Benzinga | Nov 3, 2021 09:22AM EDT

Rafael Holdings Says European Medicines Agency Grants Orphan Drug Designation To Co's CPI-613 For Treatment Of Patients With Relapsed Or Refractory Burkitt's Lymphoma

Rafael Pharmaceuticals, Inc. ("Rafael" or the "Company"), a company focused on the growing field of cancer metabolism-based therapeutics, today announced that the European Medicines Agency (EMA) has granted orphan drug designation to CPI-613(r) (devimistat) for the treatment of Burkitt's lymphoma.

In the UK, 2 out of 100 cases of non-Hodgkin's lymphoma (NHL) are identified as Burkitt's lymphoma, making it an extremely rare cancer. In addition, Burkitt's lymphoma accounts for only 1% of adult lymphoma, but up to 30% of childhood NHL --- creating a dire need for families who have children with this type of lymphoma if the first line of treatment fails. Presently, no substantial treatment options beyond first-line therapy exist.

"Relapsed or refractory Burkitt's lymphoma can be a devastating form of cancer that is in need of effective treatment options," said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. "Milestones such as this can offer hope for the patients and families who suffer from this rare form of lymphoma with few options to turn to."

This orphan drug designation is the third granted for devimistat by the EMA, following designations for pancreatic cancer and acute myeloid leukemia (AML). The Food and Drug Administration (FDA) has granted orphan drug designation for devimistat in seven indications in the United States, including in Burkitt's lymphoma.

"We have been investigating the use of devimistat for treatment in relapsed or refractory Burkitt's lymphoma. It is a testament to our work and the potential of the compound to have received another orphan drug designation," said Ariela Noy, M.D., medical oncologist for lymphoma and AIDS-associated cancers at Memorial Sloan Kettering Cancer Center (MSK), emeritus chair of the Lymphoma Working Group for the AIDS Malignancy Consortium, and principal investigator on Rafael's Phase 2 trial in Burkitt's lymphoma. "HIV dramatically increases the risk of Burkitt's lymphoma. Devimistat brings hope to the many patients with relapsed or refractory Burkitt's lymphoma, as well as their loved ones."

Disclosures: Dr. Noy has provided consulting services to Rafael Pharmaceuticals.