Fennec is looking for a second chance to approve PEDMARK, its pediatric chemotherapy-induced hearing loss prevention drug, as it resubmits its NDA for the agent to the U.S. FDA.
Fennec taking one more chance on PEDMARK approval. (Sarah Silbiger/Getty Images)
Fennec Pharmaceuticals is looking for a second chance to approve its pediatric chemotherapy-induced hearing loss prevention drug, PEDMARK, as it resubmits its New Drug Application (NDA) for the agent to the U.S. Food and Drug Administration.
Platinum-based therapies are essential components in treating several pediatric malignancies; however, these agents can cause “ototoxicity” or permanent hearing loss. Each year, over 10,000 children with solid tumors in the U.S. and Europe receive platinum-based chemotherapy. Those who develop ototoxicity will require lifelong hearing aids. The risk and incidence of this hearing loss depend primarily on the dose and duration of the therapy.
The drug, which consists of a unique formulation of sodium thiosulfate, has explicitly been investigated for the prevention of ototoxicity caused by cisplatin chemotherapy in children adolescents under 18 years of age waste, nonmetastatic solid tumors.
Currently, no established preventative agent exists for chemotherapy-induced ototoxicity in children. PEDMARK is a potential option that has undergone investigation in two Phase III clinical trials, including The Clinical Oncology Group Protocol ACCL0431 and SIOPEL 6. Findings from the SIOPEL 6 trial, announced in 2017, showed that the treatment with PEDMARK led to a significant reduction in cisplatin-induced hearing loss in patients with standard-risk hepatoblastoma.
“I am absolutely delighted that after 30 years of research we have found a safe way to reduce ototoxicity in children receiving platinum containing chemotherapy,” according to a statement made by Penelope Brock, M.D., PhD, International Chair of SIOPEL. “This means that children who are cured from cancer after receiving platinum treatment can look forward to a normal healthy life, fully integrated into society. I believe this marks a new standard of care in pediatric oncology.”
The European Medicines Agency is currently evaluating the therapy’s Marketing Authorization Application. And in March 2018, the FDA granted PEDMARK Breakthrough Therapy and Fast Track Designation.
Back in September 2020, Fennec announced it had received a U.S. patent for PEDMARK, making it eligible for listing in the FDA’s Orange Book. The patent came a long way from the therapy’s Orphan Drug Designation, granted by the FDA in 2004.
Resubmission of the NDA for PEDMARK follows Fennec’s receipt of final minutes from a Type A meeting with the FDA. In August 2020, Fennec received a Complete Response Letter (CRL) which outlined deficiencies associated with the facility that manufactured the drug product. Despite these deficiencies, the FDA found no issues with clinical safety or efficacy of the drug and required no further clinical data to move forward.
In a statement made earlier this month by Fennec’s Chief Executive Officer, Rosty Raykov, the company is planning for the potential approval and commercial launch of PEDMARK, pending a positive response from the FDA period.
In its first quarter 2021 update, the company said that it had $26.7 million in cash and cash equivalents as of March 31, 2021, with no outstanding debt. Just a couple of months before receiving the CRL, however, Fennec’s existing cash totaled approximately $38.7 million.
Fennec has given a great deal of attention to PEDMARK, stating in the business update that its decrease in cash and cash equivalents between December 2020 and March 2021 has been a result of costs related to the NDA’s development and preparation, in addition to general administrative expenses.
