European Medicines Agency Grants Orphan Drug Designation to Rafael Pharmaceuticals’ CPI-613® (Devimistat) for Treatment of Patients with Relapsed or Refractory Burkitt's Lymphoma

Rhea-AI Summary

Rafael Pharmaceuticals announced the European Medicines Agency (EMA) has granted orphan drug designation to CPI-613® (devimistat) for treating Burkitt's lymphoma, a rare cancer affecting mainly children. This designation is the third for devimistat by the EMA, complementing seven orphan drug designations by the FDA in the U.S. Burkitt's lymphoma represents about 1% of adult lymphoma cases, with few treatment options available. The company aims to address the urgent need for effective therapies for patients with relapsed or refractory cases.

Positive

• EMA granted orphan drug designation for CPI-613® (devimistat) to treat Burkitt's lymphoma.
• Devimistat has multiple orphan drug designations, enhancing its market potential.
• Urgent need for treatment options for relapsed or refractory Burkitt's lymphoma patients.

Negative

• Burkitt's lymphoma accounts for only 1% of adult lymphoma cases, limiting market size.
• Current treatment landscape remains sparse beyond first-line therapy.

CRANBURY, N.J., Nov. 03, 2021 (GLOBE NEWSWIRE) -- 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^® (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.

About CPI-613^® (devimistat)
CPI-613^® (devimistat) is a first-in-class clinical lead compound of Rafael, which targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. Devimistat is designed to target the mitochondrial tricarboxylic acid (TCA) cycle, a process essential to tumor cell multiplication and survival, selectively in cancer cells. Devimistat substantially increases cellular stress and the sensitivity of cancer cells to a diverse range of chemotherapeutic agents. This synergy allows for potential combinations of devimistat with lower doses of these generally toxic drugs to be more effective with lower patient side effects. Combination with devimistat represents a diverse range of opportunities to substantially improve patients’ benefit in many different cancers. The U.S. Food and Drug Administration (FDA) has designated devimistat as an orphan drug for the treatment of pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, peripheral T-cell lymphoma, soft tissue sarcoma, Burkitt’s lymphoma and biliary tract cancer. The European Medicines Agency (EMA) has granted orphan drug designation to devimistat for pancreatic cancer, acute myeloid leukemia and Burkitt’s lymphoma.

About Rafael Pharmaceuticals, Inc.
Rafael Pharmaceuticals is focused on the growing field of cancer metabolism. The company is developing a new, first-in-class category of metabolic oncology therapeutic candidates that are designed to attack hard-to-treat cancers by targeting the metabolic processes that these cancers need to survive, grow and proliferate. Rafael Pharmaceuticals’ lead compound, CPI-613^® (devimistat), is an investigational anti-cancer agent that is being evaluated in ongoing and completed Phase 1, 2 and 3 clinical trials. The Company's investors include Rafael Holdings, Inc. (NYSE: RFL). On June 21, 2021, we announced that we have entered into a merger agreement with Rafael Holdings, Inc. to create a publicly traded late-stage clinical oncology company focused on cancer metabolism-based therapeutics. For more information, please visit www.rafaelpharma.com.

Safe Harbor Statement
This press release contains forward-looking statements. These statements relate to future events or the company’s future financial performance. In some cases, you can identify forward-looking statements by terminology such as "may", "will", "should", "expect", "plan", "anticipate", "believe", "estimate", "predict", "potential" or "continue", the negative of such terms, or other comparable terminology. These statements are only predictions. Actual events or results may differ materially from those in the forward-looking statements as a result of various important factors. Although we believe that the expectations reflected in the forward-looking statements are reasonable, such statements should not be regarded as a representation by the company, or any other person, that such forward-looking statements will be achieved. The business and operations of the company are subject to substantial risks which increase the uncertainty inherent in forward-looking statements. We undertake no duty to update any of the forward-looking statements, whether as a result of new information, future events or otherwise.

In light of the foregoing, readers are cautioned not to place undue reliance on such forward-looking statements.

FAQ

What is the significance of the orphan drug designation for RFL?

The orphan drug designation for CPI-613® (devimistat) enhances its potential for development and market exclusivity in treating Burkitt's lymphoma.

How does CPI-613® (devimistat) work in treating Burkitt's lymphoma?

CPI-613® targets cancer cell metabolism, specifically the mitochondrial tricarboxylic acid cycle, potentially increasing cancer cell sensitivity to chemotherapy.

What are the other indications for which devimistat has received orphan drug designation?

Devimistat has received orphan drug designation for pancreatic cancer, acute myeloid leukemia, and several other cancers.

Why is Burkitt's lymphoma a critical focus for Rafael Pharmaceuticals?

Burkitt's lymphoma is a rare and aggressive cancer with limited treatment options, creating a need for effective therapies like CPI-613®.

When was the orphan drug designation for CPI-613® granted?

The orphan drug designation for CPI-613® was announced on November 3, 2021.