Investigational Treatment for Atypical Teratoid Rhabdoid Tumors Receives Rare Pediatric Disease Designation

According to a story from Markets Insider, the drug company Kazia Therapeutics Limited recently announced that its investigational therapy paxalisib has been awarded Rare Pediatric Disease designation from the US Food and Drug Administration (FDA) for the treatment of atypical teratoid rhabdoid tumor (AT/RT). This is a very aggressive and rare brain cancer of childhood. The therapy was also given Orphan Drug designation for this indication earlier in the year.

About Atypical Teratoid Rhabdoid Tumor

Atypical teratoid rhabdoid tumor (AT/RT) is a very rare tumor that is most often diagnosed in children. Though it can potentially appear in any area of the central nervous system, it most commonly originates in the brain. Only around 30 cases are diagnosed annually in the US. This cancerous tumor can be highly aggressive, and accurate diagnosis combined with prompt and appropriate treatment is vital. The cause of AT/RT is not well understood, but many of these tumors share genetic similarity, such as chromosome 22 deletion. Symptoms vary depending in tumor location, but many appear in the posterior fossa, causing ataxia, headache, vomiting, and lethargy. Treatment often involves a combination of surgery, chemotherapy, and radiation therapy, with chromatin remodeling agents also under evaluation. There is no established standard approach for treating AT/RT. Unfortunately, prognosis for this cancer is poor, but survival rates have been improving in recent years. To learn more about this disease, click here.

About Rare Pediatric Disease Designation

Rare Pediatric Disease designation is given to therapies that are in development to treat life-threatening, serious rare diseases that primarily affect people under 18 years old. A rare disease is defined as any illness that affects fewer than 200,000 people in the US. The recipient company of this designation is eligible for a pediatric Priority Review voucher if the drug ultimately gains FDA approval. These vouchers can be activated for a six month drug review, are tradeable, and can also be sold to other companies.

The announcement follows the recent presentation of encouraging preclinical data. Paxalisib is currently under evaluation in ongoing phase I and phase II clinical trials. 

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