EMA Reviewing Leniolisib for Treatment of APDS in Europe: Pharming Offers Update

Pharming Group N.V. is pleased to announce that the European Medicines Agency’s Committee for Human Medicinal Products has decided to review its Marketing Authorisation Application for leniolisib, an oral selective phosphoinositide 3-kinase delta inhibitor. Leniolisib is being evaluated as a potential treatment for activated phosphoinositide 3-kinase delta syndrome, a rare primary immunodeficiency, in adolescents and adults 12 years of age and older. This is a major step forward in the process of potentially providing a new treatment option for people with this debilitating condition.

Pharming has received a set of questions from the European Medicines Agency (EMA) that includes a request to submit new data from the ongoing long-term extension study after the interim analysis included in the original MAA. According to the timeline for our replies, we anticipate that the Committee for Human Medicinal Products (CHMP) will give its opinion on the leniolisib MAA in the second half of 2023.

Sijmen de Vries, CEO of Pharming commented:

We remain steadfastly committed to bringing leniolisib to those living with APDS around the world. As such, we are continuing to collaborate with the EMA through the MAA review process and are hopeful to obtain regulatory approval within the European Economic Area. Additionally, we are pleased to report that the FDA has granted leniolisib a priority review status, with a PDUFA goal date of March 29, 2023. We are optimistic and are dedicated to making leniolisib available for those who need it.

In October 2022, the European Medicines Agency (EMA) validated Pharming’s Marketing Authorization Application (MAA) for leniolisib, following the grant of accelerated assessment in August 2022. With the positive results from a Phase II/III study of leniolisib, announced on February 2, 2022, meeting its co-primary endpoints of reduced lymph node size and an increase in the percentage of naïve B cells in patients with APDS, the safety data from the study further showed that leniolisib was well-tolerated by participants. Moreover, an open-label extension clinical trial provided additional data to the original MAA, which further solidified leniolisib’s position as a promising treatment for APDS.

About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)

APDS is a rare primary immunodeficiency that affects approximately 1 to 2 people per million, causing severe recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy. Unfortunately, these symptoms are often confused with other conditions, leading to a median 7-year diagnostic delay and potential permanent lung damage and lymphoma. The only way to definitively diagnose this condition is through genetic testing, which reveals variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells and lead to hyperactivity of the PI3Kδ pathway. If left untreated, APDS can cause severe consequences, so it is important to identify and treat it as soon as possible.

About leniolisib

Leniolisib is an exciting new small-molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K, a molecule which plays an essential role in maintaining healthy immune system function. By inhibiting the production of PIP3, Leniolisib is able to effectively regulate a multitude of cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. PI3Kẟ is predominantly expressed in hematopoietic cells, making it a viable target for treating immune diseases like APDS. In clinical trials, Leniolisib has been shown to be well tolerated in both healthy subjects and APDS patients, providing a promising new treatment for this debilitating condition.

About Pharming Group N.V.

Pharming Group N.V. is a biopharmaceutical powerhouse that is changing the lives of patients suffering from rare and life-threatening diseases. Working with cutting-edge therapies and precision medicines, Pharming is leading the charge in developing innovative protein replacement therapies, small molecules, biologics, and gene therapies in early to late-stage development. With a global presence spanning North America, Europe, the Middle East, Africa, and Asia-Pacific, Pharming is committed to providing quality treatments to patients in more than 30 markets. Based in Leiden, Netherlands, Pharming’s dedicated team of professionals strive to make a difference in the lives of those in need.

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