Research & Development
Aprea Therapeutics passes US FDA Fast Track designation for APR-246 to treat MDS with TP53 mutation
17 April 2019 -

Swedish biopharmaceutical company Aprea Therapeutics reported on Tuesday the receipt of the US Food and Drug Administration (FDA) Fast Track designation for expediting the development, review and potential approval of APR-246 for the treatment of patients with MDS having a TP53 mutation.

Myelodysplastic syndromes (MDS) represents a spectrum of hematopoietic stem cell malignancies in which bone marrow fails to produce sufficient healthy blood cells. Approximately 30-40% of MDS patients progress to acute myeloid leukemia (AML) and mutation of the p53 tumour suppressor protein is thought to contribute to disease progression. Mutations in p53 are found in approximately 20% of MDS and AML patients and are associated with poor overall prognosis.

Concurrently, the company has received US FDA Orphan Drug Designation for APR-246 for MDS. The ODD provides seven-year period of market exclusivity in the US after product approval, US FDA assistance in clinical trial design, tax credits related to clinical trial expenses as well as exemption from US FDA user fees.

The company added that the p53 tumour suppressor gene is the most frequently mutated gene in human cancer, occurring in approximately 50% of all human tumours. These mutations are often associated with resistance to anti-cancer drugs and poor overall survival, representing a major unmet medical need in the treatment of cancer.

APR-246 has demonstrated pre-clinical anti-tumour activity in a wide variety of solid and hematological (blood) tumours, including MDS, AML and ovarian cancer. A Phase I/II clinical programme with APR-246 demonstrated a favorable safety profile, biological activity and clinical responses in hematological malignancies and solid tumours with mutations in the TP53 gene, concluded the company.