US FDA Declares New Treatment for Neuroblastoma an Orphan Drug

Biopharmaceutical company CorMEdix Inc. announced on Friday, February 23rd that its product taurolidine received orphan drug status from the United States Food and Drug Administration (US FDA). Taurolidine exists currently in a preclinical state and is part of developments to treat neuroblastoma. Keep reading to learn more, or follow along at the original source here.

CorMedix Inc. focuses primarily on the development and commercialization of therapeutic products. The company intends these products to treat a variety of diseases, including infectious ones. Researchers at CorMedix study taurolidine as part of the company’s foremost product Neutrolin®.

Neutrolin® acts as a new anti-infective treatment that reduces, and to some degree, prevents catheter-related infections and thrombosis. To this end, Neutrolin® normally helps patients who require central venous catheters as a result of end-stage renal disease.

CorMEdix’s CEO, Khoso Baluch says the company is pleased to expand the potential of its products. “We believe taurolidine could have potential activity in this rare pediatric disease area, based on onging laboratory studies,” he said in response to the FDA orphan drug designation.

“Current treatments offer poor outcomes and based on its potential synergy with other drugs approved in this area, we believe taurolidine could become an important therapy for neuroblastoma.”

Baluch concluded by expressing a desire to partner and collaborate with other companies who focus more on cancer treatment. In this way CorMedix hopes to proceed to clinical development and eventually marketing approval.

The US FDA grants orphan drug status to new treatments targeting rare disease and conditions. The US FDA classifies rare disease as one which affects fewer than 200,000 patients in the US.

The orphan drug status represents several possible benefits for CorMedix. It opens up possibilities for grant funding, assistance designing clinical trials, tax credits towards research, and waives certain filing fees. The orphan drug status also means that upon approval, CorMedix may possess a seven-year marketing exclusivity.

To accomplish further research, CorMedix contracted The Pediatric Oncology Experimental Therapeutics Investigator’s Consortium (POETIC). POETIC’s contract details experiments regarding cell lines and animal models for taurolidine treatment. Development of initial therapies have already begun. Thereapies under development include formulas for delivery to tumors, and others involving taurolidine in combination with chemotherapy. Various cell-lines confirm the usefulness of these treatments in reducing cancer cells. Animal studies are ongoing.


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