New Hope For A Rare, Debilitating Disease

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In 2005, Cealie, then in her early 40s, was working a shift as a waitress when she suddenly lost her vision. She went to the hospital where she went through testing, but left without a diagnosis. Her vision partially returned, but several months later, her boss noticed she was dragging her left foot behind her. She spent that Thanksgiving, Christmas and New Year’s Eve in the hospital with recurring attacks of paralysis and muscle spasms.

Finally, she received a difficult diagnosis: neuromyelitis optica spectrum disorder, or NMOSD — a rare, lifelong and debilitating disease of the central nervous system. The disorder, which mainly causes inflammation and damage to the optic nerves and spinal cord, impacts up to 15,000 people in the United States, and while it can affect anyone of any age, race, or gender, it is most commonly diagnosed in women in their 30s and 40s, and occurs at higher rates in people with African or Asian backgrounds.

At the time Cealie was diagnosed, there were no approved treatment options for people like her living with NMOSD. The disease causes relapses, or attacks, which can lead to loss of vision, blindness, fatigue, pain, muscle weakness, inability to walk and paralysis. These symptoms are similar to multiple sclerosis (MS) — people with NMOSD are often misdiagnosed as having MS — but the two diseases are distinct.

For people with NMOSD, the neurological damage can be irreversible. NMOSD symptoms can have a serious impact on a person’s ability to do everyday things. Historically, in just five years from diagnosis, up to half of people with NMOSD required a wheelchair, and more than 60 percent became functionally blind.

After her diagnosis, Cealie, now 60 and living in Waverly, Ohio, experienced a roller coaster of relapses and symptoms. She was hospitalized because of her NMOSD symptoms countless times in just nine years.

“It was terrifying to wake up every morning not knowing whether it would be a good or a bad day,” she said.

The cumulative impact of NMOSD over time makes it critical that people with the condition are accurately diagnosed. Thankfully, there has been progress in recent years, and medicines are now approved by the U.S. Food and Drug Administration (FDA) to specifically treat adults with anti-aquaporin-4 (AQP4) positive NMOSD, and reduce the risk of the relapses that cause permanent neurological damage and disability. AQP4 auto-antibodies are detectable in the blood of around 70-80% of NMOSD patients, and these patients tend to experience a more severe disease course.

One of these newly approved treatments, Enspryng™ (satralizumab-mwge), is the first and only FDA-approved injectable treatment option for adults living with AQP4 antibody positive NMOSD. It can be self-administered every four weeks by a person with NMOSD or a caregiver, after an initial dose and following training from a doctor. The potential for at-home use can be especially important for people during the COVID-19 pandemic.

The treatment is designed to target and inhibit a receptor called interleukin-6 (IL-6), which is involved with a variety of inflammatory processes in the body. Enspryng was studied in two Phase III clinical trials, which showed it reduced the frequency of NMOSD relapses.

Fortunately, Cealie’s neurologist recommended that she participate in one of these clinical trials.

“I was encouraged to see NMOSD research happening that examined the role of the IL-6 receptor,” said Dr. Geoffrey Eubank, Cealie’s neurologist at OhioHealth. “IL-6 is believed to play a key role in spinal cord and optic nerve inflammation associated with NMOSD.”

Cealie was relieved to have an option.

“After almost a decade of pain, set-backs, hospitalizations and no real answers, I was up to try anything,” she said.

Cealie received the medication during the trial, and was happy to find that she had good results. Now that she has found a treatment plan that works for her, Cealie feels like she has been able to move forward with her life. Fortunately, since starting treatment, she hasn’t experienced a relapse. Everyone responds differently to treatment, and people with NMOSD should speak to their doctor about what treatment options might be best for them.

Cealie is now able to focus on her hobbies, including cooking, which she loves, and spending time with her children and grandchildren. “Most importantly, I have hope for my future.”

Learn more about Enspryng and NMOSD.

What is Enspryng?

Enspryng is a prescription medicine used to treat neuromyelitis optica spectrum disorder (NMOSD) in adults who are aquaporin-4 (AQP4) antibody positive.

It is not known if Enspryng is safe and effective in children.

Important Safety Information

Patients should not take Enspryng if they:

• are allergic to satralizumab-mwge or any of the ingredients in Enspryng
• have an active hepatitis B infection
• have active or untreated inactive (latent) tuberculosis (TB)

Enspryng may cause serious side effects including:

Infections. Enspryng can increase risk of serious infections some of which can be life-threatening. Patients should speak with their healthcare provider if they are being treated for an infection and call right away if there are signs of an infection, with or without a fever, such as:

• chills, feeling tired, muscle aches, cough that will not go away or a sore throat
• skin redness, swelling, tenderness, pain or sores on the body
• diarrhea, belly pain, or feeling sick
• burning when urinating or urinating more often than usual
• A healthcare provider will check for infection and treat it if needed before starting or continuing to take Enspryng
• A healthcare provider should test for hepatitis and TB before initiating Enspryng
• All required vaccinations should be completed before starting Enspryng. People using Enspryng should not be given ‘live’ or ‘live-attenuated’ vaccines. ‘Live’ or ‘live-attenuated’ vaccines should be given at least 4 weeks before a patient starts Enspryng. A healthcare provider may recommend that a patient receive a ‘non-live’ (inactivated) vaccine, such as some of the seasonal flu vaccines. If a patient plans to get a ‘non-live’ (inactivated) vaccine it should be given, whenever possible, at least 2 weeks before starting Enspryng

Increased liver enzymes. A healthcare provider should order blood tests to check patient liver enzymes before and while taking Enspryng. A healthcare provider will dictate how often these blood tests are needed. Patients should complete all follow-up blood tests as ordered by a healthcare provider. A healthcare provider may wait to start Enspryng if liver enzymes are increased.

Low neutrophil count. Enspryng can cause a decrease in neutrophil counts in the blood. Neutrophils are white blood cells that help the body fight off bacterial infections. A healthcare provider should order blood tests to check neutrophil counts while a patient is taking Enspryng.

Serious allergic reactions that may be life-threatening have happened with other medicines like Enspryng. Patients should call their healthcare provider right away if they have any of these symptoms of an allergic reaction:

• shortness of breath or trouble breathing
• swelling of lips, face, or tongue
• dizziness or feeling faint
• moderate or severe stomach (abdominal) pain or vomiting
• chest pain

Before taking Enspryng, patients should tell their healthcare provider about all of their medical conditions, including if they

• have or think they have an infection
• have liver problems
• have ever had hepatitis B or are a carrier of the hepatitis B virus
• have had or have been in contact with someone with TB
• have had a recent vaccination or are scheduled to receive any vaccination
• are pregnant, think they might be pregnant, or plan to become pregnant. It is not known if Enspryng will harm one’s unborn baby
• are breastfeeding or plan to breastfeed. It is not known if Enspryng passes into breast milk. Patients should speak with their healthcare provider about the best way to feed one’s baby while on treatment with Enspryng

Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins and herbal supplements.

The most common side effects of Enspryng include:

• sore throat, runny nose (nasopharyngitis)
• headache
• upper respiratory tract infection
• rash
• fatigue
• nausea
• extremity pain
• inflammation of the stomach lining
• joint pain

For more information about the risk and benefit profile of Enspryng, patients should ask their healthcare provider.

Patients may report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch. Patients may also report side effects to Genentech at 1-888-835-2555.

Please see the full Prescribing Information for additional Important Safety Information.