Orphan Drug Designation Granted to Myelo001 for Treatment of Acute Radiation Syndrome

The US Food and Drug Administration (FDA) has granted an orphan drug designation to Myelo Therapeutics GmbH’s Myelo001 for the treatment of acute radiation syndrome (ARS), a rare, often fatal set set of symptoms that accompany abnormally high exposure to ionizing radiation.

Applied orally, Myelo001 has been shown to be in effective in reducing hematopoietic symptoms caused by chemotherapy and radiation in preclinical and clinical studies. Clinical studies as well as comprehensive chronic toxicology and safety studies have reaffirmed the excellent safety profile of Myelo001.

Myelo001 has been tested in a randomized, double-blind, placebo-controlled phase 2a trial to reduce chemotherapy-induced neutropenia in patients receiving adjuvant or neoadjuvant chemotherapy for the treatment of breast cancer. Results are anticipated in the spring of 2018. Myelo001 is also being investigated as a treatment for chemotherapy-induced thrombocytopenia.

“Myelo001 was found to induce the differentiation of immature myeloid precursors,” according to Myelo Therapeutics. “The effect of myelo001 is determined by both the protection of immature granulocytic cells in their early stage after cytostatic treatment and more active maturation of neutrophils.”

ARS is characterized as an acute illness that presents after exposure to high levels of radiation. These exposures are often caused by a nuclear accident or attack. Severe health consequences, including death, can be caused by ARS. As such, currently, encouragement is coming from the US government to develop new drugs to prevent or treat ARS.

ARS therapies qualify as medical countermeasures (MCMs), which are products regulated by the FDA. MCMs are intended for use in the event of a potential public health emergency caused by a biological, chemical, or radiological/nuclear material.

It is not ethical or feasible to test MCMs developed for radiological and nuclear threats on humans since human exposure to high levels of radiation would be necessary. Consequently, approval may be granted by the FDA based on well-controlled non-clinical studies.