Orchard Therapeutics will receive several expedited drug development benefits after the US Food and Drug Administration (FDA) granted priority review to the company’s gene therapy OTL-203 for Hurler syndrome.
The fast-track programme is designed to accelerate the development of medicines that treat serious conditions. Orchard is developing a haematopoietic stem cell gene (HSC) therapy to treat Hurler syndrome, which is a rare lysosomal storage disorder that is also known as mucopolysaccharidosis type I.
OTL-203 received orphan drug status and rare paediatric disease (RPD) designation from the FDA in 2020.
As per the fast-track programme, UK-headquartered Orchard can now have advanced interactions with the FDA during the drug’s development cycle. It also opens the door for potential accelerated approval and priority review.
The FDA’s decision was based on interim and follow-up data from a proof-of-concept study. Both data sets demonstrated continued growth along expected growth percentiles, improvements in skeletal health, and stable cognitive performance. The company has also presented further findings demonstrating improvements in ocular symptoms and auditory function.
Orchard stated it is initiating a multi-centre, randomised, active-controlled clinical trial (NCT06149403) to evaluate the efficacy and safety of OTL-203 in patients compared to HSCT. A total of 40 patients are planned to be enrolled across six sites in the US and Europe. The primary outcome will be event-free survival at two years. The trial is slated to start in December.
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Kyowa Kirin targeted Orchard’s stem cell gene therapy platform to boost its genetic disease portfolio. One of the rare diseases Orchard is homing in on is Hurler syndrome. Patients with this disorder have a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme.
This leads to an accumulation of glycosaminoglycans (GAGs) in tissues, which can cause dysfunction in organ systems. This means children born with the disease rarely live beyond ten years of age.
Orchard Therapeutics is also developing a treatment for mucopolysaccharidosis type IIIA, a disease more commonly known as Sanfilippo syndrome. The company first announced positive preliminary results for its candidate OTL-201 in December 2020. Early results from the trial were presented in February 2023, and updated data was issued in May 2023 with a median follow-up of 2.5 years.
In September, another of the company’s candidates, OTL-200, received FDA priority review designation for the treatment of metachromatic leukodystrophy. The drug is already marketed as Libmeldy in the European Union where it was approved in 2020.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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