- Small-cap biotech Actinogen Medical (ACW) has secured a key drug designation from the U.S. Food and Drug Administration
- The Rare Paediatric Disease Designation validation means Actinogen’s Xanamen treatment has been selected for priority review
- Xanamen is being developed to treat Fragile X syndrome, a rare genetic disorder with lifelong symptoms
- Ultimately, the validation could bring the drug candidate one step closer to commercialisation
- It comes as Actinogen prepares Xanamen for a clinical trial this year, studying the treatment’s impact on roughly 40 adolescents with Fragile X syndrome
- Today’s news saw Actinogen shares climb 42.9 per cent higher to trade at 3 cents
Small-cap biotech Actinogen Medical (ACW) has secured a key drug designation from the U.S. Food and Drug Administration (FDA).
The validation means Actinogen’s Xanamen treatment has been selected for priority review, which could bring it one step closer to market.
The drug developer is currently working on a treatment for Fragile X syndrome (FXS), a rare genetic disorder with lifelong symptoms. It’s characterised by behavioural problems, autism features, severe anxiety, cognitive impairment and disordered sleep.
Fragile X is generally diagnosed in children, so the U.S. FDA has granted Xanamen Rare Paediatric Disease Designation.
Actinogen could also secure a priority review voucher for Xanamen if the drug candidate achieves market authorisation.
The voucher also carries a commercial opportunity — in the past, biopharmas have sold the vouchers on to pharmaceutical companies for an average of over US$100 million (roughly A$131.61 million).
Actinogen’s Managing Director and CEO, Dr Bill Ketelbey, said the team was “delighted” to receive the validation.
“The significant strategic advantages from this approval include commercial, development and regulatory benefits for the development of Xanamem in FXS, with priority review designed to increase overall speed to market,” he stated today.
“Management of FXS is often complex, with lifelong treatment required for patients. FXS presents as a significant unmet medical need as there are currently no approved drugs to treat the condition.”
Today’s validation comes as Actinogen prepares Xanamen for a clinical trial this year. During the study, Actinogen will assess the treatment’s impact on roughly 40 adolescents with Fragile X syndrome.
Promisingly, the trial is fully funded and expected to wrap up less than a year after the study begins.
Today’s news saw Actinogen shares climb 42.9 per cent higher to trade at 3 cents at 11:40 am AEDT.