The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to NP-G2-044, an experimental treatment for pancreatic cancer that’s currently in clinical testing.
The FDA gives this designation to investigational medicines intended to treat conditions that affect fewer than 200,000 people in the U.S. The goal is to provide additional incentives to companies developing treatments for rare diseases. Among the perks of this designation, if NP-G2-044 is ultimately approved for use by the FDA, its developer, Novita Pharmaceuticals, will receive seven years of guaranteed market exclusivity, free from generic competitors.
“FDA Orphan Drug Designation for our fascin inhibitor represents an important regulatory milestone for Novita and validates the Company’s scientific and clinical approach in the fight against pancreatic cancer, as it remains one of the most lethal solid tumors with limited therapeutic progress over decades,” Stewart Campbell, CEO of Novita, said in a company press release.
How the therapy works
NP-G2-044 is a daily oral therapy designed to block the activity of fascin, a protein involved in the metastasis (spread) of cancer cells to other parts of the body. Metastasis plays a significant role in tumor aggressiveness, so blocking this protein aims to prevent metastasis and improve patient outcomes. Fascin inhibition may also help boost the activity of cancer-killing immune cells, enabling the therapy to act in synergy with immune-modulating cancer therapies, according to Novita.
“Fascin inhibition offers a novel approach with the potential to enhance anti-tumor immune activity and improve outcomes for patients with this devastating disease, and we look forward to continuing advancement of NP-G2-044 to address the high unmet need,” Campbell said.
Novita is currently running a Phase 1/2 clinical trial (NCT05023486) testing NP-G2-044 in adults with advanced or metastatic solid tumors, including pancreatic cancer. The trial is testing NP-G2-044 both as monotherapy (on its own) and in combination with anti-PD-1 therapy, a type of cancer treatment designed to boost the cancer-killing abilities of immune cells.
The study’s primary goals are to evaluate the safety of NP-G2-044 and identify an optimal dose for further testing; the experimental treatment’s anticancer effects will also be assessed. The trial is recruiting participants at several sites across the U.S.
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