Aprea Therapeutics said FDA has granted Fast Track designation to APR-246 for the treatment of patients with MDS having a TP53 mutation. In addition, FDA has also granted Orphan Drug Designation to APR-246 for treatment of MDS.
The FDA’s Fast Track program facilitates the development of drugs intended to treat serious conditions and that have the potential to address unmet medical needs. A drug program with Fast Track status is afforded greater access to the FDA for the purpose of expediting the drug’s development, review and potential approval.
In addition, the Fast Track program allows for eligibility for accelerated approval and priority review, if relevant criteria are met, as well as for Rolling Review, which means that a drug company can submit completed sections of its New Drug Application for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be submitted for review.
The p53 tumor suppressor gene is the most frequently mutated gene in human cancer, occurring in approximately 50% of all human tumors. 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 been shown to reactivate mutant and inactivated p53 protein—by restoring wild-type p53 conformation and function—and thereby induce programmed cell death in human cancer cells. APR-246 has demonstrated pre-clinical anti-tumor activity in a wide variety of solid and hematological tumors, including MDS, AML, and ovarian cancer, among others.
Additionally, strong synergy has been seen with both traditional anti-cancer agents, such as chemotherapy, as well as newer mechanism-based anti-cancer drugs and immuno-oncology checkpoint inhibitors. In addition to pre-clinical testing, a Phase I/II clinical program with APR-246 has been completed, demonstrating a favorable safety profile, biological activity and clinical responses in hematological malignancies and solid tumors with mutations in the TP53 gene.
