Orchard Therapeutics Receives EC Approval for Treatment of Early-Onset Metachromatic Leukodystrophy

LONDON & BOSTON – 4BIO Capital (“4BIO” or “the Group”), an international venture capital firm focused solely on the advanced therapies sector, notes that portfolio company Orchard Therapeutics, a global leader in gene therapy, announced that the European Commission (EC) granted full (standard) market authorization for Libmeldy, a lentiviral based gene therapy developed for the treatment of metachromatic leukodystrophy (MLD).


MLD is a rare hereditary disease characterized by biallelic mutations in the ARSA gene leading to neurological damage and developmental regression. Libmeldy was designed as a one-time therapy aimed at correcting the underlying genetic cause of MLD, for which there are currently no other available treatment options. This novel gene therapy may offer patients the potential for long term positive effects on cognitive development and maintenance of motor function, which, without treatment, can cause patients to develop severe motor and cognitive impairments.


The EC approval for Libmeldy is supported by clinical studies in both pre- and early symptomatic, early-onset MLD patients. Clinical efficacy was based on integrated data analysis from 29 patients with early-onset MLD and was indicative of a single-dose intravenous administration of Libmeldy being effective in modifying the disease course of early-onset MLD in most patients. Safety data obtained from 35 patients with MLD, indicated that Libmedly was generally well tolerated, with the most common adverse reaction, production of anti-ARSA antibodies, reported in five patients. Additionally, antibody titers in all five patients were generally low and no negative effects were observed in post-treatment ARSA activity in the peripheral blood or in bone marrow cellular subpopulations, nor in the ARSA activity within the cerebrospinal fluid.


Dmitry Kuzmin, Managing Partner at 4BIO Capital, commented: “Gaining EC approval is a fantastic accomplishment for the company and brings us one step closer to providing a treatment option for many children suffering from MLD.”


Click here to read the full press release from Orchard Therapeutics.

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