Orchard Therapeutics (NASDAQ:ORTX) has received Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for its experimental ex vivo autologous hematopoietic stem cell gene therapy, OTL-203. The HSC gene therapy is under development for mucopolysaccharidosis type I (MPS-1) at San Raffaele Telethon Institute for Gene Therapy in Milan, Italy.
PRIME designation important in the development of OTL-203
Anne Dupraz-Poiseau, the company’s chief regulatory officer, said that they are encouraged by the EMA’s PRIME designation for OTL-203. The grant was based on the initial clinical evaluation data that supported the potential benefit of OTL-203 for MPs-IH patients beyond the existing standard of care. Next year, Anne said they are looking forward to building upon the encouraging preliminary data in the current proof of concept study. She also said that they plan to commence a registration trial for advancing any potential treatments for patients.
Usually, PRIME designation is granted to enhance regulatory support in the European Union to develop promising experimental treatments. These are those medicines whose early clinical data has demonstrated that they could offer significant therapeutic benefits compared to the current standard of care or have the potential of benefiting patients that don’t have treatment options.
Most importantly, PRIME offers multiple benefits to the development of the drugs so that they can get to patients as soon as possible. It offers more interaction and early discussion with the regulatory agency, guidance on regulatory strategy and development plan as well as enhanced accelerated assessment for market authorization application.
Orchard to commence OTL-203 registration study in 2021
The company recently presented more interim data from the ongoing proof-of-concept clinical study evaluating the efficacy and safety of OTL-203 in the severe Hurler MPS-I subtype. In the study that completed enrolment in December last year, 8 patients were treated. By July 2002, all the patients had been monitored for at least six months, with the longest follow-up being around 24 months. The company expects to release complete proof of concept data in one year and commence the registration OTL-203 study in 2021.