With one drug in its pipeline, 26-year-old Fennec Pharmaceuticals of North Carolina has had a lot riding on its treatment for chemotherapy-induced hearing loss.
Finally, 18 years after it was designated as an orphan drug and following two complete response letters, the FDA has approved Pedmark. With news of the green light, Fennec’s share price jumped by 16%.
Pedmark becomes the first therapy to prevent cisplatin ototoxicity, the ear poisoning that can affect those who undergo platinum-based chemo.
The intravenous sodium thiosulfate drug is for pediatric patients between 1 month and 18 years old. Roughly 10,000 kids with solid-tumor cancers in the U.S. and Europe in this age group are treated annually with cisplatin chemotherapy.
Advances in chemotherapy treatments for children with localized, solid tumors have improved, producing an 85% five-year survival rate, Fennec said. But permanent hearing loss is seen in approximately 60% of children treated with platinum-based chemo.
In the absence of a preventive drug, doctors have turned to cochlear implants and hearing aids when hearing loss has been detected.
“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,” Penelope Brock, M.D., Ph.D., of Great Ormond Street Hospital in London, said in a release.
After gaining fast-track and breakthrough designations from the FDA for Pedmark in 2018 and priority review status in 2020, Fennec wrestled with manufacturing issues identified in preapproval inspections.
The first CRL came in August of 2020—for unspecified production problems—and a new approval target date was established in November of 2021. As that date approached, the FDA issued a second CRL. In April of this year, Fennec submitted its application a third time.
Meanwhile, the company said its marketing authorization application for Pedmark is under review in Europe.
The drug’s effectiveness was shown in two phase 3 studies, which compared hearing loss in patients who received platinum-based chemo with and without Pedmark. In one study, the difference was 21% (with Pedmark) versus 73% (without). The other was 33% versus 63%.
In interim data from an early-stage trial released in July, Boston-based Decibel Therapeutics showed promise with another sodium thiosulfate drug to treat hearing loss brought on by chemotherapy.