Pharming Obtains U.K. Approval for Joenja® – A New Hope
Pharming’s Breakthrough Approval for Joenja® in the U.K.
For Media and Investors
Indicated for adult and pediatric patients 12 years and older with activated phosphoinositide 3-kinase delta (PI3K?) syndrome (APDS)
Leiden, Netherlands: Pharming Group N.V. (EURONEXT Amsterdam: PHARM/NASDAQ: PHAR) has received marketing authorization from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) for Joenja® (leniolisib). This marks a significant advancement in the treatment landscape for activated phosphoinositide 3-kinase delta (PI3K?) syndrome, particularly for adult and pediatric patients aged 12 and older. Leniolisib is the first approved treatment in the U.K. targeted specifically at APDS, a rare and progressive primary immunodeficiency disease.
Innovative Treatment for a Critical Condition
Leniolisib is an oral, selective PI3K? inhibitor that addresses a dire need in the medical market. This approval is pivotal for individuals suffering from APDS, where traditional management included symptomatic treatments or the extreme option of hematopoietic stem cell transplantation. Sijmen de Vries, Chief Executive Officer of Pharming, expressed his enthusiasm, stating, “The MHRA approval of Joenja® is a major milestone for individuals in the U.K. affected by this debilitating condition. Our commitment is strengthened as we aim to become a leader in the global rare disease arena.”
The MHRA's decision follows the successful evaluation of the Marketing Authorisation Application (MAA) through the International Recognition Procedure (IRP), referencing the approval granted by the U.S. Food and Drug Administration (FDA) earlier this year.
Understanding Activated Phosphoinositide 3-Kinase ? Syndrome (APDS)
APDS is a rare form of primary immunodeficiency first identified in 2013. This condition arises from mutations in two key genes, PIK3CD or PIK3R1, which are essential for the development and functionality of immune cells. These genetic variants lead to the overstimulation of the PI3K? pathway, resulting in dysfunctional immune cell maturation. Symptoms of APDS include severe and recurrent respiratory infections, abnormal immune responses, and other complications like autoimmunity.
Challenges in Diagnosis and Treatment
Due to the complexity of symptoms associated with APDS, patients often experience misdiagnosis and delays in receiving appropriate care. Current estimates suggest that individuals can endure a median diagnostic delay of up to seven years, contributing to further complications such as lung damage and increased risk of lymphoma. Comprehensive genetic testing is crucial for accurate diagnosis, as APDS affects approximately 1 to 2 individuals per million globally.
The Impact of Leniolisib
Leniolisib revolutionizes the treatment approach for patients. As the first targeted treatment approved for APDS, it functions by inhibiting the production of phosphatidylinositol-3-4-5-trisphosphate, a primary cellular messenger that regulates numerous cell activities, including immune response and cell survival. A Phase III clinical trial highlighted significant improvements in immune dysregulation and deficiency in patients, with ongoing studies reinforcing the long-term safety profile of the drug.
Future Prospects
Pharming is seeking to extend the reach of leniolisib beyond the U.K., with regulatory reviews underway in regions such as the European Economic Area, Canada, and Australia. Plans are also in place to pursue approvals in nations like Japan and South Korea. Additionally, Pharming is conducting two Phase III trials specifically for children diagnosed with APDS, further emphasizing its commitment to addressing this rare disease.
About Pharming Group N.V.
Pharming Group N.V. (EURONEXT Amsterdam: PHARM/NASDAQ: PHAR) is a biopharmaceutical organization focused on transforming lives through innovative therapies for rare diseases. Operating globally, Pharming develops and commercializes protein replacement therapies and precision treatments, including biologics and small molecules. The company is headquartered in Leiden, Netherlands, and employs a dedicated workforce serving patients across more than 30 countries.
Frequently Asked Questions
What is Joenja® used for?
Joenja® (leniolisib) is approved for treating activated phosphoinositide 3-kinase delta (PI3K?) syndrome in patients aged 12 and older.
Why is Joenja® significant for APDS?
It is the first approved targeted therapy for APDS, addressing a significant unmet medical need for affected patients.
How does leniolisib work?
Leniolisib inhibits the PI3K? pathway, helping regulate immune cell functions and improving immune responses.
What are the next steps for Pharming?
Pharming plans to pursue further regulatory approvals in various countries and is conducting ongoing clinical trials.
Who can I contact for more information about Pharming?
You can reach Pharming Group’s Investor Relations at +1 (908) 705 1696 or email investor@pharming.com.
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