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EMA Grants Orphan Drug Designation to Devimistat for Refractory Burkitt's Lymphoma

• The European Medicines Agency (EMA) has granted orphan drug designation to Rafael Pharmaceuticals' devimistat for treating refractory or relapsed Burkitt's lymphoma. • Burkitt's lymphoma, a rare and aggressive form of non-Hodgkin's lymphoma, disproportionately affects children, representing up to 30% of pediatric NHL cases. • Devimistat targets the mitochondrial tricarboxylic acid cycle, crucial for tumor cell proliferation, and has received multiple orphan drug designations from both EMA and FDA. • A Phase 2 trial is underway to investigate devimistat's efficacy in relapsed or refractory Burkitt's lymphoma, offering hope for patients with limited treatment options.

Rafael Pharmaceuticals' lead compound, devimistat (CPI-613), has received orphan drug designation (ODD) from the European Medicines Agency (EMA) for the treatment of refractory or relapsed Burkitt's lymphoma, a rare and aggressive variant of non-Hodgkin's lymphoma (NHL). This marks the third ODD granted to devimistat by the EMA, adding to previous designations for pancreatic cancer and acute myeloid leukemia.
Burkitt's lymphoma constitutes approximately 2% of all NHL cases in the United Kingdom but accounts for up to 30% of NHL cases in pediatric patients. Current treatment options beyond first-line therapy are limited, highlighting a significant unmet medical need.

Mechanism of Action

Devimistat is designed to target the mitochondrial tricarboxylic acid cycle, a critical metabolic pathway for tumor cell multiplication and survival. By disrupting this cycle, devimistat aims to selectively inhibit cancer cell growth while sparing normal cells.

Clinical Development

Ariela Noy, a medical oncologist at Memorial Sloan Kettering Cancer Center and principal investigator of Rafael's Phase 2 trial in Burkitt's lymphoma, stated, "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."
The FDA has previously granted devimistat seven ODD indications, including one for Burkitt's lymphoma, underscoring the potential of this compound in addressing unmet needs in rare cancers. Sanjeev Luther, president and CEO of Rafael Pharmaceuticals, emphasized the significance of this milestone, stating, "Relapsed or refractory Burkitt's lymphoma can be a devastating form of cancer that is in need of effective treatment options. 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."
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Reference News

[1]
EMA Grants Orphan Drug Designation to Rafael ...
pharmtech.com · Nov 12, 2021

Rafael Pharmaceuticals' CPI-613 (devimistat) received EMA orphan drug designation for treating refractory/relapsed Burki...

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