Rare Disease Report

FDA Grants Orphan Drug Designation to Glioblastoma Multiforme and Anaplastic Astrocytoma Treatment

SEPTEMBER 05, 2017
Mathew Shanley
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to DCC-2618 for the treatment of glioblastoma multiforme (GBM) and anaplastic astrocytoma.

DCC-2618, developed by Deciphera Pharmaceuticals, is a pan-KIT and PDGFRα kinase switch control inhibitor, currently undergoing a Phase 1 clinical trial.

GBM is an aggressive brain cancer, characterized by headaches, personality changes, nausea, and stroke-like symptoms. It can be caused by genetic disorders like neurofibromatosis, or prior radiation therapy. Anaplastic astrocytoma is a malignant brain tumor developed in star-shaped brain cells – or astrocytes. Astrocytes form tissue that surrounds and protects nerve cells found within the brain and spinal cord.

Per the Central Brain Tumor Registry of the United States, 12,000 patients will be diagnosed with either of these 2 cancers annually.

“Receipt of orphan drug designation for glioblastoma multiforme and anaplastic astrocytoma marks an important milestone for the DCC-2618 development program and highlights the need for novel therapies for the treatment of these devastating brain tumors,” said Michael D. Taylor, Ph.D., Deciphera’s President and Chief Executive Officer in a press release.

“We believe that DCC-2618, which previously received orphan drug designation for the treatment of gastrointestinal stromal tumors, has the potential to serve as a much needed therapeutic option for these patients.”

The clinical trial, titled A Safety, Tolerability and PK Study of DCC-2618 in Patients with Advanced Malignancies, is intended to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and a preliminary antitumor activity of DCC-2618.

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