2023-03-27| Trials & Approvals

Pharming’s Joenja Racks Up FDA Approval For Rare Primary Immunodeficiency

by Joy Lin
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Netherlands-based Pharming has announced the US FDA approval of Joenja (leniolisib) to treat activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in patients aged 12 and older. 

Joenja is the first treatment for APDS, a rare and progressive primary immunodeficiency, to be approved in the US. The application for Joenja was evaluated under Priority Review. 

Joenja is expected to launch in the US in early April, with shipments available in mid-April. 

Related Article: FDA Extends Approval Of Regeneron’s Evkeeza For Inherited High Cholesterol

Joenja Reduced Lymph Node Size, Boosts Naïve B Cells

APDS is caused by variants in either the PIK3CD or PIK3R1 genes, which are essential for the normal development and function of immune cells. Gene variants lead to hyperactivity of the PI3Kδ pathway, causing immune cells to fail to mature, and hence, immunodeficiency. 

The FDA approval is based on results from a Phase 2/3 placebo-controlled clinical trial evaluating Joenja in 31 ADPS patients aged 12 and older. The filing for approval also included data from a long-term extension trial in which 38 patients received Joenja for a median of two years. 

According to the Phase 2/3 study, a 70mg oral dose of Joenja, taken twice daily reduced lymph node size and increased the number of naïve B cells, meeting co-primary endpoints. The adjusted mean change between Joenja and placebo for lymph node size was -0.25, while the treatment boosted naïve B cells by 37.3%. The most common side effects in the trial, occurring in more than 10% of patients, included headache, sinusitis, and atopic dermatitis. 

With the approval of Joenja, Pharming receives a priority review voucher (PRV) from the FDA. Under a 2019 license agreement with Novartis, Novartis could potentially buy the PRV from Pharming for a small minority share of the value of the PRV. The approval also triggers a $10.5 million milestone payment to Novartis and another party. Pharming has mentioned making additional milestone payments to Novartis totaling $190 million if the drug achieves certain sales goals. Furthermore, Novartis could also receive tiered royalties in the low double-digit to high-teen double-digit percentages on net sales of Joenja. 

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