Following the news that European Medicines Agency (EMA) granted accelerated assessment of OTL-200;

Vinie Varkey, Senior Analyst of Neurology and Ophthalmology at GlobalData, a leading data and analytics company, offers her view on the implications of this trial for the MLD market:

“With Orchard Therapeutics’ OTL-200 receiving the accelerated assessment, it is now more likely that an effective treatment for metachromatic leukodystrophy (MLD) patients will become available sooner than anticipated. This is crucial for patients as currently-available treatment options are ineffective. If OTL-200 is approved in the EU, it will be the first therapy in the market with the potential to cure the disease.

“According to GlobalData’s recent report, ‘Gene therapies in Neurology’, OTL-200 is the only gene therapy product that is both in phase III development stage and one that is employing a lentiviral vector. Ongoing clinical trials are evaluating both fresh and cryopreserved formulations. The latter option is understood to help optimize the quality control of the final product, as well as addressing the time constraints that are associated with a typical ex-vivo gene therapy procedure.

“While establishing a favourable safety and efficacy profile for gene therapies is of prime importance, it is also imperative that companies in this field have the know-how to manage the lifecycle of such innovative products. Orchard Therapeutics’ expertise on autologous ex-vivo gene therapies for rare diseases is expected to help them in the long-term management of this products as it is anticipated to advance from pipeline to commercial stages.”