Promising Results from Studies of DaxibotulinumtoxinA Treatment for Cervical Dystonia

Data from two studies presented at the American Academy of Neurology (AAN) 2023 annual meeting showed that repeat treatments of daxibotulinumtoxinA (Daxxify; Revance Therapeutics Inc, Nashville, TN) for cervical dystonia (CD) were efficacious, safe, and well tolerated. Significant changes from baseline were reported for Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) total scores as well as for TWSTRS subscales of pain, disability, and symptom severity and more than 2+ improvements in Clinical and Patient Global Impression of Change responder rates were seen over time in those treated with daxibotulinumtoxinA.

DaxibotulinumtoxinA is currently under regulatory review for CD treatment with a Prescription Drug User Fee Act (PDUFA) date of August 19, 2023. This novel botulinum toxin type A with proprietary excipient peptide previously showed safety and efficacy as treatment for CD in a randomized pivotal study, ASPEN-1(NCT03608397). A follow-up open-label extension study (ASPEN-OLS; NCT03617367) was a phase 3 study evaluating the long-term safety and immunogenicity for up to 4 successive treatments for CD. A total of 357 participants (271 from ASPEN-1 and 86 de novo), received 985 daxibotulinumtoxinA treatments over 52 weeks, the dosage of which could be 125U, 200U, 250U, or 300U. Common treatment-related adverse events were dysphagia, muscular weakness, and injection site pain, but there were no serious adverse events. The researchers concluded that these results show that repeat treatments with daxibotulinumtoxinA are safe and well tolerated.

A pooled analysis of results from ASPEN-1 and ASPEN-OLS showed that there were limited treatment-induced anti-drug antibodies detected in participants.