SELLAS Granted FDA Rare Paediatric Disease Designation for Acute Myeloid Leukemia

SELLAS Granted FDA Rare Paediatric Disease Designation for Acute Myeloid Leukemia

By, Admin 17 October 2024

Overview

SELLAS Life Sciences Group has received Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA). 

Paediatric Acute Myeloid leukaemia

  • Paediatric acute myeloid leukaemia (AML) is a rare but aggressive cancer affecting children's blood and bone marrow. 
  • It disrupts normal blood cell production, leading to uncontrolled growth of abnormal myeloid cells. 
  • Treatment typically involves chemotherapy, but outcomes remain poor, particularly for those who relapse or don’t respond to therapy.

GPS targets Wilms Tumor-1 (WT1) and is being developed for the treatment of pediatric acute myeloid leukemia (AML).

Need for Novel Oportunity

The designation highlights the need for new treatment options for pediatric AML, which continues to have a poor prognosis, especially in cases of relapse or resistance to current therapies. 

Relapse & Prevalence

  • Around 50% of children with AML experience relapse, and for those whose remission lasts less than 12 months, the five-year survival rate is only 15.7%. 
  • For patients who do not achieve complete remission after the first course of chemotherapy, the survival rate drops to 0%. 

Current Treatment Options

Currently, bone marrow transplants remain the only curative option for relapsed or refractory cases, with chemotherapy primarily used to induce remission before transplantation.

Previous Trial Outcomes

  • In earlier trials with adult AML patients, GPS demonstrated a median overall survival (OS) of 67.6 months and an ability to stimulate immune responses, specifically through T-lymphocytes. 
  • Younger patients, in particular, showed better outcomes, with many remaining leukemia-free for over five years. The safety profile of GPS was favourable across all age groups.

The Granted Approvals

  • The RPDD granted by the FDA is aimed at rare and serious diseases affecting fewer than 200,000 individuals under the age of 18 in the United States. 
  • Should GPS eventually receive FDA approval for pediatric AML, SELLAS could be eligible for a Priority Review Voucher (PRV), which can expedite the review of a future drug application.