ABSTRACT Background DaxibotulinumtoxinA for injection (DAXI), a novel botulinum toxin (BoNT) formulated with a custom‐engineered peptide, was recently approved for treating cervical dystonia (CD). DAXI demonstrated a long duration of symptom relief in Phase 3 trials. Objective To report findings from PrevU, an early experience, real‐world observational program initiated following DAXI's FDA approval. Methods Movement disorder specialists were provided with DAXI to treat CD patients per routine clinical practice, and were surveyed on dosing patterns and treatment intervals across the first three cycles of DAXI as doses were optimized. Results A total of 234 CD patients (206 receiving BoNT treatment and 28 BoNT‐naïve) received a total of 589 DAXI treatments. Among patients with prior BoNT therapy, breakthrough symptoms within 12 weeks of prior BoNT therapy was the most common reason for patient selection (76.2%), and onabotulinumtoxinA was the most common prior toxin (68.4%). DAXI dose was titrated from a mean of 244.4 U (median 250 U) to 314.7 U (median 300 U) by Cycle 3. The ratio of DAXI dose to normalized prior BoNT dose was approximately 1.1:1 at initiation and 1.4:1 at Cycle 3. The mean (SD) time to re‐treatment across all DAXI cycles was 16.1 (5.6) weeks for all patients and 15.7 (4.5) weeks for patients with a history of breakthrough before 12 weeks with prior toxin. The most common adverse events were muscle weakness (2.4% of treatments), injection pain (1.4% of treatments), and dysphagia (0.3% of treatments). Conclusions In this real‐world setting, DAXI demonstrated extended clinical benefit and a favorable safety profile.
Bahroo et al. (Tue,) studied this question.