Oxford’s SynaptixBio granted second FDA designation to develop pioneering treatment
Oxford biotech firm SynaptixBio has received a designation from the US Food and Drug Administration which will advance its development of a pioneering drug to treat TUBB4A leukodystrophy, a rare, incurable, and deadly disease.
The Orphan Drug Designation enables firms to cut research expenses by a quarter through tax credits, as well as exempting them from testing requirements and some regulatory fees.
This builds upon last month’s Rare Paediatric Disease Designation, with SynaptixBio now setting its sights on a Priority Review Voucher to accelerate market access for therapeutics. It is hoped that human clinical trials for the new drug can begin next year.
CEO and Co-founder Dr Dan Williams said: “These designations form a key part of our growth strategy. By enabling us to cut red tape and costs, we are able to shorten the timescales around developing a potential drug – bringing it to market quicker and changing more lives.”
Read more - Oxford biotech secures FDA designation in bid to develop treatment for rare and incurable disease
TUBB4A leukodystophy belongs to a group of about 30 rare neurodegenerative disorders, which according to the University of Utah could affect 20,000 people each year.
This particular disease is caused by a mutation in the TUBB4A gene which disrupts the myelin sheath around nerve fibres, interrupting signals between nerve cells in the brain. At its most severe, the condition can lead to significant impairment of motor skills such as walking or even swallowing.
As part of a sponsored research agreement with the Children’s Hospital of Philadelphia (CHOP), SynaptixBio’s research efforts are focused on antisense oligonucleotides (ASOs), small molecules which alter the production of proteins in the body.
ASOs have already produced promising results in the treatment of conditions such as Duchenne muscular dystrophy and spinal muscular atrophy.
Dr Williams said that this form of treatment could “modify the underlying mechanisms of the disease, increase survival and significantly improve motor skills development.
“We hope our work will have a significant impact on the lives of patients and their families. Developing the first treatment for TUBB4A would be a monumental step forward in addressing this debilitating and life-threatening condition.
“By securing these designations, we are very much on course to take our research and development to clinic next year.”
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