FDA Grants Orphan Drug Designation to CLR 131 in Rhabdomyosarcoma

The U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development recently granted Orphan Drug designation to CLR 131 for the treatment of rhabdomyosarcoma, a rare pediatric cancer.

“Rhabdomyosarcoma is the most common type of tissue sarcoma in children.  While initial response to treatment is generally favorable, there is an important need for new treatments, especially in children who experience relapse,” said John Friend, MD, Chief Medical Officer of Cellectar Biosciences

Orphan drug designation provides 7-year market exclusivity, increased engagement and assistance from the FDA, tax credits for certain research, research grants, and a waiver of the New Drug Application user fee. Rhabdomyosarcoma is recognized by the FDA as an orphan disease, usually defined as a condition that affects fewer than 200,000 people nationwide.

About Rhabdomyosarcoma

Rhabdomyosarcoma, a malignant tumor of mesenchymal origin, is the most common soft-tissue sarcoma in children, accounting for approximately 40% of childhood soft-tissue sarcomas in the United States. The incidence is about 4.5 cases per 1 million per year in children younger than 15 years. More than 50% are younger than 10 years at diagnosis. Approximately 340 new cases are diagnosed each year in North America, and the prognosis is favorable, with a 64% 5-year survival in children aged birth to 19 years. At least one-third of all patients will experience disease progression or relapse, and 95% of all failures occur within 3 years. The median progression-free survival following the first recurrence or progression is approximately 9 months.

About CLR 131

CLR 131 is an investigational radioiodinated phospholipid drug conjugate therapy that exploits the tumor-targeting properties of proprietary phospholipid ether (PLE) and PLE analogs to selectively deliver radiation to malignant tumor cells, thus minimizing radiation exposure to normal tissues. 

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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