FDA approves first pediatric indication for XEOMIN^® (incobotulinumtoxinA) for the treatment of upper limb spasticity, excluding spasticity caused by cerebral palsy

• Designed to offer doctors and patients ultimate flexibility, XEOMIN is the first and only botulinum toxin approved for treatment in 12 upper limb muscles
• XEOMIN was also granted FDA Priority Review for an application for pediatric sialorrhea, reinforcing Merz Therapeutics’ commitment to expanding options for pediatric patients

RALEIGH, N.C.  –  August 19, 2020 – Merz Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has approved a supplemental Biologics License Application (sBLA) for the first pediatric indication for XEOMIN^® (incobotulinumtoxinA), for the treatment of patients aged 2 years and older with upper limb spasticity, excluding spasticity caused by cerebral palsy. The FDA also granted XEOMIN Priority Review for its sBLA for the treatment of pediatric patients with sialorrhea with an expected decision this December.

The pediatric upper limb spasticity approval is based on a Phase 3, randomized, double-blind, multi-center dose-response study evaluating the safety and efficacy of XEOMIN in 350 patients aged 2-17 years with spasticity primarily caused by cerebral palsy. The primary efficacy endpoints were defined as the change from baseline on the five-point Ashworth Scale (AS) and a seven-point Investigator’s Global Impression of Change Scale (GICS) at week four of treatment. XEOMIN demonstrated sustained improvements as measured by the AS and GICS in muscle tone for all upper limb muscle groups studied over 12-16 weeks, including flexed elbow, flexed wrist, forearm pronators, clenched fist, and thumb-in-palm. The study also confirmed XEOMIN is safe and effective for the bilateral treatment of upper limb spasticity with up to 400 units per treatment. In more than 900 pediatric spasticity patients treated with XEOMIN in clinical trials, none developed clinical resistance to XEOMIN due to neutralizing antibodies, confirming its unique purifying process through XTRACT Technology™. *

“With the need for prolonged treatment, it is critical for children and adolescents with upper limb spasticity to have a long-term flexible treatment option to help manage their condition over time. At Merz Therapeutics, we designed our clinical trial program with 12 upper limb muscles to offer the most flexibility of any botulinum toxin, in order to offer patients, caregivers and doctors a safe and effective treatment option that meets the needs of pediatric patients,” said Kevin O’Brien, President of North America, Merz Therapeutics. “This approval, along with our Priority Review for pediatric sialorrhea, reinforces our commitment to putting patients first and addressing the unmet needs of those living with movement disorders – now including children and adolescents.”

Spasticity is a condition in which the voluntary movement of muscles is disrupted and may be associated with discomfort or pain.¹ In upper limb spasticity, the muscles of the arms and neck remain stiff and contracted, and resist stretching. Spasticity can result following injury to the brain or spinal cord, particularly in areas which control stretching and reflexes.^2,3 In growing children, spasticity can be especially harmful as it can affect muscles and joints.¹

The safety findings were in line with previously conducted adult studies and with the known safety profile of XEOMIN. There was also no evidence that higher dosing caused increased incidence of adverse reactions.

A pediatric assessment for XEOMIN demonstrates that XEOMIN is safe and effective in cerebral palsy patients. However, XEOMIN is not approved for this specific patient population due to marketing exclusivity for another botulinum toxin.

Merz Therapeutics recently celebrated 10 years since XEOMIN’s first therapeutic approval by the FDA. With this most recent approval, XEOMIN now holds five first-line indications in the U.S. and has helped more than three million patients worldwide. 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

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.

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

Uses
XEOMIN is a prescription medicine used in adults:

• that is injected into glands that make saliva and is used to treat long-lasting (chronic) drooling (sialorrhea).
• that is injected into muscles and used to:
  • treat increased muscle stiffness in the arm because of upper limb spasticity.
  • treat the abnormal head position and neck pain with cervical dystonia (CD).
  • treat abnormal spasm of the eyelids (blepharospasm) in adults.

It is not known if XEOMIN is safe and effective in children under 18 years of age.

Warnings
XEOMIN may cause serious side effects that can be life threatening. Call your doctor or get medical help right away if you have any of these problems anytime (hours to weeks) after treatment with XEOMIN:

• Problems with swallowing, speaking, or breathing can happen within hours to weeks after an injection of XEOMIN 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 the end of this Guide for a list of ingredients in XEOMIN), had an allergic reaction to any other botulinum toxin product such as rimabotulinumtoxinB (MYBLOC^®), 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 bleeding problems
• have drooping eyelids
• have plans to have 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
• 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:
See “Warnings.”

• 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 people with chronic sialorrhea include:

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

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

The most common side effects of XEOMIN in people 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.

* 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.

¹ American Association of Neurological Surgeons. Spasticity page. http://www.aans.org/Patients/Neurosurgical-Conditions-and-Treatments/Spasticity.
² Tsao C, et al. J Neuroeng Rehabil. 2007; 4:45.