Ultomiris

Approved Conditions

Ultomiris is used to treat the following medical conditions:

• Paroxysmal Nocturnal Hemoglobinuria (PNH): A rare blood disorder where red blood cells are destroyed by the immune system. It is approved for use in adults and children aged 1 month and older.
• Atypical Hemolytic Uremic Syndrome (aHUS): A condition that causes abnormal blood clots in small blood vessels, which can lead to kidney failure and other organ damage. It is used in adults and children aged 1 month and older to prevent complement-mediated thrombotic microangiopathy.
• Generalized Myasthenia Gravis (gMG): A chronic autoimmune disease that causes muscle weakness. Ultomiris is specifically used for adults who are anti-acetylcholine receptor (AChR) antibody-positive.
• Neuromyelitis Optica Spectrum Disorder (NMOSD): A rare autoimmune disease that affects the central nervous system, primarily the optic nerves and spinal cord. It is used for adults who are anti-aquaporin-4 (AQP4) antibody-positive.

This medication is typically administered by a healthcare professional as an intravenous infusion or, in some cases, as a subcutaneous injection for certain conditions.

The Complement System

Ultomiris belongs to a class of medications called complement inhibitors. The complement system is a group of proteins in the blood that form part of the body's natural immune defense. In certain conditions, this system becomes overactive and begins to attack and destroy healthy cells instead of just fighting germs.

Targeting the C5 Protein

This medication works by binding to a specific protein in the complement system known as C5. By attaching to this protein, Ultomiris prevents it from breaking down and triggering a cascade of immune responses that cause damage. This process helps to:

• Prevent red blood cell destruction: In conditions like paroxysmal nocturnal hemoglobinuria (PNH), it stops the immune system from breaking apart red blood cells.
• Protect blood vessels: In atypical hemolytic uremic syndrome (aHUS), it prevents the formation of small blood clots and damage to the lining of blood vessels.
• Reduce nerve and muscle damage: In generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD), it blocks the immune attacks that lead to muscle weakness and inflammation of the central nervous system.

Ultomiris is engineered to remain active in the bloodstream for a long period, providing sustained suppression of the complement system to manage symptoms and prevent disease progression.

Common Side Effects

Many people using Ultomiris experience mild side effects. These may include:

• Headache
• Common cold symptoms, such as a sore throat, runny nose, or cough
• Diarrhea
• Nausea or vomiting
• Back, joint, or muscle pain
• Dizziness
• Feeling tired or weak
• Abdominal pain

Serious Side Effects

Ultomiris can cause serious health problems that require immediate medical care. Contact your doctor right away if you notice:

• Signs of serious infection: Ultomiris affects the immune system and can increase the risk of meningococcal infections. Seek emergency help for fever, headache with a stiff neck or back, confusion, muscle aches with flu-like symptoms, or a skin rash.
• Infusion-related reactions: Some patients may react to the medication during or shortly after the infusion. Symptoms can include difficulty breathing, chest pain, swelling of the face or tongue, or feeling faint.
• High blood pressure: New or worsening symptoms of hypertension may occur during treatment.
• Severe allergic reactions: Signs of anaphylaxis, such as hives, intense itching, or severe swelling.

This is not a complete list of side effects. Always consult with a healthcare professional about any new or worsening symptoms while receiving this medication.

Meningococcal Infections

Ultomiris increases the risk of serious and life-threatening meningococcal infections. These infections can become rapidly fatal if not recognized and treated early. Because of this risk, the medication is only available through a restricted program called the Ultomiris REMS (Risk Evaluation and Mitigation Strategy).

• Vaccination: Patients must be vaccinated against meningococcal disease at least two weeks before receiving the first dose. If urgent treatment is needed and the patient is not up to date on vaccinations, healthcare providers may start the medication and provide antibiotics to reduce infection risk.
• Monitoring: It is important to monitor for early signs of meningitis, such as a high fever, headache with nausea or vomiting, stiff neck, or a rash. Immediate medical attention is necessary if these symptoms occur.

Other Serious Infections

This medication may increase the risk of other types of serious infections, including those caused by Streptococcus pneumoniae and Haemophilus influenzae type b. Healthcare providers typically ensure patients are up to date on vaccinations for these bacteria as well.

Infusion Reactions

Some individuals may experience reactions during or shortly after the intravenous infusion. Symptoms can include lower back pain, abdominal pain, muscle aches, or feeling faint. Healthcare providers monitor patients during the infusion and may slow or stop the treatment if a reaction occurs.

Contraindications

Ultomiris should not be started in individuals who have an active meningococcal infection.

Pregnancy

Data regarding the use of Ultomiris in pregnant women is currently insufficient to determine if there is a drug-associated risk of birth defects or miscarriage. However, it is known that human antibodies can cross the placenta, especially during the later stages of pregnancy.

If you are pregnant or planning to become pregnant, consult your healthcare provider to discuss the necessity of the treatment and any potential risks to the fetus.

Breastfeeding

It is unknown if Ultomiris is present in human breast milk or how it might affect a nursing child. Because many drugs are excreted in breast milk and could cause serious adverse reactions, you should discuss with your doctor whether to continue breastfeeding or stop the medication.

Intravenous Infusion

Ultomiris is supplied as a concentrated solution in single-dose vials. This form is administered by a healthcare professional as an infusion into a vein (intravenous) after being diluted.

Subcutaneous Injection

Ultomiris is also available as a solution in a single-dose prefilled cartridge. This form is administered as an injection under the skin (subcutaneous) using a wearable on-body injector.

FDA Approval and Indications

Ultomiris was first approved by the U.S. Food and Drug Administration (FDA) in December 2018. It is currently approved for the treatment of the following conditions:

• Paroxysmal nocturnal hemoglobinuria (PNH): To treat adults and children one month of age and older.
• Atypical hemolytic uremic syndrome (aHUS): To treat adults and children one month of age and older to inhibit complement-mediated thrombotic microangiopathy.
• Generalized myasthenia gravis (gMG): To treat adults who are anti-acetylcholine receptor (AChR) antibody-positive.
• Neuromyelitis optica spectrum disorder (NMOSD): To treat adults who are anti-aquaporin-4 (AQP4) antibody-positive.

Boxed Warning

Ultomiris carries a boxed warning regarding the risk of serious and life-threatening meningococcal infections. These infections can become fatal if they are not recognized and treated early. Because the medication interferes with the body's immune response, it increases susceptibility to specific types of bacteria.

REMS Program

Due to the risk of meningococcal infections, Ultomiris is only available through a restricted program called the Ultomiris REMS (Risk Evaluation and Mitigation Strategy). Requirements of this program include:

• Healthcare providers who prescribe the medication must be specially certified and enrolled in the program.
• Patients must be vaccinated against meningococcal infection at least two weeks before receiving their first dose, unless the treatment is urgent and cannot be delayed.
• Patients must be monitored for early signs of infection and receive a Patient Safety Card that they must carry at all times during treatment.

Factors Influencing Cost

The total cost of treatment with Ultomiris depends on several variables, including the patient's weight, the specific condition being treated, and the frequency of the maintenance doses. Because it is a specialty medication administered by healthcare professionals, costs also include the fees associated with the infusion site, such as a hospital or specialized clinic.

Insurance Coverage

Ultomiris is typically covered by most insurance plans, including Medicare and private insurance. However, the level of coverage and the resulting out-of-pocket expenses can vary. It is often processed under medical insurance benefits rather than a standard prescription drug plan.

Financial Support Options

Financial assistance may be available for patients who meet certain eligibility criteria. These options often include:

• Co-pay Cards: Programs designed for patients with commercial insurance to help lower out-of-pocket costs.
• Patient Assistance Programs: Support for individuals who are uninsured or underinsured, potentially providing the medication at a reduced cost or no cost.

Patients should consult with their insurance provider and healthcare team to obtain a precise estimate of their treatment costs and to explore available support programs.