Pharming announces US FDA approval of Joenja® (leniolisib) as the first and only treatment indicated for APDS
APDS (activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome) is a rare and progressive primary immunodeficiency
Joenja® is a targeted treatment of APDS for adult and pediatric patients 12 years of age and older
Joenja® is expected to launch in the US in early April
"The FDA approval of Joenja® is an exciting moment for the APDS community and offers to transform the treatment pathway for patients and families affected by this rare disease. This approval means that they will, for the first time, have access to an approved treatment, which has the potential to change the standard of care for the patient population suffering from APDS."
"The approval of
"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. 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
APDS is a rare primary immunodeficiency that was first characterized in 2013 and is currently estimated to affect 1 to 2 people per million. It is caused by genetic variants in either one of two identified genes, known as PIK3CD or PIK3R1, which are vital to the normal development and function of immune cells in the body. While people with APDS may suffer from a wide variety of symptoms, the most common are frequent and severe infections of the ears, sinuses, and upper and lower respiratory tracts. Infections usually begin in infancy. People with APDS are susceptible to swollen lymph nodes or an enlarged spleen (splenomegaly), as well as autoimmunity and inflammatory symptoms. People with APDS may also be at higher risk for cancers like lymphoma.
The FDA evaluated the New Drug Application (NDA) for Joenja® under priority review and has approved the drug 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.
Results from the 12-week randomized, placebo-controlled study in 31 patients with APDS aged 12 years and older demonstrated clinical efficacy of Joenja® 70mg twice daily over placebo, and was significant in the co-primary endpoints which evaluated improvement in lymphoproliferation as measured by the reduction in lymph node size and increase in naïve B cells, reflecting the impact on immune dysregulation and normalization of immunophenotype in these patients, respectively. The adjusted mean change (95% CI) between Joenja® and placebo for lymph node size was -0.25 (–0.38,-0.12; P=0.0006; N=26) and for percentage of naïve B cells was 37.30 (24.06, 50.54; P=0.0002; N=13). The most common adverse reactions in the clinical trial (incidence >10%) were headache, sinusitis, and atopic dermatitis.
With the approval of Joenja®, as a treatment for a rare pediatric disease, the FDA granted
For more information regarding APDS, please visit AllAboutAPDS.com.
The Marketing Authorisation Application (MAA) for leniolisib with the
US Important Safety Information for Joenja® (leniolisib)
INDICATIONS AND USAGE
Joenja® (leniolisib) is a kinase inhibitor indicated for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older.
IMPORTANT SAFETY INFORMATION
Verify pregnancy status in females of reproductive potential prior to initiating treatment with Joenja®.
Joenja® may cause fetal harm when administered to a pregnant woman. Advise patients of the potential risk to a fetus and to use highly effective methods of contraception during treatment with Joenja® and for 1 week after the last dose of Joenja®.
Live, attenuated vaccinations may be less effective if administered during Joenja® treatment.
Use of Joenja® in patients with moderate to severe hepatic impairment is not recommended. There is no recommended dosage for patients weighing less than 45 kg.
The most common adverse reactions (incidence >10%) seen in clinical trials were headache, sinusitis, and atopic dermatitis.
Seven (33%) patients receiving Joenja® developed an absolute neutrophil count (ANC) between 500 and 1500 cells/microL. No patients developed an ANC <500 cells/microL and there were no reports of infection associated with neutropenia.
About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)
APDS is a rare primary immunodeficiency that was first characterized in 2013. APDS is caused by variants in either one of two identified genes known as PIK3CD or PIK3R1, which are vital to the development and function of immune cells in the body. Variants of these genes lead to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway, which causes immune cells to fail to mature and function properly, leading to immunodeficiency and dysregulation.1,2,3 APDS is characterized by a variety of symptoms, including severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.4,5 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, it has been reported that people with APDS are frequently misdiagnosed and suffer a median 7-year diagnostic delay.6 As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma.4-7 A definitive diagnosis can be made through genetic testing. APDS affects approximately 1 to 2 people per million worldwide.
About Joenja® (leniolisib)
Joenja® (leniolisib) is an oral small molecule phosphoinositide 3-kinase delta (PI3Kẟ) inhibitor approved in the US as the first and only targeted treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Joenja® inhibits the production of phosphatidylinositol-3-4-5-trisphosphate, which serves as an important cellular messenger and regulates a multitude of cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Results from a randomized, placebo-controlled Phase II/III clinical trial demonstrated clinical efficacy of Joenja® in the co-primary endpoints; demonstrating statistically significant impact on immune dysregulation and normalization of immunophenotype within these patients, and interim open label extension data has supported the safety and tolerability of long-term Joenja® administration.8 Leniolisib is currently under regulatory review by the
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al Blood. 2023 Mar 2;141(9):971-983.
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