Rare Disease Report

Orphan Drug Designation Granted to Potential Acute Radiation Syndrome Treatment

JANUARY 22, 2018
Kaitlynn Ely
Enzychem Lifesciences has announced that EC-18, a potential therapy for Acute Radiation Syndrome (ARS), has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA).

Historically, ARS has primarily affected survivors of the nuclear bombings in Hiroshima and Nagasak, Japan, and the first responders of the Chernobyl Nuclear Power Plant disaster in  the Ukraine in 1986.

The small molecule compound was initially developed to be orally administered to treat immune and inflammatory related diseases, including psoriasis, rheumatoid arthritis, asthma, atopic dermatitis, and sepsis.

ARS is developed after a patient is exposed to high amounts of radiation in a short period of time, breaking down immature parenchymal stem cells in tissues due to the irradiation of the entire body. For someone to develop ARS, the radiation dose must be large, external and capable of penetrating internal organs.

ARS can cause neutropenia, nervous system damage, and pneumonia with a high fatality rate. Early symptoms of the condition occur within the first few days after initial exposure, and include: nausea, vomiting, anorexia, and possible diarrhea. Patients whose symptoms do not improve after experiencing these can suffer from more severe symptoms like the destruction of bone marrow causing infection, irreparable changes in the GI tract, or in the most extreme cases, the complete collapse of the nervous system. Without proper treatment, most patients will die within a year of exposure.

In a press release, Enzychem Lifesciences Chief Executive Officer Ki Young Sohn stated, "with award of the Orphan Drug Designation for EC-18, Enzychem aims to develop medicines that enhance patients' health and, especially, to address unmet clinical needs from the current market."

As a segment of the agency’s monthly Public Health Grand Rounds, the Centers for Disease Control and Prevention (CDC) held a session on January 16 to discuss safety measures and the training of medical response teams, in the event that a nuclear detonation were to occur in the United States.

Enzychem Lifesciences is currently conducting phase II clinical trials on treatments for chemotherapy-induced neutropenia and chemo-radiotherapy induced oral mucositis.

If EC-18 receives FDA approval as an ARS drug, the treatment could be granted a Priority Review Voucher as a drug against bioterrorism. The voucher offers companies the opportunity to create various strategies in utilizing EC-18 and speeds up the approval process of potential breakthrough therapies in the United States.

The priority review voucher is tradable and allows companies to develop an array of strategies regarding the utilization of PRV. From previous transactions on sales of vouchers, the average market price is $200 million.

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