NIJMEGEN, the Netherlands – August 4, 2025 – Khondrion, a Phase 3 clinical-stage Dutch biopharmaceutical company pioneering therapies for primary mitochondrial diseases, caused per definition by defects in mitochondrial Oxidative Phosphorylation (OXPHOS), today announced that the European Commission has granted Orphan Drug Designation (ODD) to its lead investigational therapy, sonlicromanol, for the treatment of all inherited mitochondrial oxidative phosphorylation (OXPHOS) defects.
This regulatory milestone marks a significant validation of Khondrion’s development strategy and opens the door to a range of development and commercial incentives, including protocol assistance, reduced regulatory fees, and market exclusivity upon approval.
“This approval represents a major turning point in the regulatory landscape for primary mitochondrial diseases in Europe,” said Prof. Dr. Jan Smeitink, CEO of Khondrion. “Unlike previous designations granted for individual mitochondrial disorders, the European Commission’s recognition of OXPHOS defects as a unified group under a single designation— an approach we, together with our regulatory consultant Steve Pinder from Envestia, have championed for over a decade—broadens the therapeutic reach of sonlicromanol and simplifies the development path across multiple rare disease indications.”
The designation follows a positive opinion from the EMA Committee for Orphan Medicinal Products (COMP), signaling growing regulatory alignment with Khondrion’s innovative and scalable approach to treating mitochondrial dysfunction.
With ODD now in place, Khondrion is well-positioned to advance sonlicromanol into a Phase 3 randomized, double-blind, placebo-controlled clinical trial, expected to enroll its first patient in Q4 2025.
“This is more than a regulatory success—it’s a critical inflection point that enables value creation through regulatory incentives, strategic partnerships, and potential market exclusivity.” added Smeitink. “We’re thrilled to take this next step toward delivering a transformative therapy for patients and unlocking long-term value for our stakeholders.”
Primary mitochondrial diseases are devastating, progressive, multi-system disorders that affect 1:5000 live births. These rare diseases are caused by mutations in two different genomes: the nuclear DNA and the mitochondrial DNA which disturb the mitochondrial oxidative phosphorylation system (OXPHOS). OXPHOS is the final biochemical pathway involved in the production of the cell’s energy (ATP).
This press release may contain certain forward-looking statements regarding, among other things, the results, conduct, progress and timing of the company’s clinical trials and presentation of data from clinical trials for sonlicromanol. Although the company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the company’s control. These statements may include, without limitation, any statements preceded by, followed by or including words such as “target,” “believe,” “expect,” “aim,” “goal,” “intend,” “may,” “anticipate,” “foreseen,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “potential,” “should,” “would,” “could” and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the company’s control that could cause the company’s actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. Except as required by law, the company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.
Khondrion BV
E-mail: info@khondrion.com
Tel: +31-24-7635000
www.khondrion.com
