Khondrion granted Orphan Drug Designation
Khondrion, a clinical-stage pharmaceutical company discovering and developing therapies targeting mitochondrial disease, today announces that the European Commission has granted Orphan Drug Designation (ODD) to its lead asset, KH176, for the treatment of patients with maternally inherited diabetes and deafness (MIDD). MIDD is a subtype of diabetes often accompanied by hearing loss that is caused by a mutation within mitochondrial DNA.
KH176, Khondrion’s wholly-owned lead asset is a potential first-in-class oral small molecule in phase IIb clinical development to treat a range of mitochondrial diseases. The compound, which belongs to a new class of drugs used to control oxidative and redox pathologies, has previously been granted ODDs for MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders and Leigh disease by the EMA, and for all inherited mitochondrial respiratory chain
disorders by the US Food and Drug Administration (FDA).
An orphan designation allows a pharmaceutical company to benefit from incentives to develop a medicine for a rare disease, such as reduced fees in protocol assistance and a period of market exclusivity following regulatory approval. European applications for Orphan Designation are examined by the Committee for Medicinal Products (COMP), which adopts an opinion that is forwarded to the European Commission. The European Commission then decides whether to grant an orphan designation for the medicine in question.
Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, said: “MIDD is a rare and extremely debilitating form of diabetes with a critical need for new treatments. This designation is an important incentive for companies like Khondrion investing in rare disease research and brings us another step closer to advancing the science of mitochondrial disease for the benefit of patients.”