The FDA Has Granted Rare Pediatric Disease Designation to an Investigational Drug For the Treatment of Ewing’s Sarcoma

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An investigational drug called CLR 131 has received Rare Pediatric Disease Designation from the US FDA for the treatment of Ewing’s sarcoma. The source press release can be found here, at Cellectar Biosciences’s website.

About Ewing’s Sarcoma

According to the NHS, Ewing’s sarcoma (or Ewing sarcoma) is a rare form of cancer that mainly occurs in people between the ages of ten and twenty. It affects the bones and/or tissue around the bones, often in the legs, arms, pelvis, spine, or ribs. People who are affected by Ewing’s sarcoma may develop symptoms such as pain in the bones, fragile bones, a persistent fever, tiredness, weight loss, and a tender lump or swollen area.

About CLR 131

CLR 131 is an investigational drug that a spokesperson for Cellectar says, “has shown early promise.” According to Cellctar, CLR 131 is a radioiodinated PCD therapy that is designed to selectively deliver radiation to tumour cells, while minimising the effects of this on healthy tissue.

About Rare Pediatric Disease Designation (RPDD)

The US Food and Drug Administration’s Rare Paediatric Disease Designation is intended to encourage the development of new drugs that could benefit patients with rare paediatric diseases. The FDA defines ‘rare’ as conditions that affect 200,000 people or less in the United States, and the FDA, in this case, considers a condition ‘paediatric’ if more than half of the people affected by it in the US are between the ages of 0 and 18. You can find out more about the RPDD from the FDA by clicking here.

The US FDA’s decision to award the investigational drug CLR 131 RPDD follows previous decisions to give the same drug RPDDs for other indications, including neuroblastoma and rhabdomyosarcoma.

Future Research Plans

There are plans to investigate CLR 131 in a Phase 1 study of paediatric patients diagnosed with Ewing’s sarcoma and several other forms of cancer. For more information about this planned trial, you can click here to visit the government clinical trials website. The study uses the identifier number NCT03478462.


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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