FDA approves pediatric indication for XEOMIN^® (incobotulinumtoxinA) For the treatment of chronic sialorrhea

• XEOMIN is the first and only neuromodulator approved in the U.S. to treat pediatric patients with chronic sialorrhea
• No patients developed clinical resistance or secondary treatment failure due to neutralizing antibodies, supporting the importance of XEOMIN’s unique purification process

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.

Merz Therapeutics

At Merz Therapeutics, we seek 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. With our patient-centric approach, cutting-edge research and development efforts, highly-scientific medical affairs resources and dedicated commercial teams, we continue the advancement of new and individualized treatment standards, including botulinum toxin. Merz Therapeutics, a business of Merz Pharmaceuticals GmbH, 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 serve unmet patient and customer needs.

*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^® (incobotulinumtoxinA) IMPORTANT CONSUMER SAFETY INFORMATION



Read the Medication Guide before you start receiving XEOMIN^® (Zeo-min) and each time XEOMIN is given to you as there may be new information.  The risk information provided here is not comprehensive. To learn more:

• Talk to your health care provider or pharmacist
• Visit www.xeomin.com to obtain the FDA-approved product labeling
• Call 1-844-4MYMERZ (1-844-469-6379)

Uses 
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

Warnings

XEOMIN may cause serious side effects that can be life threatening.  Get medical help right away if you have any of these problems any time (hours to weeks) after injection of XEOMIN:

• Problems swallowing, speaking, or breathing can happen if the muscles that you use to breathe and swallow become weak. Death can happen as a complication if you have severe problems with swallowing or breathing after treatment with XEOMIN.
  • People with certain breathing problems may need to use muscles in their neck to help them breathe and may be at greater risk for serious breathing problems with XEOMIN.
  • Swallowing problems may last for several months, and during that time you may need a feeding tube to receive food and water. If swallowing problems are severe, food or liquids may go into your lungs. People who already have swallowing or breathing problems before receiving XEOMIN have the highest risk of getting these problems.
• Spread of toxin effects. In some cases, the effect of botulinum toxin may affect areas of the body away from the injection site and cause symptoms of a serious condition called botulism. The symptoms of botulism include: loss of strength and muscle weakness all over the body, double vision, blurred vision and drooping eyelids, hoarseness or change or loss of voice, trouble saying words clearly, loss of bladder control, trouble breathing, trouble swallowing.

These symptoms can happen hours to weeks after you receive an injection of XEOMIN. These problems could make it unsafe for you to drive a car or do other dangerous activities.

Do not take XEOMIN if you: are allergic to XEOMIN or any of the ingredients in XEOMIN (see below for a list of ingredients in XEOMIN), had an allergic reaction to any other botulinum toxin product such as rimabotulinumtoxinB (Myobloc^®), onabotulinumtoxinA (Botox^®, Botox^® Cosmetic), or abobotulinumtoxinA (Dysport^®) or have a skin infection at the planned injection site.

Before receiving XEOMIN, tell your doctor about all of your medical conditions, including if you:

• have a disease that affects your muscles and nerves (such as amyotrophic lateral sclerosis [ALS or Lou Gehrig’s disease], myasthenia gravis or Lambert-Eaton syndrome)
• have had any side effect from any other botulinum toxin in the past
• have a breathing problem, such as asthma or emphysema
• have a history of swallowing problems or inhaling food or fluid into your lungs (aspiration)
• have drooping eyelids
• have had eye surgery
• have had surgery on your face
• are pregnant or plan to become pregnant. It is not known if XEOMIN can harm your unborn baby.
• are breastfeeding or plan to breastfeed. It is not known if XEOMIN passes into breast milk.

Tell your doctor about all of the medicines you take, including prescription and over-the-counter medicines, vitamins and herbal supplements. Talk to your doctor before you take any new medicines after you receive XEOMIN.

Using XEOMIN with certain other medicines may cause serious side effects. Do not start any new medicines until you have told your doctor that you have received XEOMIN in the past.
Especially tell your doctor if you:

• have received any other botulinum toxin product in the last four months
• have received injections of botulinum toxin such as rimabotulinumtoxinB (MYOBLOC^®), onabotulinumtoxinA (BOTOX^®, BOTOX^® COSMETIC) and abobotulinumtoxinA (DYSPORT^®) in the past.  Be sure your doctor knows exactly which product you received. The dose of XEOMIN may be different from other botulinum toxin products that you have received.
• have recently received an antibiotic by injection or inhalation
• take muscle relaxants
• take an allergy or cold medicine
• take a sleep medicine

Ask your doctor if you are not sure if your medicine is one that is listed above.

Know the medicines you take. Keep a list of your medicines with you to show your doctor and pharmacist each time you get a new medicine.

Possible Side Effects

XEOMIN can cause serious side effects including:

• Injury to the cornea (the clear front surface of the eye) in people treated for blepharospasm. People who receive XEOMIN to treat spasm of the eyelid may have reduced blinking that can cause a sore on their cornea or other problems of the cornea. Call your healthcare provider or get medical care right away if you have eye pain or irritation after treatment with XEOMIN.
• XEOMIN may cause other serious side effects including allergic reactions. Symptoms of an allergic reaction to XEOMIN may include: itching, rash, redness, swelling, wheezing, trouble breathing, or dizziness or feeling faint.  Tell your doctor or get medical help right away if you get wheezing or trouble breathing, or if you get dizzy or faint.

The most common side effects of XEOMIN in adults with chronic sialorrhea include:

The most common side effects of XEOMIN in children 2 to 17 years of age with chronic sialorrhea include:

The most common side effects of XEOMIN in adults with upper limb spasticity include:

The most common side effects of XEOMIN in children 2 to 17 years of age with upper limb spasticity include:

The most common side effects of XEOMIN in adults with cervical dystonia include:

The most common side effects of XEOMIN in adults with blepharospasm include:

These are not all the possible side effects of XEOMIN.
Call your doctor for medical advice about side effects.
You may report side effects to FDA at 1-800-FDA-1088.

General information about the safe and effective use of XEOMIN
Medicines are sometimes prescribed for purposes other than those listed in a Medication Guide. You can ask your pharmacist or doctor for information about XEOMIN that is written for health professionals.

Active Ingredient: botulinum toxin type A

Inactive Ingredients: human albumin and sucrose

Copyright © 2020 Merz Pharmaceuticals, LLC. All rights reserved. MERZ, the MERZ logo, and XEOMIN are registered trademarks of Merz Pharma GmbH & Co. KGaA. Botox, Dysport and Myobloc are registered trademarks of their respective owners.

For more information, please see the XEOMIN full Prescribing Information and Medication Guide at www.xeomin.com.

¹Scielo Brazil Scientific Electronic Library. Sialorrhea in children with cerebral palsy. https://www.scielo.br/scielo.php?script=sci_arttext&pid=S0021-75572016000700549. Accessed December 2020.
²CerebralPalsy.org. Prevalence of Cerebral Palsy. https://www.cerebralpalsy.org/about-cerebral-palsy/prevalence-and-incidence. Accessed December 2020.
³Hockstein, NG, et al. “Sialorrhea: a Management Challenge.” American Family Physician Journal, 1 June 2004, pp. 2628–34. https://www.ncbi.nlm.nih.gov/pubmed/15202698.
⁴Bavikatte, Ganesh, et al. “Management of Drooling of Saliva.” British Journal of Medical Practitioners, vol. 5, no. 1, Mar. 2012, www.bjmp.org/content/management-drooling-saliva.