-activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), the company announced Friday.

Joenja is the first drug approved in the U.S. in this indication and is scheduled to launch in early April.

Data from a Phase II/III trial supported Joenja’s New Drug Application, showing that Joenja could address immune dysregulation and normalize the immune phenotype in 31 patients with APDS 12 years and older.

Compared with placebo, Joenja led to a significantly greater reduction in lymph node size and improved naïve B cell counts by 37%, both of which indicate a “correction of the underlying immune defect,” the FDA wrote in its approval announcement.

As for safety, typical side effects associated with Joenja were headache, sinusitis and atopic dermatitis. These findings are reflected in the drug’s label, which also carries a warning for embryo-fetal toxicity. Joenja is also not advisable for patients with moderate to severe hepatic impairment or for breastfeeding women.

The FDA evaluated Joenja under priority review and had previously granted it Orphan Drug and Rare Pediatric Disease designations.

Leniolisib is also being reviewed by the European Medicines Agency, with an opinion from the Committee for Human Medicinal Products expected in the second half of 2023.

APDS is a rare primary immunodeficiency caused by mutations in either the PIK3CD or PIK3R1 genes, which are essential for immune cells’ normal development and function. Variations in these genes hyperactivate the PI3Kδ pathway, leading to the immune system’s dysregulation and deficiency.

APDS has no cure; patients rely on symptomatic treatment. Joenja’s approval will open new treatment opportunities for these patients.

This development could change the standard of care in this indication, said Eveline Wu, M.D., M.S.C.R., division chief, Pediatric Rheumatology, The University of North Carolina School of Medicine, in a statement.

Joenja is an oral small molecule inhibitor of PI3Kδ and works by inhibiting the action of the enzyme. Pharming first licensed leniolisib from Novartis in August 2019 for an upfront payment of $20 million. Per the agreement, Friday’s FDA approval will entitle Novartis and another company to a $10.5 million milestone payment.

After Joenja hits the market in April, Novartis and the other party will also be eligible for up to $190 million in sales milestones and tiered royalties of low double-digit to a high-teen double-digit percentage of net sales.

Along with the approval of Joenja, the FDA also gave Pharming a priority review voucher, which Novartis has the right to purchase by the 2019 agreement.

Source: BioSpace