Oryzon leukemia drug granted orphan designation

By Biocat

Oryzon Genomics announced that the European Medicines Agency (EMA) considers its acute myeloid leukemia (AML) drug ORY-1001 an orphan drug. The Agency’s Committee for Orphan Medicinal Products (COMP), which is responsible for reviewing applications for this designation, has included ORY-1001 on the list of drugs for rare diseases.

ORY-1001 is a highly selective oral inhibitor of LSD1, which regulates gene expression by modifying certain compounds in the histones (proteins that package and order DNA into structural units called nucleosomes). ORY-1001, thus, reduces leukemic stem-cell potential by preventing proliferation and colony formation. The drug developed by Oryzon has successfully passed regulatory toxicology studies and is now entering phase I clinical trials with AML patients.

The orphan drug designation facilitates development of treatments for rare diseases, which according to the European Union are those that affect less than five in 10,000 people. Although, for economic reasons, it is difficult to get the pharmaceutical industry to produce these drugs, they meet a public health need.

ORY-1001 being cataloged as an orphan drug recognizes its scientific importance in treating AML and Oryzon will benefit from certain advantages like 10-year market exclusivity, fee reductions and grants for research associated with this type of blood cancer. Oryzon CEO Carlos Buesa explains that "this will help us in the clinical development of our compound as we have identified now a subset of diseases in which this mechanism looks particularly efficient."

Oryzon has a wide-reaching program for drug discovery, with nearly 900 compounds and two preclinical candidates, and specializes in biomarkers for various oncological and neurodegenerative diseases. In this field, in 2011, the company received a €300,000 grant for research into new Alzheimer therapies and presented its advances at a congress in the United States last June.