The US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to AskBio’s investigational Parkinson’s disease (PD) gene therapy.
The FDA grants RMAT designation to regenerative therapies being developed for serious or life-threatening diseases and conditions.
The Bayer subsidiary’s AB-1005 will now benefit from improved access to the regulator, which may include comprehensive guidance on efficient drug development, rolling Biologics License Application review, and other measures to accelerate both the development and approval process of the therapy.
More than ten million people worldwide are estimated to be living with PD, a progressive neurodegenerative disease characterised by symptoms such as tremor, muscle rigidity and slowness of movement.
Recombinant glial cell line-derived neurotrophic factor (GDNF), a protein that helps neurons survive and differentiate, has long been evaluated as a potential treatment for diseases marked by progressive degeneration of midbrain dopaminergic neurons, including PD.
AB-1005, formerly known as AAV2-GDNF, is designed to allow for stable and continuous expression of GDNF in localised regions of the brain following direct neurosurgical injection.
Among the evidence supporting the FDA’s decision were the 36-month results from an open-label phase 1b trial of AB-1005, which demonstrated continued positive trends in assessed clinical outcome measures and showed that the candidate was well tolerated and not associated with any product-related serious adverse events.
The first patients in the phase 2 REGENERATE-PD study of AB-1005 have already been randomised in the US, and additional study sites in the US, UK, Germany and Poland are expected to open for enrolment in the first half of 2025.
AskBio’s chief executive officer, Gustavo Pesquin, said: “The FDA’s decision to grant RMAT designation to AB-1005 is exciting news for people living with PD and their loved ones.
“This milestone could potentially expedite the development of our important investigational gene therapy programme, and it highlights our promising data and the potential of AB-1005 for patients and the medical community. We look forward to working closely with the FDA to accelerate our programme”
Christian Rommel, executive vice president, global head of research at Bayer, added: “The RMAT designation for AB-1005 underscores the high unmet medical need and the potential of this investigational gene therapy to make a difference for patients with PD.”