Designation granted for the treatment of MELAS (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes) syndrome. 

Khondrion, a clinical-stage biopharmaceutical company discovering and developing therapies targeting mitochondrial disease, today announces that it received a rare pediatric disease (RPD) designation from the United States (US) Food and Drug Administration (FDA) for sonlicromonal for the treatment of patients with MELAS syndrome.

Sonlicromanol is Khondrion’s wholly-owned, potentially first-in-class oral small molecule and one of the most clinically-advanced disease-modifying drug treatments for mitochondrial disease in development. Currently in Phase IIb clinical development,  sonlicromanol has already been granted Orphan Drug Designations for MELAS, Leigh disease and patients with maternally inherited diabetes and deafness (MIDD) syndrome in Europe, and for all inherited mitochondrial respiratory chain disorders in the US.

The FDA grants RPD designation for serious and life-threatening diseases that primarily affect children aged 18 years or younger and fewer than 200,000 people in the United States. Subject to FDA approval of sonlicromanol for the treatment of MELAS syndrome, Khondrion may be granted a priority review voucher from the FDA that can be redeemed to obtain priority review for any subsequent marketing application or be sold or transferred to another company.

Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, said: We are delighted to have received this rare pediatric disease designation for our lead clinical stage development program. It signals the FDA’s recognition that MELAS syndrome presents a serious and unmet medical need and highlights the potential of sonlicromanol to become the first disease-modifying treatment for this devastating mitochondrial disease”.

A Phase IIb study (KHENERGYZE) of sonlicromanol in MELAS spectrum disorders is currently ongoing across a number of internationally recognised mitochondrial disease centres in Europe. Khondrion intends to submit a pre-IND (Investigational New Drug) request in the short term to further discuss the development plan for sonlicromanol in the US.