FDA gives thumbs-down on hearing loss drug, citing manufacturing issues, and sends Fennec stock reeling
A small, North Carolina-based biotech has run into some headwinds in its efforts to develop a compound that prevents hearing loss associated with cisplatin chemotherapy treatments.
The FDA handed down a complete response letter to Fennec Pharmaceuticals $FENC for its Pedmark drug late Monday night, citing manufacturing issues with the company’s supplier. Fennec hopes to meet with regulators sometime within the next month or two, CEO Rosty Raykov said in a call with investors Tuesday morning, in order to resolve the issue.
Raykov further emphasized during the call that there were no clinical safety or efficacy problems and an additional trial was not required.
Nonetheless, the news rattled Wall Street, as the biotech’s stock was down more than 35% in morning trading.
Per Raykov, Fennec received a Form 483 from the agency, which listed conditions or practices that are required to be resolved prior to Pedmark’s approval. When asked by investors what specifically needed to be fixed, Raykov demurred and reiterated that there were no safety issues.
Though the company’s goal is to meet with the FDA sometime soon, Raykov couldn’t provide an exact timeline due to the CRL being issued late last night, he said, and Fennec is still hashing out a plan.
Despite the issues cited Monday night, Fennec is not planning on switching manufacturers, Raykov said. Given that the same facility produces planned Pedmark batches for both the American and European markets, Fennec aims to iron out the headaches as quickly as possible. Fennec’s European application for Pedmark is still under EMA review.
There are currently no approved drugs that prevent ototoxicity, or hearing loss, in platinum-based chemotherapies. Pedmark, a formulation of sodium thiosulfate, was aiming for a label in pediatric patients older than one month to 18 years old as the hearing loss stemming from such chemotherapies is often long-lasting and permanent. Typically, patients who end up losing their hearing must opt for cochlear implants.
Pedmark received fast track designation in March 2018 following two Phase III studies in the survival and reduction of ototoxicity in five pediatric cancers that typically require intensive cisplatin treatments.
Raykov reaffirmed Tuesday that Pedmark’s main function is to “preserve hearing without reducing the effectiveness of cisplatin treatment,” and he is confident the company can appease the FDA in short order.