EMA Grants Orphan Drug Designation to Rafael Pharmaceuticals’ CPI-613 (Devimistat)

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European Medicines Agency (EMA) granted Rafael’s CPI-613 (devismat) an orphan drug designation for the treatment of refractory or relapsed Burkin’s lymphoma.

Rafael Pharmaceuticals, a pharmaceuticals company focused on cancer metabolism-based therapeutics, announced that the European Medicines Agency (EMA) had granted orphan drug designation (ODD) to their lead compound, CPI-613 (devimistat), for the treatment of refractory or relapsed Burkin’s lymphoma, a rare variant of non-Hodgkin’s lymphoma (NHL).

According to a company press release, approximately two out of 100 cases of NHL in the United Kingdom are identified as Burkitt’s lymphoma. However, it disproportionately affects children with NHL, affecting up to 30% of pediatric patients. Besides first-line therapy, no substantial treatment options currently exist for this form of NHL.

“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, in the press release. “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.”

According to the release, devimistat is designed to target the mitochondrial tricarboxylic acid cycle, which is essential to tumor cell multiplication and survival. This is the third ODD granted to devimistat by EMA, following designations for pancreatic cancer and acute myeloid leukemia. Additionally, FDA has granted devimistat seven ODD indications, including one for 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, 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, in the press release. “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.”

Source: Rafael Pharmaceuticals