Freeline Granted ILAP Designation in United Kingdom by MHRA for FLT201, an Investigational Gene Therapy for the Treatment of Gaucher Disease
2023-05-23
基因疗法孤儿药临床研究
Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced that FLT201 has been awarded the Innovation Passport for the treatment of Gaucher disease type 1 under the Innovative Licensing and Access Pathway (ILAP) process by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA). The Innovation Passport is the first step in the ILAP process, which in collaboration with MHRA and partner agencies, aims to accelerate time to market and facilitate patient access to medicines in the UK for life-threatening or seriously debilitating conditions, or conditions for which there is a significant patient or public health need.
“We believe FLT201 has life-changing potential for people with Gaucher disease type 1, the most common type of the disease,” said Pamela Foulds, MD, Chief Medical Officer of Freeline. “There remains a significant unmet need in Gaucher disease. Existing therapies come with a heavy life-long treatment burden, and even with treatment, many patients continue to experience serious symptoms. We believe that FLT201 may improve outcomes for patients with a one-time gene therapy. We look forward to working with MHRA to bring FLT201 to patients in the UK as efficiently as possible.”
The Innovation Passport is granted by the UK's ILAP Steering Group, which consists of representatives from MHRA, the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC), the All Wales Therapeutics and Toxicology Centre (AWTTC), and the National Health Service (NHS) England. Benefits of ILAP include opportunities for enhanced regulatory interactions through the development and approval process, as well as access to a range of development tools, such as the potential for a 150-day accelerated Marketing Authorization Application (MAA) assessment, rolling review and a continuous benefit-risk assessment.
About Gaucher Disease
Gaucher disease is caused by a mutation in the GBA gene that results in abnormally low levels of glucocerebrosidase (GCase), an enzyme needed to metabolize a certain type of lipid. As a result, harmful substrates glucosylceramide (Gb-1) and glucosylsphingosine (lyso-Gb1) build up in cells that then accumulate in various organs, causing inflammation and dysfunction. Gaucher disease is hereditary and presents in various subtypes. Freeline is currently focused on Gaucher disease type 1, the most common form of the disease, which affects the health of the spleen, liver, bone and lungs. Despite treatment with existing treatments, many people with Gaucher disease continue to experience symptoms and disease progression. Gaucher disease affects approximately 18,000 people in the United States, United Kingdom, European Union and Israel.
About FLT201
FLT201 is an adeno-associated virus (AAV) gene therapy candidate that is currently being investigated in the GALILEO-1 Phase 1/2 clinical trial in adults with Gaucher disease type 1. FLT201 is designed to generate durable increases in glucocerebrosidase (GCase) and reduce the accumulation of harmful substrates, with the aim of providing a one-time treatment that can stop disease progression, improve outcomes and free people from lifelong treatment. FLT201 uses Freeline’s proprietary AAVS3 capsid to introduce a novel transgene into liver cells to produce a rationally designed GCase variant. In preclinical studies, the GCase variant has demonstrated a 20-fold increase in half-life at lysosomal pH conditions compared to wild-type human GCase. Preclinically, FLT201 has shown robust GCase expression, as well as significant GCase uptake and substrate reduction in key tissues. FLT201 has been granted Orphan Drug Designation for the treatment of Gaucher disease by US and EU regulatory authorities.
About Freeline Therapeutics
Freeline is a clinical-stage biotechnology company focused on developing transformative gene therapies for chronic debilitating diseases. Freeline uses its proprietary, rationally designed AAV vector and capsid (AAVS3), along with novel promoters and transgenes, to deliver a functional copy of a therapeutic gene into human liver cells, thereby expressing a persistent functional level of the missing or dysfunctional protein into a patient’s bloodstream. The company is currently advancing FLT201, a highly differentiated gene therapy candidate that delivers a novel transgene, in a Phase 1/2 clinical trial in people with Gaucher disease type 1. Freeline is headquartered in the UK and has operations in the United States. For more information, visit www.freeline.life or connect with Freeline on LinkedIn and Twitter.
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