On 22 February 2024, the European Medicines Agency ratified transition of Holoclar from conditional to full Marketing Authorisation

On 22 February 2024, the European Commission ratified, with decision no. 1299 (2024) the passage of the European marketing authorization for Holoclar, cell therapy for the orphan designation of Limbal Stem Cell Deficiency, from ”conditional” to FULL . Holoclar is a therapy consisting of an ex-vivo reconstructed autologous human corneal epithelium containing stem cells.

This step had received, at the end of 2023, a positive opinion from the CHMP (Committee for Medicinal Products for Human Use) of EMA, the European Medicines Agency, following the completion of the interventional and prospective clinical study HOLOCORE.
The results of the study are published in the European register for clinical trials – EU Clinical Trials Register.

Subsequently, the HOLOCORE -FU follow-up study was completed, the results of which are available in the European register for clinical trials – EU Clinical Trials Register .
The conversion to full marketing authorization was also approved by the MHRA in the United Kingdom on 8 March 2024.

In total, 223 patients received transplants with Holoclar in the HLSTM01, HLSTM02, HLSTM04 and HOLOCORE clinical trials. This is the largest cohort of LSCD patients participating in a clinical development observed to date. Overall, 14 patients received the second implant with Holoclar.
The HOLOCORE study results on mid- and long-term safety and efficacy data presented confirm the Holoclar benefits and safety profile observed in previous retrospective studies. The overall success rate from a minimum of 51% in the Study HOLOCORE (measured according to Global Consensus standard guidelines) up to 77% (measured by ophthalmologists) with an overall improvement of 60% the population affected by LSCD since 153 months on average is sufficiently meaningful, as clinical assessments by ophthalmologist would be the most reliable parameter to define treatment success.
The PASS ‘registry_like’ HOLOSIGHT study, which involves the observation of patients for up to 5 years after Holoclar implantation, has completed the enrollment of the expected 100 patients treated in a commercial setting and is still ongoing.