Celgene Corporation has recently announced two exciting updates for the rare disease community!
The first big announcement from Celgene was that their supplemental New Drug Application (sNDA) for an investigative therapy for marginal zone lymphoma and follicular lymphoma has received Priority Review Designation by the FDA.
The therapy is called REVLIMID (lenalidomide) and it is being investigated for use in combination with rituximab, otherwise known as R². R² is also a product created by Celgene. They received approval on their Marketing Authorization Application for R² from the EMA earlier this year. The company believes that the combined treatment regime of R² could become a chemotherapy free treatment option for lymphoma patients. Their aim is to make it available for these individuals as soon as possible.
Their sNDA for REVLIMID is possible thanks to a Phase 3 clinical trial called AUGMENT. This study examined R² in comparison to placebo plus rituximab. It was a randomized and double-blind trial. The trial’s primary endpoint was progression-free survival. It also analyzed a wide array of secondary endpoints including the time to the patient’s next anti-lymphoma therapy, event-free survival, overall response rate, complete response rate, duration of response, duration of complete response, durable complete response rate, and overall survival.
The results from AUGMENT were presented at the American Society of Hematology’s 2018 Annual Meeting and Exposition.
Celgene’s recent Priority Review Designation for REVLIMID is a huge step forward in the process of getting this new potential therapy to follicular lymphoma and marginal zone lymphoma patients.
The second major update from Celgene is that one of their Biologics License Application (BLA) anticipated submission dates has changed. This application is for luspatercept which aims to treat anemia caused by myelodysplastic syndromes or beta thalassemia. Specifically, it is for adults with these diagnoses who require blood transfusions. Celgene now plans to submit their BLA to the FDA in April of 2019.
You can read more about these updates from Celgene here.