FDA grants Fast Track and Orphan Drug Designation to Aprea Therapeutics’ APR-246 Drug for the Treatment of MDS

The U.S. Food and Drug Administration has granted a Fast Track designation to Aprea Therapeutics’ APR-246 for treating patients with MDS TP53 mutation. Aprea is a Stockholm, Sweden and Boston, Massachusetts based privately held biopharmaceutical company which develops novel anticancer drugs targeting p53 tumor suppressor protein.


Additionally, FDA has also given APR-246 an Orphan Drug Designation for treatment of MDS. The Fast Track program of FDA is intended for the development of drugs for treating serious conditions and to address potential unrealized medical needs. A drug with a Fast Track Status is given preferential access to FDA for faster expediting the development, approval and review of the drug.


The Fast Track review process also includes Priority Review, Accelerated Approval and Rolling Review meaning that a drug company can submit selected sections of the NDA (New Drug Application) for review by the FDA instead of waiting for the entire section of NDA to be completed before submitting a complete application.


Getting an Orphan Drug Designation can also provide many commercial and development related incentives for designated medicines and compounds. It also provides a market exclusivity period of 7 years in the U.S after the drugs approval, tax credits and FDA assistance in clinical trials.


The tumor suppressor gene p53 is the most mutated gene in human cancer with an occurrence rate of 50% in all human tumors. These mutations are associated with overall poor survival and resistance to anti-cancer drugs.


APR-246 reactivates the inactivated and mutant p53 protein by restoring the p53 wild-type function and conformation thereby inducing programmed cell death in cancer cells. Anti-tumor pre-clinical activity has been demonstrated by APR-246 in a wide range of hematological and solid tumors including ovarian cancer, AML, MDS and others.  A strong synergy has been found with traditional cancer therapies such as Chemotherapy and also new immuno-oncology checkpoint inhibitors and mechanism-based anti-cancer drugs. A Phase I/II pre-clinical testing program with APR-246 has been successfully completed which demonstrated a favorable clinical and biological activity and safety profile in solid tumors and hematological malignancies with TP53 gene mutations.


MDS or Myelodysplastic syndromes represents a range of hematopoietic stem cell malignancies where the human bone marrow stops producing sufficient quantities of healthy blood cells. 30-40% of patients with MDS progress to develop acute myeloid leukemia or AML. The tumor suppressor protein p53 is thought to be the chief contributor for disease progression. 20% of AML and MDS patients have been found to have mutations in p53 gene leading to an overall poor prognosis.

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About the Author: Shambhu Nath Jha

Mr. Shambhu Nath Jha is a seasoned consultant and researcher. His research papers have been published in reputed websites and trade journals. His interests include philosophy and history.

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