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