RALEIGH, N.C., December 21, 2020 – Merz Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has approved a supplemental Biologics License Application (sBLA) for Xeomin (incobotulinumtoxinA) for the treatment of patients aged 2 years and older with chronic sialorrhea, or drooling. Xeomin is the first and only FDA-approved neuromodulator with this indication and was granted a priority review designation upon acceptance by the FDA.
“Pediatric patients living with chronic sialorrhea have suffered with this debilitating condition without a viable long-acting treatment option,” said Kevin O’Brien, President of North America, Merz Therapeutics. “Merz Therapeutics is proud to offer Xeomin, the first and only FDA-approved neurotoxin treatment that is uniquely purified to provide safe and effective treatment for this condition. This approval reinforces our ongoing commitment to advancing new indications for Xeomin to meet the needs of adult and pediatric patients living with movement disorders.”
The pediatric chronic sialorrhea approval is based on a Phase 3 prospective, randomized, double-blind, placebo-controlled, multicenter study evaluating the safety and efficacy of Xeomin in 255 children and adolescents aged 2 – 17 years. The study duration was 64 weeks, with a 16-week main period followed by three additional injections during a 48-week open label extension period. The co-primary endpoints among patients aged 6 ‒ 17 years were defined as the change in unstimulated Salivary Flow Rate (uSFR) from baseline to Week 4 and the Global Impression of Change Scale (GICS) score from baseline to Week 4, representing the functional improvement in drooling, as assessed by the caregiver.
Xeomin demonstrated significantly reduced uSFR and improved GICS versus placebo at Week 4 among patients aged 6 ‒ 17 years, and sustained efficacy over 64 weeks. Improvement in chronic sialorrhea increased with each injection cycle vs baseline. GICS scores were comparable among patients ages 2 ‒ 5 years, all of whom received Xeomin treatment and not placebo throughout the study. No patients demonstrated clinical resistance or secondary treatment failure due to neutralizing antibodies (Nab), supporting the importance of Xeomin’s unique purification process through XTRACT Technology™.
“It’s estimated that 300,000 children in the U.S. suffer from chronic drooling due to cerebral palsy or brain injury, but this approval provides them a safe and efficacious treatment option that they can use long-term with low probability they’ll build up an immunity to it with repeated use,”1,2 said Elizabeth Moberg-Wolff, M.D., Pediatric Rehabilitation Medicine Associates.* “Having Xeomin approved to treat another symptom that children living with movement disorders experience is an important milestone for patients, caregivers and healthcare providers.”**
The safety findings were similar to previous adult and pediatric studies and in line with the known safety profile of Xeomin. The most common adverse reactions affecting ≥1% of patients aged 6 – 17 years were bronchitis, headache and nausea/vomiting. The most common adverse reaction affecting patients aged 2 – 5 years was nasopharyngitis.
Sialorrhea is a commonly occurring condition in neurologically impaired children who have cerebral palsy or who have suffered a traumatic brain injury. The condition can occur from difficulty retaining saliva inside the mouth, issues with swallowing and from problems controlling facial muscles.3,4 Sialorrhea can cause a variety of physical and psychosocial complications, including dehydration, odor, and social stigmatization, which can be devastating for patients and their families.3
Xeomin now holds six first-line therapeutic indications in the U.S. and has helped 3.6 million patients worldwide for various indications. Merz Therapeutics is committed to ensuring Xeomin is accessible and affordable to all patients through our MERZ CONNECT™ savings and assistance programs. Learn more at https://www.xeomin.com/patient-savings-program.
*Dr. Elizabeth Moberg-Wolff is a paid spokesperson for Merz Therapeutics.
**The direct impact of the non-therapeutic proteins on long term safety or efficacy has not been established. Information about the unique XEOMIN manufacturing process and the properties of incobotulinumtoxinA is not intended to imply superiority over other botulinum toxin type A products.
Xeomin is a prescription medicine:
- that is injected into glands that make saliva and is used to treat long-lasting (chronic) drooling (sialorrhea) in adults and in children 2 to 17 years of age.
- that is injected into muscles and used to:treat increased muscle stiffness in the arm because of upper limb spasticity in adults.
- treat increased muscle stiffness in the arm in children 2 to 17 years of age with upper limb spasticity, excluding spasticity caused by cerebral palsy.
- treat the abnormal head position and neck pain with cervical dystonia (CD) in adults.
- treat abnormal spasm of the eyelids (blepharospasm) in adults.
It is not known if Xeomin is safe and effective in children younger than:
- 2 years of age for the treatment of chronic sialorrhea
- 2 years of age for the treatment of upper limb spasticity
- 18 years of age for the treatment of cervical dystonia or blepharospasm
Merz Therapeutics, a business of Merz Pharmaceuticals GmbH, is a global healthcare leader dedicated to improving the lives of patients around the world. With our relentless research, development, and culture of innovation, we strive to serve unmet patient needs and realize better outcomes. Merz Therapeutics seeks to address the unique needs of people who suffer from movement disorders, neurological conditions and other health conditions that severely impact patients’ quality of life. Merz Therapeutics is headquartered in Frankfurt, Germany and is represented in more than 90 countries, with a North America affiliate based in Raleigh, North Carolina. Merz Pharmaceuticals GmbH is part of the Merz Group, a privately held, family-owned company that has dedicated more than 110 years to developing innovations that meet patient and customer needs.
Source: Merz Therapeutics
Posted: December 2020
Xeomin (incobotulinumtoxinA) FDA Approval History