RTP’s Fennec Pharma an FDA Fast Track Breakthrough Therapy for Pediatric Hearing Loss Prevention
The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy and Fast Track designations to Fennec Pharma’s drug designed to protect the hearing of children undergoing chemotherapy.
The Research Triangle Park company’s treatment, PEDMARK, is a formulation of sodium thiosulfate (STS) used to prevent platinum-induced chemotherapy toxicity in pediatric patients. Cisplatin, a widely used platinum-based chemotherapy agent, can cause irreversible and permanent hearing loss in children.
The FDA designations speed up the process for bringing a drug to market in an effort to treat serious conditions and fill unmet medical needs. Fennec is about to file a New Drug Application (NDA) with the FDA to get clearance to bring PEDMARK to the clinic.
“We believe the recent receipt of Fast Track designation, and today, Breakthrough Therapy designation highlights the current lack of safe and effective treatments and overwhelming need to address this serious condition,” said Rosty Raykov, president and CEO of Fennec. “We look forward to the more-frequent interactions with the agency that the Fast Track designation provides as we prepare for the NDA filing.”
For Fennec, the Fast Track designation allows for closer collaboration with the review team within the Oncology Division at FDA. The designation may also result in a priority review of PEDMARK to coincide with the company’s NDA filing.
Last year, the company completed the second of two trials that paved the way for regulatory approval.
Identifying a need in pediatric cancer patients
According to Fennec, each year more than 7,000 children are diagnosed with cancerous tumors that are often treated with platinum-based chemotherapy. The good news: the survival rate for treatment of these tumors exceeds 80 percent.
While platinum-based chemotherapy treatment is effective, the incidence of high-frequency hearing loss related to ototoxicity in pediatric patients exceeds 60 percent. Hearing difficulties in children can impact language, speech and literacy, as well as social and educational development.
Two Phase 3 studies evaluated the reduction of ototoxicity and impact on survival. Preliminary results in the European randomized study, SIOPEL 6, showed that the addition of STS reduced the incidence of cisplatin-induced hearing loss. SIOPEL is the international society for pediatric oncology.
The numbers in the SIOPEL 6 study speak for themselves. Of the 94 patients who participated in the study, hearing loss occurred in 29 of 44, or about 70 percent, of participants who received Cisplatin only. However, only 19 out of 50 patients, or 38 percent, experienced hearing loss when STS was administered following Cisplatin chemotherapy.