BRIEF—Khondrion gets RPD designation for sonlicromanol

Dutch biopharma company Khondrion says it received a rare pediatric disease (RPD) designation from the US Food and Drug Administration for sonlicromanol 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 USA.

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.

A Phase IIb study (KHENERGYZE) of sonlicromanol in MELAS spectrum disorders is currently ongoing across a number of internationally recognized mitochondrial disease centers 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 USA.