Khondrion granted Orphan Drug Designation for KH176 for the treatment of maternally inherited diabetes and deafness (MIDD) from European Commission
NIJMEGEN, the Netherlands –July 2, 2019: 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.”
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Khondrion is a clinical-stage pharmaceutical company discovering and developing therapies targeting mitochondrial disease. Founded by Prof. Jan Smeitink, a world-leader in mitochondrial medicine, the company is advancing its proprietary science through a wholly-owned clinical and preclinical small molecule pipeline of potential medicines.
Khondrion’s lead asset, KH176, is a potential first-in-class oral small molecule in phase IIb clinical development to treat a range of mitochondrial diseases including m.3243A>G spectrum disorders including MELAS, MIDD and mixed phenotypes. It has been granted Orphan Drug Designation for MELAS spectrum disorders and Leigh disease in Europe and for all inherited mitochondrial respiratory chain disorders in the USA.
The company’s in-house discovery engine is using unique live-cell imaging technologies, patient-derived cell lines and predictive cell-based disease models to build a portfolio of promising compounds. Active discovery programmes are underway developing new therapies, biomarkers and new read-out technologies in the field of mitochondrial diseases.
To accelerate the discovery and development of its potential medicines for mitochondrial diseases, Khondrion collaborates with a global clinical and academic network and patient organisations internationally. Khondrion is headquartered in Nijmegen, The Netherlands. For more information visit www.khondrion.com
About mitochondrial disease
Mitochondrial disease occurs when mitochondria, found within all cells of the human body and responsible for producing the energy necessary for life, are defective. This can result in a wide range of serious and debilitating illnesses, signs and symptoms of which can include: cognitive problems, learning disabilities, blindness, deafness, heart failure, diabetes, fatigue, intolerance to exercise, muscle weakness and gait problems, and stunted growth. Orphan diseases of the oxidative phosphorylation system like Leigh disease, MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders, MIDD (maternally inherited diabetes and deafness), LHON (Leber’s hereditary optic neuropathy) and other respiratory chain/ oxidative phosphorylation disorders, are all examples of mitochondrial disease.