The US Food and Drug Administration (FDA) has awarded breakthrough therapy designation to Fennec Pharmaceuticals’ Pedmark formulation for cisplatin-related ototoxicity in pediatric patients with standard risk hepatoblastoma (SR-HB).

Breakthrough therapy designation is given when preliminary clinical evidence has been provided to show that a treatment effect may represent substantial improvement over available therapies for the treatment of a serious condition. The designation includes all of the fast track programme features, as well as more intensive FDA guidance on an efficient drug development programme.

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“The breakthrough therapy designation highlights the current lack of safe and effective treatments and overwhelming need to address this serious condition.”

Cisplatin and other platinum compounds are necessary chemotherapeutic components for various pediatric malignancies.

However, platinum-based therapies cause ototoxicity in many patients, and are particularly dangerous to survivors of pediatric cancer.

Two Phase lll studies have previously evaluated STS for survival and reduction of ototoxicity.

Fennec Pharmaceuticals president and CEO Rosty Raykov said: “The decision by the FDA to grant Pedmark the first breakthrough therapy designation for the prevention of cisplatin ototoxicity reflects a recognition of the promising efficacy and safety data generated from SIOPEL 6 and COG ACCL0431 studies.

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“We believe the recent receipt of fast track designation, and today, breakthrough therapy designation highlights the current lack of safe and effective treatments and overwhelming need to address this serious condition.

“This designation is another significant milestone for the advancement of Pedmark, as we work closely with the agency to expedite the NDA filing.”