LONDON, UK — Orchard Therapeutics, a Kyowa Kirin company, announced it has been granted Innovation Passport designation for OTL-201, an investigational haemopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A). The designation was awarded under the recently refreshed Innovative Licensing and Access Pathway (ILAP) in the UK, which provides a single integrated platform for sustained collaboration between developers, the Medicines and Healthcare products Regulatory Agency (MHRA), health technology assessment (HTA) authorities, and the National Health Service (NHS). The Innovation Passport is the entry point for companies to participate in ILAP.
MPS-IIIA, also known as Sanfilippo syndrome type A, is a rare, fatal neurometabolic disorder caused by genetic mutations that lead to the toxic accumulation of heparan sulfate in the body. This results in a neurodegenerative disease characterised by progressive loss of cognitive and motor function. Children with MPS-IIIA rarely survive past adolescence, and there currently are no approved disease-modifying therapies to treat this disease.
OTL-201 is one of three investigational therapies initially awarded Innovation Passport under the recently refreshed ILAP, which focuses on potentially transformative medicines that address significant medical needs.
“Being selected as one of the first investigational therapies granted the Innovation Passport designation under the new ILAP is further validation that the life-changing value OTL-201 has the potential to provide children with MPS-IIIA and their families,” said Andrew Olaye, general manager, UK and Ireland at Orchard Therapeutics. “We commend the MHRA for introducing the designation and addressing the need to speed development timelines and quicken access to innovative new treatment options for patients in need.”
About the Innovative Licensing and Access Pathway (ILAP)
The ILAP was first launched in January 2021 to offer developers of promising new medicines a single platform to collaborate with the MHRA and the UK HTA bodies to accelerate the time taken for innovative medicines to get to patients. The refreshed ILAP was launched in January 2025. It is the only end-to-end access pathway in the world where multi-stakeholder engagement is established at an early stage of clinical development. The new pathway features enhanced input and interactions with the MHRA and the ILAP partners, including the All Wales Therapeutics and Toxicology Centre (AWTTC), the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC) and the NHS. Other benefits of the new ILAP include access to a range of services that support clinical development, market access, and health system adoption, reducing the duration for product development and facilitating rapid access to the UK market. More information about the ILAP can be found here.
About MPS-IIIA
Mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A) is a rare, fatal neurometabolic disorder cause by mutations in both copies of the N-sulphoglucosamine sulphohydrolase (SGSH) gene. This impairs production of functional SGSH enzyme needed to break down heparan sulfate. As a result, heparan sulfate builds up to toxic levels in the body including the central nervous system, leading to neurodegenerative disease characterised by progressive loss of cognitive and motor function. MPS-IIIA occurs in approximately one in every 100,000 live births. Life expectancy of children born with MPS-IIIA is estimated to be between 10-25 years (median 14.5 years).
About OTL-201
OTL-201 is an investigational ex vivo autologous HSC gene therapy being developed for the treatment of MPS-IIIA. It uses a modified virus to insert a functional copy of the SGSH gene into a patient’s harvested HSCs. OTL-201 has received rare paediatric disease designation and orphan drug designation from the FDA, orphan designation from the EMA, and was granted Innovation Passport designation under the ILAP in the UK. OTL-201 is currently being evaluated in an ongoing investigator-sponsored proof-of-concept clinical trial in the UK (NCT04201405). It has not been approved by any regulatory agency or health authority.
About Orchard Therapeutics
Orchard Therapeutics, a Kyowa Kirin company, is a global gene therapy leader focused on ending the devastation caused by genetic and other severe diseases by discovering, developing, and commercialising new treatments that tap into the curative potential of HSC gene therapy. In this approach, a patient’s own blood stem cells are genetically modified outside of the body and then reinfused, with the goal of correcting the underlying cause of disease with a single treatment.
Founded in 2015, Orchard’s roots go back to some of the first research and clinical developments involving HSC gene therapy. Our team has played a central role in the evolution of this technology from a promising scientific idea to a potentially life-transforming reality. Today, Orchard is advancing a portfolio of HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.
For more information, please visit www.orchard-tx.com.
About Kyowa Kirin
Kyowa Kirin aims to discover and deliver novel medicines and treatments with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, we have invested in drug discovery and biotechnology innovation for more than 70 years and are currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients with high unmet medical needs, such as bone & mineral, intractable haematological diseases/hemato oncology, and rare diseases. A shared commitment to our values, to sustainable growth, and to making people smile unites us across the globe. You can learn more about the business of Kyowa Kirin at www.kyowakirin.com.
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