RESEARCH TRIANGLE PARK – An unusual therapy under development by Fennec Pharmaceuticals is getting an expedited opportunity to protect the hearing of children undergoing chemotherapy.

The small Research Triangle Park company announced this week that the U.S. Food and Drug Administration accepted its New Drug Application and granted priority review for PEDMARK.


Cisplatin is a widely used platinum-based chemotherapy agent. But even though it can save lives, it can also cause irreversible and permanent hearing loss in children. PEDMARK is an investigational drug for the prevention of ototoxicity induced by cisplatin chemotherapy in pediatric patients with localized, non-metastatic, solid tumors.

“The FDA filing acceptance of our NDA and granting of priority review represents a significant milestone in the development of PEDMARK and we look forward to working closely with the agency during this review process,” said Rosty Raykov, chief executive officer of Fennec.

In the U.S. and Europe, it is estimated that each year, over 10,000 children diagnosed with cancerous tumors may receive platinum-based chemotherapy. Over 60 percent of pediatric patients treated with platinum-based chemotherapy suffer from high-frequency hearing loss. Currently, there is no established preventive agent for their hearing loss, and inner ear implants can be both expensive and technically difficult for children to master.

Infants and young children who suffer hearing loss lag behind their peers in speech language development and literacy. Older children and adolescents with ototoxicity can lag in social-emotional development and educational achievement.

The FDA grants priority review to applications for medicines that treat a serious condition, and, if approved, would demonstrate the potential to be a significant improvement in the safety or effectiveness of the treatment, diagnosis or prevention of a serious condition. Having granted Fennec’s PEDMARK priority review status, the FDA should complete its review within six months instead of their standard review period of 10 months.

(C) N.C. Biotech Center