Hearing loss therapy: Fennec Pharma gets USFDA nod for PEDMARK

"The FDA approval of PEDMARK represents an important breakthrough for pediatric patients with localized, non-metastatic solid tumors at risk for cisplatin-induced hearing loss. Cisplatin is a critical, standard of care agent, used in the treatment of pediatric cancers; however, even though effective, it could be harmful to children, frequently causing permanent and irreversible bilateral hearing loss. With PEDMARK, physicians now have an approved treatment option to reduce the risk of cisplatin-induced hearing loss in pediatric patients," said Rosty Raykov, chief executive officer of Fennec Pharmaceuticals. "We would like to thank the patients, their families, physicians, investigators, employees, consultants and the entire research team at Oregon Health and Science University, who have contributed to the development of PEDMARK."

The FDA approval of PEDMARK was based upon safety and efficacy data from two pivotal open-label, randomized Phase 3 trials (SIOPEL 6 and COG ACCL0431), which compared PEDMARK plus cisplatin-based regimen to cisplatin-based regimens alone for the reduction of cisplatin-induced hearing loss in pediatric patients. In both studies, the incidence of hearing loss was consistently and significantly lower in the PEDMARK plus cisplatin arm compared with the cisplatin alone arm [21.4% vs. 73.3% (p = 0.005) and 32.7% vs. 63% (p = 0.002) with hearing loss in COG ACCLO431 and SIOPEL6, respectively].

"Historically, there have been no approved treatments for preventing cisplatin-induced hearing loss. As a physician focused in pediatric cancer for many years, and a primary investigator in the pivotal PEDMARK Phase 3 Clinical Oncology Group (COG) trial, the FDA approval of PEDMARK addresses an enormous unmet need and for many children and young adults, has the potential to greatly improve everyday activities for these patients," said >David R. Freyer, DO, MS, Primary Investigator, COG ACCL0431, and Director of the Survivorship & Supportive Care Program, Cancer and Blood Disease Institute, Children's Hospital Los Angeles.

Advances in chemotherapy-based treatment approaches for pediatric patients with localized, solid tumors have improved, resulting in an 85 percent or higher five-year survival rate for these patients. However, use of platinum-based chemotherapy, still the treatment of choice in many cases, can be toxic to the ears and cisplatin treatment frequently causes permanent and irreversible bilateral (affecting both ears) hearing loss. Permanent hearing loss can be seen in approximately 60 percent of children treated with cisplatin and can be as high as 90 percent. Until now, interventions with management strategies such as cochlear implants and hearing aids only occurred after hearing loss had been detected and these interventions do not return normal hearing.

"Hearing loss can have a profound impact on a person's life, especially in children who are critically dependent upon normal hearing for cognitive, psychosocial, and speech development," said Penelope "Peppy" R. Brock, M.D., Ph.D., of Great Ormond Street Hospitalin London and International Chair of the SIOPEL 6 trial. "Incorporating PEDMARK into current treatment strategies with the goal to preserve hearing in children and young adults without reducing the effectiveness of their cisplatin treatment – is a welcome step towards helping to improve long-term outcomes for these patients."