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FDA Approves Long-Acting Botulinum Toxin for Cervical Dystonia
Daxxify is a long-acting botulinum toxin that will launch in 2024. It previously received FDA approval to treat frown lines.
The FDA has approved the first therapeutic indication for Daxxify (daxibotulinumtoxinA-lanm) to treat adults with cervical dystonia in adults. Cervical dystonia is a chronic, debilitating disease in which the neck muscles contract involuntarily, causing abnormal movements, pain and awkward posture of the head and neck. It affects about 60,000 Americans.
Botulinum toxin injections are the standard of care for cervical dystonia, which works by relaxing the affected muscles and reducing spasming and stiffness. But the treatment effect gradually wears off, which is why patients need to be retreated on a regular basis.
Daxxify injection is a long-acting acetylcholine release inhibitor and neuromuscular blocking agent. Developed by Revance Therapeutics, Daxxxify was developed with a stabilizer that allows for longer treatment effect, between 15 weeks and 28 weeks. It will be available beginning in 2024 after a company’s sponsored education program, CD PrevU. A list price will be available when it is launched, according to a company spokesperson.
Peter McAllister, M.D.
“Currently, patients experience painful and life-limiting symptom recurrence as early as eight to 10 weeks in clinical practice but cannot be re-treated until 12 weeks. Daxxify is the first long-acting neuromodulator that has the potential to address this significant unmet need -- demonstrating durable symptom relief between treatment cycles and providing the opportunity to extend treatment intervals,” Peter McAllister, M.D., trial investigator and co-founder and medical director of the New England Institute for Neurology and Headache, said in a press release.
Related: FDA Approves Longer-Lasting Treatment for Frown Lines
Daxxify was approved by the FDA in September 2022 for the temporary improvement of glabellar lines (frown lines) in adults.
The approval of Daxxify to treat patients with cervical dystonia was based on data generated in the phase 3 clinical program (ASPEN 1, ASPEN OLS), which included 382 patients and 1,240 treatments across up to five injection cycles over an 88-week time span. Data were presented at the annual meeting of Association of Academic Physiatrists (AAP), in Anaheim, Calif., February 2023.
In the ASPEN study, Daxiffy was shown to be effective and and well tolerated across two different dosing groups with a median duration of effect of 24.0 and 20.3 weeks for the two dose groups. Based on the ASPEN OLS study, symptoms continued to improve with successive Daxxify treatments, while adverse events remained low. In particular, dysphagia rates (difficulty swallowing) remained low.
Separately, Revance has partnered with Viatris to develop a biosimilar to Botox (onabotulinumtoxinA).
FDA Approves Xolremdi for Ultra Rare Immune Disorder
April 29th 2024Xolremdi is the first therapy for WHIM syndrome, which can cause recurrent lung infections and papillomavirus-related warts. It’s available in two doses: 400 mg for an annual cost of $496,400 and 300 mg for an annual cost of $372,300.
FDA Approves Pfizer’s Gene Therapy Beqvez for Hemophilia
April 26th 2024Beqvez (fidanacogene elaparvovec) is priced at $3.5 million, which is on parity with Hemgenix, the first one-time therapy to treat adults with hemophilia B. Pfizer’s warranty will refund insurers and continue to provide coverage for patients if they change insurers.
