FDA Grants Orphan Drug Designation to Devimistat for Soft Tissue Sarcoma


The FDA has granted an Orphan Drug designation to CPI-613 for the treatment of soft tissue sarcoma.

The FDA has granted an Orphan Drug designation to CPI-613 (devimistat) for the treatment of soft tissue sarcoma, Rafael Pharmaceuticals announced in a press release.

In the soft tissue sarcoma treatment landscape, few treatment options exist due to the rarity of the disease. To address the unmet medical need for a new therapy in this space, Rafael Pharmaceuticals recently launched a phase 1/2 study to find the maximum tolerated dose (MTD) of CPI-613 in patients with relapsed/refractory soft tissue sarcoma and determine the efficacy and safety of the drug.

“There is a significant unmet need in treatment for soft tissue sarcoma,” said Sanjeev Luther, president and chief executive office of Rafael Pharmaceuticals, in a statement. “When a disease is rare, it often does not receive the attention and focus necessary to develop effective treatments for it,” he added.

In addition to the goal of finding the MTD of CPI-613, the open-label trial is measuring objective response rates to determine efficacy, and the dose-limiting toxicities of CPI-613 as coprimary end points of the study. The secondary end points include duration of response, progression-free survival, and overall survival.

To be eligible for enrollment, patients are required be aged 11 to 75 years with either a Karnofsky performance status of 60 or an ECOG performance status of 0 to 1. Patients are also required to have adequate bone marrow function, renal function, liver function, and neurological function as well as good laboratory results at baseline.

The study will exclude patients who have received chemotherapy within 7 days of treatment initiation, who have other curative anticancer treatment options, are hypersensitive to CPI-613 or hydroxychloroquine. Patients are also ineligible for this trial is f they have any medical or phycological condition that may interfere with study treatment, uncontrolled infection, of any life-threatening or severe malignancy complications.

CPI-613 is an experimental anti-mitochondrial agent that is currently under investigation in different tumors. Notably, Rafael Pharmaceutical recently announced progressed in the enrollment of a phase 3 clinical trial of CPI-613 in patients with acute myeloid leukemia. In addition to AML, this drug is under investigation in clinical trials of patients with pancreatic cancer, myelodysplastic syndrome, peripheral T-cell lymphoma, and Burkitt lymphoma.


FDA Grants Rafael Pharmaceuticals Orphan Drug Designation for CPI-613® (devimistat) for treatment of soft tissue sarcoma. News release. Rafael Pharmaceuticals. October 29, 2020. Accessed October 29, 2020. https://bit.ly/35G6qfC

Related Videos
Related Content