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FDA Updates

FDA Updates in Ovarian Cancer, NHL, and MRTs

doi: 10.1097/01.COT.0000481886.72393.f6
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Fast Track Status to Ovarian Cancer Drug


The U.S. Food and Drug Administration has granted Fast Track designation to the multiple-epitope Folate Receptor Alpha Peptide Vaccine (TPIV 200) with GM-CSF adjuvant for maintenance therapy in patients with platinum-sensitive advanced ovarian cancer whose disease is stable or who have had a partial response following completion of standard of care chemotherapy.

The Fast Track designation, established under the FDA Modernization Act of 1997, is designed to facilitate frequent interactions with the FDA review team to expedite clinical development and submission of a New Drug Application for medicines with the potential to treat serious or life-threatening conditions and address unmet medical needs. The designation permits the drug developer the opportunity to submit sections of an NDA on a rolling basis as data become available, allowing the FDA to review those materials on a rolling basis as well.

The FDA had granted orphan drug designation to TPIV 200 for the same indication late last year.

A Phase II trial for TPIV 200 is being planned, according to a news release from the drug company developing the agent, TapImmune Inc.

Breakthrough Therapy Designation to KTE-C19 for Refractory, Aggressive NHL

The FDA has also recently granted Breakthrough Therapy designation to KTE-C19 for the treatment of patients with refractory diffuse large B cell lymphoma (DLBCL), primary mediastinal B cell lymphoma (PMBCL), and transformed follicular lymphoma (TFL). KTE-C19 is an investigational therapy in which a patient's T cells are genetically modified to express a chimeric antigen receptor designed to target the antigen CD19.

The Breakthrough Therapy designation, enacted as part of the FDA's 2012 Safety and Innovation Act, was created to expedite the development and review time of a potential new drug for serious or life-threatening disease where early clinical evidence suggests the drug may demonstrate substantial improvement compared with existing therapies.

A Phase II clinical trial for the drug for the treatment of patients with DLBCL, PMBCL, and TFL is currently enrolling patients. KTE-C19 is being developed by Kite Pharma, Inc.

Orphan Drug Status to Tazemetostat for MRTs

And the FDA has also granted Orphan Drug designation to tazemetostat (also known as EPZ-6438) for the treatment of patients with malignant rhabdoid tumors (MRTs). Tazemetostat is a first-in-class inhibitor of EZH2, a histone methyltransferase that is increasingly understood to play a potentially oncogenic role in a number of cancers.

The Orphan Drug designation—to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.—grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.

Tazemetostat is being investigated in an ongoing Phase II study in adults and a Phase I study in children with genetically-defined solid tumors, both of which began late last year and include patients with MRTs (tumors defined by loss of INI1 protein as measured by immunohistochemistry). The studies also include patients with INI1-negative tumors and synovial sarcoma. Interim data from the trial in adult patients with genetically defined solid tumors, including MRT, other INI1-negative tumors, and synovial sarcoma, are expected to be presented at a medical meeting sometime this year, according to a news release from the drug company developing the drug, Epizyme, Inc.

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