ZILBRYSQ® (zilucoplan) Is Now Commercially Available in the U.S. for the Treatment of Generalized Myasthenia Gravis (gMG) in Adult Patients Who Are Anti-Acetylcholine Receptor (AChR) Antibody Positive

• ZILBRYSQ^® (zilucoplan) is now commercially available by prescription in the United States and dispensed by UCB’s selected exclusive specialty pharmacy PANTHERx^® Rare Pharmacy.
• ZILBRYSQ is FDA-approved as the first and only self-administered C5 complement inhibitor indicated for the treatment of gMG in adult patients who are anti-AChR antibody positive.¹
• ZILBRYSQ is available only through a restricted program called ZILBRYSQ REMS (Risk Evaluation and Mitigation Strategy) due to the risk of serious meningococcal infections.¹
• The ZILBRYSQ U.S. launch comes approximately five months after the U.S. launch of RYSTIGGO^® (rozanolixizumab-noli) for the treatment of gMG in adult patients who are anti-AChR or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.^2,3

ATLANTA, Jan. 3, 2024 /PRNewswire/ -- UCB, a global biopharmaceutical company, announced today that ZILBRYSQ^® (zilucoplan) is now available in the U.S. for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. ZILBRYSQ was approved by the U.S. Food and Drug Administration (FDA) on October 17, 2023.¹

ZILBRYSQ is available by prescription as a ready-to-use pre-filled syringe that is a once-daily administration.^1,4 Healthcare providers who prescribe ZILBRYSQ must enroll in the ZILBRYSQ REMS because of the risk of serious meningococcal infections.¹ Further information is available at www.ZILBRYSQREMS.com or 1-877-414-8353. The most common side effects of ZILBRYSQ include injection site reactions, upper respiratory tract infections, and diarrhea.

Myasthenia gravis is a rare, chronic, autoimmune neuromuscular disease, which is characterized by muscle weakness that worsens after periods of activity and improves after periods of rest.^5,6 In the U.S., there are 35,000 to 60,000 people living with myasthenia gravis who experience a variety of symptoms, including drooping eyelids, double vision, and difficulty in swallowing, chewing, and talking.⁷ Exacerbations are unpredictable and are characterized by potentially severe symptoms affecting activities of daily living.⁸

Now that ZILBRYSQ is approved and commercially available, the ONWARD™ program will be in place to provide further care and support for those navigating the complexities of this disease. With UCB’s two FDA-approved targeted treatment options, which have different mechanisms of action and methods of administration, UCB will continue to deliver on our commitment towards differentiated solutions for this community where there are great unmet needs.

UCB is committed toward our goal to enable affordable access to our medicines for all people who need them, in a way that is sustainable for patients, society, and UCB. Affordability and access are always important to the patient experience when talking about rare disease treatment. Based on the differentiated value of ZILBRYSQ as the first and only FDA-approved self-administered C5 complement inhibitor for AChR+ adults, UCB aimed to price ZILBRYSQ to be the lowest cost complement inhibitor treatment for the U.S. gMG population overall.^9,10 Additional affordability information can be found at: https://www.ucb-usa.com/Sustainability/Affordability/ZILBRYSQ-Pricing-Info and www.ZILBRYSQ.com.

ONWARD, a personalized patient support program, will be available to eligible patients and caregivers to provide support throughout their prescribed treatment with medicines within the UCB Rare Disease Portfolio, including ZILBRYSQ and RYSTIGGO.^* Through ONWARD, eligible patients will have access to important resources, including a dedicated Care Coordinator† who will provide personalized support, tools, and resources to get started on treatment. This support includes refresher injection coaching and information on symptom tracking and ongoing treatment support, as well as help in understanding insurance coverage. If an eligible patient or caregiver is looking for support, please visit http://www.UCBONWARD.com or call 1-844-ONWARD.

About ZILBRYSQ (zilucoplan)
ZILBRYSQ is a prescription medicine that is used to treat adults with a disease called generalized myasthenia gravis in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. It is injected under the skin.¹

About RYSTIGGO (rozanolixizumab-noli)
RYSTIGGO is a prescription medicine used to treat adults with a disease called generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.³

Important Safety Information for ZILBRYSQ^®

IMPORTANT SAFETY INFORMATION INCLUDING BOXED WARNING

What is the most important information I should know about ZILBRYSQ?
ZILBRYSQ is a medicine that affects part of your immune system. ZILBRYSQ may lower the ability of your immune system to fight certain infections.

• ZILBRYSQ increases your chance of getting serious and life-threatening meningococcal infections. Meningococcal infections may become life-threatening or fatal if not recognized and treated early.
  • You must complete or update two types of meningococcal vaccines (for both serogroup B infections and serogroup A, C, W, and Y infections) at least 2 weeks before your first dose of ZILBRYSQ if you have not already had these vaccines.
  • If your healthcare provider decided that urgent treatment with ZILBRYSQ is needed, you should receive meningococcal vaccination(s) as soon as possible.
  • If you have not completed or updated vaccinations for meningococcal infections at least 2 weeks before your first ZILBRYSQ dose and ZILBRYSQ therapy must be started right away, you must also receive antibiotics.
  • If you had a meningococcal vaccine in the past, you might need additional vaccination before starting ZILBRYSQ. Your healthcare provider will decide if you need additional meningococcal vaccination.
  • Meningococcal vaccines do not prevent all meningococcal infections. Call your healthcare provider or get emergency medical care right away if you get any of these signs and symptoms of a meningococcal infection:

Your healthcare provider will give you a Patient Safety Card about the risk of meningococcal infection. Carry it with you at all times during treatment and for 2 months after your last ZILBRYSQ dose. Your risk of meningococcal infection may continue for several weeks after your last dose of ZILBRYSQ. It is important to show this card to any healthcare provider who treats you. This will help them diagnose and treat you quickly.

ZILBRYSQ is only available through a program called the ZILBRYSQ REMS. Before you can receive ZILBRYSQ, your healthcare provider must:

• enroll in the ZILBRYSQ REMS.
• counsel you about the risk of meningococcal infection.
• give you the Patient Guide, including information about the signs and symptoms of meningococcal infection.
• give you a Patient Safety Card about your risk of meningococcal infection, as discussed above.
• make sure that you are vaccinated with two types of meningococcal vaccines and, if needed, get revaccinated with the meningococcal vaccines. Ask your healthcare provider if you are not sure if you need to be revaccinated.

ZILBRYSQ may also increase the risk of other types of serious infections.

• ZILBRYSQ may increase your chance of getting Streptococcus pneumoniae and Haemophilus influenzae type b. Your healthcare provider will tell you if you should receive the Streptococcus pneumoniae and Haemophilus influenzae type b vaccinations.
• Certain people may have an increased risk of gonorrhea infection. Talk to your healthcare provider about whether you are at risk for gonorrhea infection, about gonorrhea prevention, and about regular testing.

Call your healthcare provider right away if you have new signs or symptoms of infection.

Who should not use ZILBRYSQ?
Do not use ZILBRYSQ if you have a Neisseria meningitidis infection.

Before you use ZILBRYSQ, tell your healthcare provider about all of your medical conditions, including if you:

• have an infection or fever.
• are pregnant or plan to become pregnant. It is not known if ZILBRYSQ will harm your unborn baby.
• are breastfeeding or plan to breastfeed. It is not known if ZILBRYSQ passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby if you use ZILBRYSQ.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What are the possible side effects of ZILBRYSQ?

ZILBRYSQ may cause serious side effects, including:

• See “What is the most important information I should know about ZILBRYSQ?”
• Inflammation of the pancreas (pancreatitis) and other pancreatic problems. Pancreatitis and pancreatic cysts have happened in people who use ZILBRYSQ. Your healthcare provider will do blood tests to check your pancreas before you start treatment with ZILBRYSQ.
• Call your healthcare provider right away if you have pain in your stomach area (abdomen) that will not go away. Your healthcare provider will tell you if you should stop using ZILBRYSQ. The pain may be severe or felt going from your abdomen to your back. The pain may happen with or without vomiting. These may be symptoms of pancreatitis.

The most common side effects of ZILBRYSQ include:

• injection site reactions.
• upper respiratory tract infections.
• diarrhea.

Tell your healthcare provider about any side effect that bothers you or that does not go away. These are not all the possible side effects of ZILBRYSQ. For more information, ask your healthcare provider or pharmacist. Call your doctor for medical advice about side effects. You may report side effects to FDA at www.fda.gov/medwatch or 1-800-FDA-1088. You may also report side effects to UCB, Inc. by calling 1-844-599-CARE [2273].

See the detailed Instructions for Use that comes with ZILBRYSQ for information on how to prepare and inject a dose of ZILBRYSQ, and how to properly throw away (dispose of) used ZILBRYSQ prefilled syringes.

INDICATION

What is ZILBRYSQ?

• ZILBRYSQ is a prescription medicine used to treat adults with a disease called generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive.
• It is not known if ZILBRYSQ is safe and effective in children.

Please see the full Prescribing Information and Medication Guide for ZILBRYSQ, including Boxed Warning regarding serious meningococcal infections. Please see the Instructions for Use for the ZILBRYSQ Single-Dose Prefilled Syringe. Talk to your healthcare provider about your condition or your treatment. For more information, go to www.ZILBRYSQ.com or call 1-844-599-2273.

Important Safety Information for RYSTIGGO^®

What is RYSTIGGO?
RYSTIGGO is a prescription medicine used to treat adults with a disease called generalized myasthenia gravis (gMG) who are acetylcholine receptor (anti-AChR) antibody positive or muscle-specific tyrosine kinase (anti-MuSK) antibody positive.

What is the most important information I should know about RYSTIGGO (rozanolixizumab-noli)?

RYSTIGGO may cause serious side effects, including:

• Infection: RYSTIGGO may increase the risk of infection. In clinical studies, the most common infections were upper respiratory tract infections, COVID-19, urinary tract infections, and herpes simplex infections. Your healthcare provider should check you for infections before starting and during treatment with RYSTIGGO. Tell your healthcare provider if you have any history of infections. Tell your healthcare provider right away if you have signs or symptoms of an infection during treatment with RYSTIGGO. Some of the signs and symptoms may include fever, chills, frequent and/or painful urination, cough, runny nose, wheezing, shortness of breath, fatigue, sore throat, excess phlegm, nasal discharge, back pain, and/or chest pain.
• Aseptic Meningitis: RYSTIGGO could cause aseptic meningitis. Tell your healthcare provider right away if you develop any signs or symptoms of meningitis during treatment with RYSTIGGO such as severe headache, neck stiffness, drowsiness, fever, sensitivity to light, painful eye movements, nausea, and vomiting.
• Hypersensitivity Reactions: RYSTIGGO can cause swelling and rash. Your healthcare provider should monitor you during and after treatment and discontinue RYSTIGGO if needed. Tell your healthcare provider immediately about any undesirable reactions you experience after administration.

Before taking RYSTIGGO, tell your healthcare provider about all of your medical conditions, including if you:

• Have a history of infection or think you have an active infection.
• Have received or are scheduled to receive a vaccine (immunization). The use of vaccines during RYSTIGGO treatment has not been studied, and the safety with live or live-attenuated vaccines is unknown. Administration of live or live-attenuated vaccines is not recommended during treatment with RYSTIGGO. Completion of age-appropriate vaccines according to vaccination guidelines before starting a new treatment cycle with RYSTIGGO is recommended.
• Are pregnant or plan to become pregnant or are breastfeeding or plan to breastfeed.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What are the possible side effects of RYSTIGGO?

RYSTIGGO may cause serious side effects, including:

• See “What is the most important information I should know about RYSTIGGO?”

The most common side effects of RYSTIGGO include:

• headache
• infections
• diarrhea
• fever
• hypersensitivity reactions
• nausea

These are not all the possible side effects of RYSTIGGO. For more information, ask your healthcare provider or pharmacist. Tell your healthcare provider about any side effect that bothers you or that does not go away. Call your healthcare provider for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to UCB, Inc. by calling 1-844-599-CARE [2273].

Please see the full Prescribing Information and talk to your healthcare provider about your condition or your treatment. For more information, go to www.RYSTIGGO.com or call 1-844-599-2273.

For further information, contact UCB:

U.S. Communications, Rare Diseases
Daphne Teo
T +1.770.880.7655
daphne.teo@ucb.com

Brand Communications, Rare Diseases
Jim Baxter
T+32.2.473.78.85.01
jim.baxter@ucb.com

Corporate Communications, Media Relations
Laurent Schots
T+32.2.559.92.64
Laurent.schots@ucb.com

Investor Relations
Antje Witte
T +32.2.559.94.14
antje.witte@ucb.com

About UCB
UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With approximately 8,700 people in approximately 40 countries, the company generated revenue of €5.5 billion in 2022. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCBUSA.

Forward looking statements
This press release may contain forward-looking statements including, without limitation, statements containing the words “believes”, “anticipates”, “expects”, “intends”, “plans”, “seeks”, “estimates”, “may”, “will”, “continue” and similar expressions. These forward-looking statements are based on current plans, estimates and beliefs of management. All statements, other than statements of historical facts, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial information, expected legal, arbitration, political, regulatory or clinical results or practices and other such estimates and results. By their nature, such forward-looking statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties and assumptions which might cause the actual results, financial condition, performance or achievements of UCB, or industry results, to differ materially from those that may be expressed or implied by such forward-looking statements contained in this press release. Important factors that could result in such differences include: the global spread and impact of COVID-19, changes in general economic, business and competitive conditions, the inability to obtain necessary regulatory approvals or to obtain them on acceptable terms or within expected timing, costs associated with research and development, changes in the prospects for products in the pipeline or under development by UCB, effects of future judicial decisions or governmental investigations, safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, product liability claims, challenges to patent protection for products or product candidates, competition from other products including biosimilars, changes in laws or regulations, exchange rate fluctuations, changes or uncertainties in tax laws or the administration of such laws, and hiring and retention of its employees. There is no guarantee that new product candidates will be discovered or identified in the pipeline, will progress to product approval or that new indications for existing products will be developed and approved. Movement from concept to commercial product is uncertain; preclinical results do not guarantee safety and efficacy of product candidates in humans. So far, the complexity of the human body cannot be reproduced in computer models, cell culture systems or animal models. The length of the timing to complete clinical trials and to get regulatory approval for product marketing has varied in the past and UCB expects similar unpredictability going forward. Products or potential products, which are the subject of partnerships, joint ventures or licensing collaborations may be subject to differences disputes between the partners or may prove to be not as safe, effective or commercially successful as UCB may have believed at the start of such partnership. UCB’s efforts to acquire other products or companies and to integrate the operations of such acquired companies may not be as successful as UCB may have believed at the moment of acquisition. Also, UCB or others could discover safety, side effects or manufacturing problems with its products and/or devices after they are marketed. The discovery of significant problems with a product similar to one of UCB’s products that implicate an entire class of products may have a material adverse effect on sales of the entire class of affected products. Moreover, sales may be impacted by international and domestic trends toward managed care and health care cost containment, including pricing pressure, political and public scrutiny, customer and prescriber patterns or practices, and the reimbursement policies imposed by third-party payers as well as legislation affecting biopharmaceutical pricing and reimbursement activities and outcomes. Finally, a breakdown, cyberattack or information security breach could compromise the confidentiality, integrity and availability of UCB’s data and systems.

Given these uncertainties, you should not place undue reliance on any of such forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labelling in any market, or at any particular time, nor can there be any guarantee that such products will be or will continue to be commercially successful in the future.

UCB is providing this information, including forward-looking statements, only as of the date of this press release and it does not reflect any potential impact from the evolving COVID-19 pandemic, unless indicated otherwise. UCB is following the worldwide developments diligently to assess the financial significance of this pandemic to UCB. UCB expressly disclaims any duty to update any information contained in this press release, either to confirm the actual results or to report or reflect any change in its forward-looking statements with regard thereto or any change in events, conditions or circumstances on which any such statement is based, unless such statement is required pursuant to applicable laws and regulations.

Additionally, information contained in this document shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any offer, solicitation or sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such jurisdiction.

References:

1. ZILBRYSQ^® U.S. Prescribing Information.
2. UCB press release, July 21, 2023. https://www.ucb-usa.com/stories-media/UCB-U-S-News/detail/article/ucb-announces-us-availability-of-rystiggo-rozanolixizumab-noli-for-the-treatment-of-generalized-myasthenia-gravis-gmg-in-adult-patients-who-are-anti-acetylcholine-receptor-achr-or-anti-muscle-specific-tyrosine
3. RYSTIGGO^® U.S. Prescribing Information.
4. Howard JF Jr, Bresch S., Genge A, et al. RAISE Study Team. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravies (RAISE)” a randomised, double-blind, placebo-controlled, phase 3 study. Lancet Neurol. 2023;22(5):395-406
5. National Institute of Neurological Disorders and Stroke. What is Myasthenia Gravis. www.ninds.nih.gov/health-information/disorders/myasthenia-gravis. Accessed December 2023.
6. Punga AR, Maddison P, Heckmann JM, Guptill JT, Evoli A. Epidemiology, diagnostics, and biomarkers of autoimmune neuromuscular junction disorders. Lancet Neurol. 2022;21(2):176-188. doi:10.1016/S1474-4422(21)00297-0
7. Howard, James F. Myasthenia Gravis Foundation of America. Clinical Overview of MG. https://myasthenia.org/Professionals/Clinical-Overview-of-MG. Accessed December 2023
8. Bril V. Efficacy and safety of rozanolixizumab in patients with generalised myasthenia gravis: a randomised, double-blind, placebo-controlled, adaptive Phase 3 study MyCarinG study. Lancet Neurol. 2023;22(5):383-94.
9. Mahic M, Bozorg AM, DeCourcy JJ, et al. Physician-reported perspectives on myasthenia gravis in the United States: a real- world survey. Neurol Ther. 2022;11:1535-1551
10. Millman White Paper. Drug and administration costs in the commercial market for generalized Myasthenia Gravis pharmaceutical therapies, Commissioned by UCB. https://in.milliman.com/-/media/milliman/pdfs/2023-articles/11-16-23_gmg-treatment-cost-analysis.ashx. Accessed December 2023.

*ONWARD is provided as a service of UCB and is intended to support the appropriate use of UCB medicines; ONWARD may be amended or canceled at any time without notice. Some program and eligibility restrictions may apply.
^†ONWARD Care Coordinators do not provide medical advice and will refer patients to their healthcare professional for any questions related to their treatment plan.

RYSTIGGO^® and ZILBRYSQ^® are registered trademarks, and ONWARD^TM is a trademark, of the UCB Group of Companies.

©2024 UCB, Inc., Smyrna, GA 30080. All rights reserved. US-P-ZL-MG-230014

View original content:https://www.prnewswire.com/news-releases/zilbrysq-zilucoplan-is-now-commercially-available-in-the-us-for-the-treatment-of-generalized-myasthenia-gravis-gmg-in-adult-patients-who-are-anti-acetylcholine-receptor-achr-antibody-positive-302025019.html

SOURCE UCB