On March 24, 2023, the Pharming Group received approval from the U.S. Food and Drug Administration (FDA) for Joenja® (leniolisib) tablets. It is the first drug indicated for the treatment of patients who have activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS). Candidates for therapy with it must be at least 12 years old and weigh 45kg (about 100 pounds) or more. The recommended dose for Joenja is 70mg twice a day. Availability is planned for mid-April 2023 with an expected wholesale acquisition cost (WAC) of about $750 per tablet. Complete prescribing information can be found here.
At a Glance
- Brand (Generic) Name: Joenja (leniolisib)
- Manufacturer: Pharming
- Date Approved: March 24, 2023
- Indication: for the treatment of APDS for patients 12 years of age and older
- Dosage Forms Available: 70mg tablets
- Launch Date: mid-April 2023
- Estimated Annual Cost: WAC will be about $550,000 per year.
- At an estimated prevalence of one or two patients per one million in the population, fewer than 1,000 patients in the U.S. have APDS.
- It is a gradually worsening primary immune deficiency caused by deviations in PIK3CD and/or PIK3R1 genes, which help to regulate immune system activity. Immune cell production speeds up, resulting in underdeveloped cells with lessened activity.
- Patients who have APDS have repeated infections – mainly in their ears, lungs, respiratory tracts, and sinuses. Many have chronic viral infections and they are more likely to develop lymphomas.
- Hallmarks of APDS are swellings in lymph nodes and the spleen.
- By inhibiting an enzyme, phosphoinositide 3-kinase delta (PI3Kδ), Joenja slows down immune cell production, allowing cells to develop more fully.
- In the key clinical trial, patients taking Joenja had a 37.3% increase in naïve B-cells. Joenja also significantly reduced lymph node size compared to the placebo.
- The most common side effects of Joenja were atopic dermatitis, headache, and sinusitis.
- No other drugs currently are approved to treat the cause of APDS. Present treatment aims to prevent and treat infections, lessen symptoms and manage other conditions that can occur along with APDS.
- Designated as an Orphan Drug, Joenja was approved under Priority Review. It also was granted a Rare Pediatric Disease Priority Review Voucher (PRV), which a licensing agreement will allow Novartis to buy and use to expedite FDA approval for one of its future drug submissions to the FDA.