EMA Says No to Knee Cartilage Repair Therapy Jelrix
Jelrix is an advanced therapy medicinal product that uses a patient's own cartilage cells that are expanded in a lab and then implanted back into the knee to repair the defect. It was developed for patients with symptomatic cartilage defects of 2 cm² to 12 cm² whose bones have finished growing.
In its decision, the EMA cited concerns about the lack of data to ensure consistent quality of the medicine through its manufacturing process and control strategy.
No Verifiable Improvement
The application was supported by a main study involving 100 patients with cartilage defects in the knee. The primary effectiveness measure was the patient-reported Knee injury and Osteoarthritis Outcome Score (KOOS), which evaluates pain, symptoms, and quality of life on a 0-100 scale, with higher scores indicating fewer symptoms. Assessments were conducted at 24 and 60 months post-treatment. Results were compared indirectly to outcomes from a separate microfracture registry rather than via a randomized control group.
While patients who received Jelrix showed improvement in their KOOS scores at 24 and 60 months, the EMA noted that the study lacked a comparator group. This means the improvements could not be definitively attributed to Jelrix, as they might have resulted from the surgery and rehabilitation all participants received. Therefore, the agency concluded that the benefits of Jelrix did not outweigh its risks.
The company confirmed there are no ongoing clinical trials or compassionate use/expanded access programs involving Jelrix in the EU.
The applicant, Tissue Engineering Technologies AG, may request reexamination within 15 calendar days of receiving EMA's opinion. If pursued, CHMP will reassess their application based on any additional data provided.
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Patients who completed the study had the option to participate in an open-label Extension Phase, with some patients having been dosed for over 5 years. About IntraBio IntraBio Inc. is a global biopharmaceutical company that develops and commercializes targeted therapies for rare and common neurological and neurodevelopmental diseases. IntraBio's platform technologies result from decades of research and collaboration with universities and institutions worldwide, and leverages the expertise of its scientific founders from the University of Oxford and the University of Munich. For more information about IntraBio, please visit the company's website at and follow on LinkedIn (@IntraBio-Inc). References 1. Bremova-Ertl T, et al. J Neurol. 2022;269(3):1651-1662 2. Patterson, Marc C., et al. "Disease-modifying, neuroprotective effect of N-acetyl-l-leucine in adult and pediatric patients with Niemann-Pick disease type C." Neurology 105.1 (2025): e213589. 3. Geberhiwot T, et al. Orphanet J of Rare Dis. 2018;13:50 4. Patterson MC, et al. Orphanet J Rare Dis. 2013;8:12; 3. NORD. NPC Signs & Symptoms. Published Dec 12, 2023. Accessed May 19, 2024. 5. AQNEURSA. Prescribing Information. IntraBio Inc 6. Burton BK, Ellis AG, Orr B, et al. Estimating the prevalence of Niemann-Pick disease type C (NPC) in the United States. Mol Genet Metab 7. Vanier MT. Orphanet J Rare Dis. 2010;5:16.
