For Leniolisib, Pharming submits a Marketing Authorization Application to the European Medicines Agency.

European Medicines Agency

Pharming submits a Marketing Authorization Application to the European Medicines Agency

An oral, selective phosphoinositide 3-kinase delta (PI3K) inhibitor called leniolisib has been submitted by Pharming Group to the European Medicines Agency (EMA) as a treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, in adults and children 12 years of age and older.

On August 1, 2022, Pharming reported that the EMA’s Committee for Medicinal Products for Human Use has awarded the leniolisib MAA rapid review (CHMP). The review period is shortened by the accelerated evaluation from 210 days to 150 days. The EMA will expedite the review of an MAA upon request if they determine that the product is highly relevant to public health, particularly in terms of therapeutic innovation. In the EEA, leniolisib’s marketing authorization is anticipated in H1 2023.

“This MAA submission, under an accelerated regulatory pathway, is an important step towards approval of our second product in the EEA and highlights Pharming’s ongoing commitment to advancing leniolisib as a treatment for patients with APDS. There is a significant unmet need for therapies to improve outcomes for these patients, which, if left untreated, can result in permanent lung damage and lymphoma. Leniolisib has the potential to be the first approved treatment for this rare and orphan-designated disease, and we look forward to continuing our work with key stakeholders to bring this new product to patients.”

Anurag Relan, Chief Medical Officer of Pharming

Positive results from a Phase II/III research of leniolisib, which were released on February 2, 2022, support the MAA. This study showed that leniolisib accomplished its co-primary goals of reducing lymph node size and correcting immunodeficiency in the target group. Furthermore, the study’s safety data revealed that subjects tolerated leniolisib well. Data from a lengthy, open-label extension clinical trial including leniolisib treatment in APDS patients were also included in the MAA.

Activated Phosphoinositide 3-Kinase δ Syndrome (APDS) :

A primary immunodeficiency that is uncommon (affecting 1–2 individuals per million), APDS. Variants in either of the two genes, PIK3CD or PIK3R1, which control the development of white blood cells, are the cause. Hyperactivity of the PI3K (phosphoinositide 3-kinase delta) pathway is caused by variants of these genes. For the immune system to operate normally, the PI3K pathway needs to be signalled in a balanced manner. Immune cell maturation and function are compromised when this system is overactive, which causes immunodeficiency and dysregulation.

Severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy are symptoms of APDS. People with APDS experience a median 7-year diagnostic delay due to the symptoms’ wide range of possible causes, including various primary immunodeficiencies.

This delay could result in an accumulation of harm over time, including lymphoma and chronic lung damage because APDS is a progressive disease. Genetic testing is the only reliable way to diagnose this illness.

Leniolisib :

Leniolisib is a small-molecule inhibitor of the 110 kDa catalytic subunit of class IA PI3K with potential anti-neoplastic and immunomodulatory properties. Phosphatidylinositol-3,4,5-trisphosphate synthesis is inhibited by leniolisib (PIP3). PIP3 controls a wide range of cell processes including proliferation, differentiation, cytokine generation, cell survival, angiogenesis, and metabolism. It also acts as a key cellular messenger by activating AKT (through PDK1).

PI3K and PI3K are predominantly expressed in cells of hematopoietic origin, in contrast to PI3K and PI3K, which are ubiquitously expressed. Strong evidence suggests that PI3K is a valid and potentially effective therapeutic target for a number of immune diseases due to its central role in regulating numerous cellular functions of the innate immune system (neutrophils, mast cells, and macrophages) as well as the adaptive immune system (B-cells and, to a lesser extent, T cells). Leniolisib has so far been well tolerated during both the Phase II/III registration-enabling study and the Phase 1 first-in-human trial in healthy volunteers.

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