Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to OTL-203, an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.
“We are encouraged by EMA’s decision to grant PRIME designation to OTL-203, which was based on an initial clinical assessment of data supporting the potential benefit of our HSC gene therapy for patients with MPS-IH beyond the current standard of care,” said Anne Dupraz-Poiseau, PhD., chief regulatory officer of Orchard. “In 2021, we look forward to building upon the promising early data in the ongoing proof-of-concept study and plan to initiate a registrational trial to advance a potential new treatment for patients.”
The PRIME program is designed to enhance regulatory support in the EU for the development of promising investigational medicines that, based on early clinical data, may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. PRIME aims to provide multiple benefits so that these medicines can reach patients earlier: enhanced interaction and early dialogue with EMA, guidance on the overall development plan and regulatory strategy, and the potential for accelerated assessment of the marketing authorization application. For more information please visit the EMA website at www.ema.europa.eu.
Additional interim data was recently presented from the ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203 in the severe Hurler subtype of MPS-I. Eight patients have been treated in the study, which completed enrollment in December 2019. As of July 2020, all patients had been followed for a minimum of six months, with the longest follow-up extending out to 24 months. Orchard expects to release full proof-of-concept results at one year and initiate a registrational study for OTL-203 in 2021.
About OTL-203 and MPS-I
Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to break down sugar molecules called glycosaminoglycans (also known as GAGs). The accumulation of GAGs across multiple organ systems results in symptoms including neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births. There are three subtypes of MPS-I; approximately 60 percent of children born with MPS-I have the most severe subtype, called Hurler syndrome, and rarely live past the age of 10 when untreated.
Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an investigational ex vivo autologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy.
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