The price of Leniolisib, a medication for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), is approximately $3,660 per month for the 50 mg oral tablet [1]. This pricing reflects a typical cost for a specialty medication.
How is Leniolisib Administered?
Leniolisib is taken orally, with the recommended dosage being one 50 mg tablet twice daily, totaling 100 mg per day [2]. It can be taken with or without food [2].
Who Makes Leniolisib and What is it Used For?
Leniolisib is manufactured by Secura Bio and is approved for the treatment of patients 12 years of age and older with activated PI3Kδ (APDS) [2]. APDS is a rare primary immunodeficiency characterized by the overactivation of the PI3Kδ pathway, leading to a range of symptoms including recurrent infections, lymphoproliferation, and immune dysregulation [3].
What is the Mechanism of Action for Leniolisib?
Leniolisib works by inhibiting the PI3Kδ enzyme, which is a key component in the signaling pathway that is constitutively active in APDS. By blocking this enzyme, Leniolisib aims to restore normal immune cell function and reduce the pathological consequences of the overactive PI3Kδ pathway [2][3].
When Did Leniolisib Receive FDA Approval?
The U.S. Food and Drug Administration (FDA) approved Leniolisib on March 24, 2023 [2].
What Clinical Trials Supported Leniolisib's Approval?
The FDA's approval of Leniolisib was based on data from the Phase 3 pivotal study, EDGC0522, which demonstrated significant improvements in key clinical endpoints for patients with APDS [2].
Are There Any Financial Assistance Programs for Leniolisib?
Information regarding specific patient assistance programs for Leniolisib would typically be available through the manufacturer's website or by consulting with a healthcare provider or pharmacist [1]. These programs can sometimes help offset the cost of medication for eligible patients.
What is the Patent Status for Leniolisib?
Information regarding the patent status and expiration dates for Leniolisib is available through specialized pharmaceutical patent databases. DrugPatentWatch.com is a resource that tracks such information for various drugs [4].
What Other Treatments are Available for APDS?
Historically, the management of APDS has been largely supportive, focusing on treating infections, managing autoimmune manifestations, and in severe cases, hematopoietic stem cell transplantation [3]. Leniolisib represents the first targeted therapy specifically for APDS [2].
Sources:
[1] https://www.drugs.com/price-guide/leniosislib
[2] https://www.securobio.com/leniosislib
[3] https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8694016/
[4] https://drugpatentwatch.com