Green light for potential AAV gene therapy 

MHRA

Complement Therapeutics’ lead asset CTx001, an AAV-based gene therapy for the treatment of geographic atrophy (GA), has been awarded an Innovation Passport by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA). 

CTx001 was awarded the Innovation Passport based on three criteria: that the condition addressed is seriously debilitating with a significant patient need; an advanced therapy medicinal product classified as innovative; and that the medicine has the potential to offer transformative benefits to patients. 

“Receiving the Innovation Passport Designation is a key regulatory accomplishment and has the potential to help GA patients in the UK gain faster access to our novel gene therapy,” said Dr Rafiq Hasan, CEO of Complement Therapeutics. “It provides recognition from the MHRA of the unique mechanism of action of CTx001, with the potential to change the treatment landscape for GA patients. We look forward to working with the MHRA and other agencies to advance CTx001 development programme.” 

The Innovation Passport is the first step in the Innovative Licensing and Access Pathway (ILAP) by providing opportunities for enhanced regulatory support from the MHRA and its partner agencies, including the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC) and National Health Service (NHS) England. CTx will work with these agencies on a Target Development Profile (TDP) for CTx001, that will set out a roadmap for regulatory and development milestones to enable early patient access in the UK.  

Other benefits of ILAP, such as the potential for a 150-day accelerated Marketing Authorisation Application (MAA) assessment, a rolling review and a continuous risk-benefit assessment. 

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