On August 13, 2018 Cellectar Biosciences (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to CLR 131, the company’s lead Phospholipid Drug Conjugate (PDC) product candidate, for the treatment of Ewing’s sarcoma, a rare pediatric cancer (Press release, Cellectar Biosciences, AUG 13, 2018, View Source [SID1234528817]).

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"We are delighted to announce receipt of our third RPDD from the FDA, which underscores Cellectar’s commitment to rare pediatric cancers. There is a critical need to develop new therapies to fight deadly childhood cancers such as Ewing’s sarcoma, and CLR 131 has shown early promise in this arena," said John Friend, M.D., chief medical officer of Cellectar Biosciences. "This designation, combined with our receipt of FDA Orphan Drug Designation for Ewing’s sarcoma last month, will help support our efforts to optimize the drug development path in this indication and, if successful, enable this new therapeutic candidate is made available to patients as rapidly as possible."

Since March 2018 the FDA has granted RPDDs to CLR 131 for the treatment of three separate rare disease indications including neuroblastoma, rhabdomyosarcoma and now Ewing’s sarcoma. Should CLR 131 be approved by the FDA in any of these indications, the RPDD may enable Cellectar to receive a priority review voucher. Priority review vouchers can be used by the sponsor to receive priority review designation for a future NDA or BLA submission, which could reduce the FDA review time from twelve months to eight months. Currently, these vouchers can also be transferred or sold to another entity. Since the beginning of 2017, six priority review vouchers were sold for between $80 million and $150 million each.

The FDA grants RPDD for diseases that primarily affect children from birth to age 18, and affect fewer than 200,000 persons in the U.S. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

Cellectar plans to evaluate CLR 131 in a Phase 1 clinical study for the treatment of pediatric patients with Ewing’s sarcoma, rhabdomyosarcoma, osteosarcoma, neuroblastoma, high-grade glioma and lymphomas. Cellectar has received clearance from the FDA for an accelerated Phase 1 trial designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of CLR 131 in pediatric patients with these cancer types. Further details about the trial can be found at clinicaltrials.gov using the identifier number NCT03478462.

About Ewing’s Sarcoma

Ewing’s sarcoma is the second most common bone malignancy among children and adolescents. According to a study published in the Journal of Hematology/Oncology, the incidence is about 3 cases per 1 million per year in children younger than age 20. Despite the favorable prognosis, an American Cancer Society study showed that approximately 30-40% of patients develop metastases or local recurrence, and the long-term survival rate for refractory or recurrent disease is only 22-24%. The relapsed and refractory statistics underscore the need for new treatment options.

About CLR 131

CLR 131 is Cellectar’s investigational radioiodinated PDC therapy that exploits the tumor-targeting properties of the company’s proprietary phospholipid ether (PLE) and PLE analogs to selectively deliver radiation to malignant tumor cells, thus minimizing radiation exposure to normal tissues. CLR 131, is in a Phase 2 clinical study in relapsed or refractory (R/R) MM and a range of B-cell malignancies and a Phase 1 clinical study in patients with (R/R) MM exploring fractionated dosing. The company is currently initiating a Phase 1 study with CLR 131 in pediatric solid tumors and lymphoma and is planning a second Phase 1 study in combination with external beam radiation for head and neck cancer.