Acute Radiation Syndrome Treatment Awarded Orphan Drug Status

Myelo Therapeutics GmbH, an Affiliate of Eckert & Ziegler Strahlen- und Medizintechnik AG, has announced that the European Medicines Agency (EMA) has granted an Orphan Drug Designation to its orally applied new chemical entity Myelo001 (Imidazolyl ethanamide pentandioic acid) for the treatment of Acute Radiation Syndrome (ARS).

This successful ruling from the EMA follows on from the 2018 ruling by the Federal Drug Administration to award an Orphan Drug Designation (ODD) to Myelo001 for the treatment of ARS. An Orphan Drug Designation is a status assigned to medicines developed for rare conditions. ODD's are grounded on proven medical plausibility of the orphan condition, and potentially significant benefits of the proposed treatment.

ARS, also known as radiation toxicity or radiation sickness, is an acute illness that presents after exposure to high levels of radiation, caused by a nuclear accident or attack. It can lead to severe health consequences, including death. The European Union (EU) and the US government, amongst other countries, are encouraging the development of new drugs to prevent or treat ARS.

Myelo001 is a new, clinical-stage adjuvant cancer therapy for the treatment of chemotherapy- and radiotherapy-induced myelosuppression. Preclinical and clinical studies have shown that Myelo001 applied orally is effective in reducing hematopoietic symptoms caused by chemotherapy and radiation. Comprehensive chronic toxicology and safety studies, as well as clinical studies, have confirmed an excellent safety profile of Myelo001.

The EU orphan designation program provides incentives to companies that are developing therapies for diseases which affect fewer than 5 in 10,000 people within the territory of the EU. The benefits of achieving Orphan Drug Designation include scientific advice by EMA on study protocols, regulatory fee reductions and waivers, as well as access to EU grants for drug development. Upon marketing approval, Myelo001 will benefit from 10 years of market exclusivity for the ARS indication within the EU’s territory.

Therapies for ARS qualify as medical countermeasures (MCMs), which may be used in the event of a potential public health emergency caused by a biological, chemical, or radiological/nuclear material. MCMs are purchased and stockpiled by the EU through its Joint Procurement Agreement for MCMs, the US Department of Health and Human Services and US Department of Defense, as well as various other foreign governments and militaries



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