Orphan Drug Designation Has Been Awarded to an Investigational Treatment for Burkitt Lymphoma

The US FDA has awarded Orphan Drug Designation to the experimental drug CPI-613, for the treatment of Burkitt lymphoma. The full article can be read here, at Globe Newswire.

About Burkitt Lymphoma

Burkitt lymphoma (BL) is one of a group of cancers called non-Hodgkin lymphomas. BL occurs when abnormal B-cells (also known as B lymphocytes) are produced. B-cells are a type of white blood cell and are an important part of the immune system. It is a fast-growing cancer that can affect people at any age, including in childhood. Both men and women can develop it, but it is about three times more common in men. According to Lymphoma Action, around 210 people are diagnosed with BL each year in the UK.

About CPI-613

Rafael Pharmaceuticals Inc. is developing the drug CPI-613 as a possible treatment for BL. It works by inhibiting specific enzyme targets involved in the tricarboxylic acid cycle, which is a process that produces energy for the body. The evidence for the experimental drug has so far been encouraging, and Dr Noy, principal investigator for a study into it, said,

“This could be a potentially life-saving therapy.”

Research into CPI-613

The drug is still in the fairly early stages of research. A Phase I trial of it has been carried out in patients with advanced haematological malignancies (cancers that affect the blood and lymph system). One female with relapsed BL who took part in the study was given a CPI-613 monotherapy for 51 weeks (17 cycles) and reached and maintained a partial response status after three cycles. After 17 cycles she discontinued treatment and underwent surgery, and has not shown evidence of disease for over 36 months.

A Phase II clinical study of CPI-613 is now planned for patients who have refractory or relapsed Burkitt lymphoma or leukaemia and certain forms of high-grade B-cell lymphoma (see the original article for more information).


Anna Hewitt

Anna Hewitt

Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.

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