Actigen Receives Positive FDA Response for Rare Disease Treatment

Actigen is one step closer to conducting clinical trials in the US for a potential breakthrough in rare disease treatment, following a recent positive pre-Investigational New Drug (IND) meeting with the US Food and Drug Administration (FDA).

Actigen filed for a pre-IND advice meeting with the FDA to discuss the scientific, regulatory and safety issues related to developing GNR-055, a potential new rare disease treatment for mucopolysaccharidosis II (MPS II, also known as Hunter syndrome). The company received a positive response, including helpful and constructive feedback, strengthening the case for clinical trials in the US.

The next major step in the application process in the US is submitting the IND application and, subject to successful FDA approval, Actigen will be leading new clinical trials for GNR-055 in MPS II patients.

Occurring in around 1 in 100,0000-170,000 births, MPS II presents almost exclusively in males and has a major impact on the physical and neurological health of those affected. Like many rare diseases, MPS II lacks adequate treatment options. The current treatment options, such as enzyme replacement therapy, only offer a partial solution. While they can help improve the patient’s internal organ function, they cannot cross the blood-brain barrier, resulting in ongoing neurological issues.

GNR-055 treatment uses an innovative combination of an Iduronate 2-Sulfatase (IDS) enzyme and antibody fragment to access the brain cells. GNR-055 is expected to break down the glycosaminoglycans (GAGs) that cause neurological manifestation in the brain. This pioneering treatment could fulfil an unmet clinical need and offer life-changing improvements for patients affected by MPS II.

Actigen’s Managing Director, Michael Braunagel, said, “The positive response from the pre-IND meeting strengthens our case for clinical trials in the US and shows that our approach is valid. We are looking forward to continuing our discussions with the FDA on specific issues raised and are optimistic to be in a position to conduct our clinical trials in the near future. GNR-055 is a potentially game-changing treatment for MPS II patients, with the ability to address the serious neurological difficulties they experience.

MPS II has serious, life-limiting effects on those affected, and we are hopeful that our forthcoming clinical trials will demonstrate the treatment’s ability to transform patients’ quality of life. We are confident that we will successfully progress to the IND stage based on our initial discussions.”