According to a story from BioSpace, the cancer drug company Gibson Oncology, LLC has recently announced that the US Food and Drug Administration (FDA) has awarded Rare Pediatric Disease designation to the company’s investigational therapy LMP-400. The designation was given to the drug for treating Ewing’s sarcoma, a rare cancer that affects children. Gibson is committed to the development of innovative, cost-effective therapies for cancer.
About Ewing’s Sarcoma
Ewing’s sarcoma is a type of cancerous small, round, blue cell tumor. It is cancer which can appear in soft tissue or bone. This cause of this rare cancer is due to genetic exchange between chromosomes 11 and 22 in 85 percent of cases. This exchange causes the EWS gene found on 22 to fuse with the FLI1 gene found on 11. It most commonly occurs in people between age 10 and 20. It more commonly affects males. Symptoms of Ewing’s sarcoma include pain at the site of the tumor, inflammation, increased red blood cell sedimentation, anemia, fever, swelling, and elevated levels of white blood cells. Treatment includes surgery, chemotherapy, and radiation. This cancer should be treated aggressively because metastatic disease is difficult to detect. The five year survival rate for this disease is around 70 to 80 percent. About 16 percent of primary bone cancer cases are Ewing’s sarcoma. To learn more about Ewing’s sarcoma, click here.
About Rare Pediatric Disease Designation
Rare Pediatric Disease designation is granted to therapies that are in development for the treatment of serious, life-threatening rare diseases that affect people age 18 or younger. A rare disease is defined as any disease that affects less than 200,000 people in the US. When this designation is granted the recipient is eligible to receive a Priority Review voucher which is can choose to use on a future therapy in its development pipeline.
LMP-400 has completed Phase I clinical trials and has demonstrated anti-cancer activity and good safety characteristics compared to similar therapies. LMP-400 is effective against tumors that express high levels of Schlafen11. As Ewing’s sarcoma tumors often so express Schlafen11, LMP-400 should be a highly effective therapy for this disease.
Gibson is currently developing plans for Phase 2 trials of LMP-400 for patients with Ewing’s sarcoma.