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ARTICLE | Clinical News

Orchard planning MAA for leukodystrophy stem cell gene therapy

March 29, 2019 9:14 PM UTC

Orchard plans to submit an MAA to EMA in 2020 for OTL-200 to treat metachromatic leukodystrophy after reporting that the therapy met a co-primary endpoint of improving motor function at two years following treatment in a Phase I/II trial. Orchard Therapeutics plc (NASDAQ:ORTX) also said it is preparing a BLA submission to FDA for the ex vivo autologous hematopoietic stem cell-based gene therapy that delivers the arylsulfatase A (ARSA) gene.

On the motor function endpoint, OTL-200 led to significantly greater Gross Motor Function Measure (GMFM) scores at 2 years following treatment in both late infantile (72.5% vs. 7.4%, p<0.001) and early juvenile patients (76.5% vs. 36.6%, p=0.026) vs. an age-matched natural history control cohort of 31 untreated early onset patients. Those improvements were maintained at 3 years following treatment in both late infantile (73.9% vs. 2.4%, p<0.001) and early juvenile patients (71.7% vs. 31.3%, p=0.02) vs. the control cohort...

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Orchard Therapeutics plc

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Arylsulfatase A (ARSA)