FDA Orphan Drug Designation for a New Neuroendocrine Tumor Treatment!

Neuroendocrine tumors are growths which form from neuroendocrine cells in various places throughout the body. They can occur in the gastrointestinal track, the pancreas, the lungs, the thymus, and other locations as well. These tumors form when healthy cells replicate too quickly. There are a few treatment options for neuroendocrine tumors, but researchers are working to develop even better therapies for patients.

One of these has just received Orphan Drug Designation by the FDA.

Orphan Drug Designation

What exactly does that mean? This designation is given to treatments which have the potential to benefit people living with rare conditions. They provide drug developers certain benefits to help the drug reach patients faster. Some of these benefits include tax credits for clinical testing of the drug or prescription user fee exemption. After the drug is approved by the FDA, the drug developers will receive marketing exclusivity of the drug for seven years.


AlphaMedixTM is the drug for neuroendocrine tumors which has just received this designation. It works to deliver alpha emitters (which are toxic to the cells) to the neuroendocrine tumors. Alpha emitters are radioactive particles and the effect they have on the tumor cells is irreversible. That means once the tumor cells die, they wouldn’t be able to form again. A phase 1 trial of the drug is currently recruiting patients to confirm the treatments safety and efficacy. The trial will be based in Houston, Texas.

Stay tuned for updates on this new drug development and for results from its first clinical trial! This drug’s approval could be life-changing for patients with neuroendocrine tumors and its orphan drug designation is an exciting first step for researchers!

You can read more about this drug, the upcoming clinical trial, and the researchers working on this development here.

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