FDA Rejects Cancer Drug, Melblez (Melphalan)
Drug treats rare eye cancer that spreads to the liver (September 13)
The FDA has issued a complete response letter (CRL) regarding the New Drug Application (NDA) for the Melblez Kit (Melblez [melphalan] for Injection for use with the Delcath Hepatic Delivery System) for the treatment of patients with unresectable ocular melanoma metastatic to the liver.
A CRL is issued by the FDA when the review of a file is completed and questions remain that preclude approval of the NDA in its current form.
The FDA comments included a statement that the drug’s developer (Delcath Systems, Inc.) must perform another “well-controlled randomized trial to establish the safety and efficacy of [the] Melblez Kit using overall survival as the primary efficacy outcome measure,” and which “demonstrates that the clinical benefits of [the] Melblez Kit outweigh its risks.”
In a clinical trial, 7% of 122 patients died due to side effects related to treatment with the kit.
About 4 months ago, an independent panel of FDA advisors unanimously recommended against approving the therapy.