The Food and Drug Administration on Tuesday approved the first drug to prevent hearing loss in pediatric cancer patients caused by an often-used chemotherapy.
Fennec Pharmaceuticals’ Pedmark was cleared for use children at least 1 month old alongside cisplatin-based chemotherapy, a common treatment for pediatric liver, brain and bone tumors. Children who receive chemotherapy have a high survival rate, but treatment can cause permanent hearing loss in 22% to 70% of them, studies have shown.
An estimated 10,000 children take the chemotherapy drugs in the U.S. and Europe annually, often at a critical time for speech development, Fennec said in a statement. The only available options are cochlear implants and hearing aids.
In two Phase 3 trials, Fennec’s drug reduced the onset of hearing loss in 21% and 33% of patients taking chemotherapy. By comparison, 73% and 63% of placebo recipients in the two studies, respectively, lost hearing.
The approval ends a lengthy drug development journey. Fennec, formerly known as Adherex Technologies, has been working Pedmark for more than two decades. It received a U.S. orphan drug designation from the FDA — which brings additional market exclusivity — in 2004, and, 14 years later, a fast-track designation that speeds up drug reviews.
The company finally brought the drug to regulators shortly thereafter. The FDA twice rejected the drug’s application because of deficiencies at the company’s manufacturing facility in 2020 and 2021, however. Fennec’s chief operating officer resigned in January, two months before the company resubmitted its application.
The drug’s approval “addresses an enormous unmet need and for many children and young adults,” said study investigator David Freyer, the director of a survivorship and supportive care program at the Children’s Hospital Los Angeles’s Cancer and Blood Disease Institute, in a statement.
Pedmark is currently under a regulatory review in Europe.