Senhwa Biosciences Granted Rare Pediatric Disease Designation for Rare Brain Cancer Treatment

Rare Daily Staff

The U.S. Food and Drug Administration has granted Senhwa Biosciences Rare Pediatric Disease designation for silmitasertib for the treatment of a rare pediatric brain cancer.

The rare brain cancer, recurrent sonic hedgehog medulloblastoma, is one of the four major sub-groups of medulloblastoma, the most common cancerous brain tumor in children. There are about about 80 to100 new cases per year, and treatment usually includes surgery followed by radiation or chemotherapy, or both. Currently there is no targeted drug available.

Senhwa is focused on the development of next generation DNA damage response cancer therapeutics. Silmitasertib is a first-in-class small molecule drug that targets CK2 and acts as a CK2-inhibitor. In a phase 1/2 study, silmitasertib achieved clinical benefit, resulting in stable disease and extending the duration of treatment in patients who are unresponsive to standard of care therapy. The combination of silmitasertib with DNA-damaging agents such as gemcitabine plus cisplatin has been shown to synergistically improve the efficacy of cholangiocarcinoma treatments.

Silmitasertib was safe and well-tolerated in humans. To date, three phase 1 trials of silmitasertib in cancer patients have been completed; currently, there are one ongoing phase 1 and two ongoing phase 2 studies. 

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes silmitasertib eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Sarepta sold its priority review voucher to Vifor Pharma $111 million in February 2020.

The FDA has also granted Orphan Drug designation for silmitasertib for the treatment of cholangiocarcinoma.

Editor’s note: This story was updated to correct the most recent sales of a priority review voucher.