The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorisation for NEZGLYAL® (leriglitazone) for the treatment of boys aged 2 to 12 years with early-stage cerebral adrenoleukodystrophy (cALD). The European Commission is expected to issue its final decision before the end of September 2026.
For Minoryx Therapeutics, this milestone is the culmination of more than a decade of scientific research, clinical development and regulatory work. For patients and their families, it represents the prospect of accessing, for the first time in Europe, a pharmacological treatment for a rare neurodegenerative disease that until now has had no approved therapeutic options.
"At Columbus Venture Partners, we are proud to have supported Minoryx throughout this journey, helping transform a scientific breakthrough into a therapy that is now on the verge of reaching the patients who need it most," said the founders of Columbus Venture Partners, referring to the firm's investment in the company through its third life sciences fund.
From scientific discovery to an approved medicine: the real challenge of biotechnology
"Discovering a promising molecule is only the beginning. The real challenge is transforming that discovery into a safe and effective medicine capable of successfully navigating one of the world's most demanding regulatory pathways. The CHMP recommendation represents precisely that validation," said Damià Tormo, board member of Minoryx Therapeutics and founder of Columbus Venture Partners.
The recommendation is based on results from the Phase 2/3 NEXUS trial, supported by additional real-world evidence generated through compassionate use programmes. The data demonstrated that paediatric patients treated with leriglitazone remained clinically and radiologically stable over extended periods—an especially significant outcome in a disease characterised by its rapid neurological progression.
In the field of rare diseases, where clinical development is particularly challenging due to the limited number of patients, reaching a regulatory milestone of this magnitude represents scientific, clinical and industrial validation of exceptional value.
A new hope for a devastating disease
X-linked adrenoleukodystrophy (X-ALD) is a rare inherited neurodegenerative disorder. Between 30% and 35% of affected boys develop its cerebral form, cALD, typically between the ages of two and twelve.
The disease causes inflammatory brain lesions that destroy myelin, leading to rapid neurological deterioration, progressive loss of motor and cognitive functions and, without treatment, a median survival of only three to four years after symptom onset.
"Until now, therapeutic options have been extremely limited. Haematopoietic stem cell transplantation can halt disease progression in selected patients, but it is a highly invasive procedure that is not suitable or available for everyone," explained Marc Martinell, CEO of Minoryx Therapeutics. "If the European Commission confirms the CHMP recommendation, NEZGLYAL® will become the first pharmacological treatment approved in Europe for this disease, offering a new therapeutic option for children with early-stage cerebral lesions."
A clear example of the value of long-term investment in biotechnology
Minoryx's journey demonstrates that developing an innovative medicine requires far more than scientific excellence. It demands long-term vision, sustained investment, strong execution and close collaboration between researchers, clinicians, patients, regulators and investors.
Founded in Barcelona in 2011, the company has advanced leriglitazone from early research to the threshold of European approval while continuing to expand its clinical programme into additional indications, including adult cALD and Rett syndrome.
For Columbus Venture Partners, this milestone reflects the philosophy that has guided its investment strategy since inception: identifying transformative science and supporting it through every stage of development until it becomes a real solution for patients.
Minoryx Therapeutics is a portfolio company of Columbus Life Sciences Fund III.
