Sanfilippo A gene therapy candidate granted access to UK’s ILAP program
New innovation passport designation for OTL-201 aims to speed development
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Regulatory authorities in the U.K. have granted innovation passport designation to OTL-201, an experimental gene therapy for Sanfilippo syndrome type A, generally the disease’s most severe form.
This new status gives treatment developer Orchard Therapeutics (recently acquired by Kyowa Kirin) access to a U.K. program called ILAP — fully, the Innovative Licensing and Access Pathway. The program aims to speed the development of new medicines with the potential to be transformative in the management of serious illnesses.
The ILAP will allow Orchard to collaborate with the Medicines and Healthcare products Regulatory Agency (MHRA) and other U.K. authorities from the early stages of clinical development through potential approval. The overall goal is to speed clinical development and access to the therapy, if it’s proven effective.
The ILAP was launched in 2021 but refreshed last year to include a wider range of support and input from more U.K. regulatory authorities.
“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,” Andrew Olaye, general manager for the U.K. and Ireland at Orchard Therapeutics, said in a company press release announcing the new status.
Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA or MPS-IIIA, is the most common form of Sanfilippo syndrome. It is caused by mutations in the SGSH gene, which leave the body unable to produce enzymes needed to break down a large sugar molecule called heparan sulfate. That, in turn, allows the sugar to build up to toxic levels in cells, ultimately driving disease symptoms.
4 of 5 children with Sanfilippo A saw benefits with gene therapy
OTL-201 is designed to deliver a functional version of the SGSH gene to a patient’s hematopoietic stem cells (HSCs), which reside in the bone marrow and are responsible for generating new blood and immune cells.
The treatment involves collecting HSCs from a patient, modifying them in a laboratory with the therapeutic gene, and then infusing them back into the patient. The goal is to allow patients to make blood and immune cells that carry a functional version of the gene; these cells can then travel throughout the body to help reduce the toxic buildup of heparan sulfate.
In a small, early clinical trial (NCT04201405), 4 of 5 children with Sanfilippo A experienced continued gains in cognitive skills following treatment with OTL-201. That trial is still ongoing to collect longer-term data.
According to Orchard, OTL-201 is one of three experimental treatments selected for ILAP since its recent refresh.
“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,” Olaye said.
