Soliris

Conditions Treated

Soliris is approved for the treatment of the following rare conditions:

• Paroxysmal nocturnal hemoglobinuria (PNH): A blood disorder where the immune system destroys red blood cells. Soliris helps reduce this destruction and the associated risk of blood clots.
• Atypical hemolytic uremic syndrome (aHUS): A condition that causes abnormal blood clots in small blood vessels, which can lead to kidney failure and damage to other organs.
• Generalized myasthenia gravis (gMG): A chronic autoimmune disease that causes muscle weakness. Soliris is used in adults who are anti-acetylcholine receptor (AChR) antibody-positive.
• Neuromyelitis optica spectrum disorder (NMOSD): An autoimmune disease of the central nervous system that affects the optic nerves and spinal cord. It is used in adults who are anti-aquaporin-4 (AQP4) antibody-positive.

This medication is intended to help manage symptoms and prevent complications associated with these specific disorders. It is not used to treat Shiga toxin-producing E. coli-related hemolytic uremic syndrome (STEC-HUS).

The Complement System

Soliris belongs to a class of medications known as complement inhibitors. The complement system is a group of proteins in the blood that are part of the body's natural immune response. While these proteins normally help fight off bacteria and viruses, in certain rare diseases, the system becomes overactive and begins attacking the body's own healthy cells.

Targeting the C5 Protein

The active ingredient in Soliris, eculizumab, is a laboratory-made protein called a monoclonal antibody. It is designed to target and bind to a specific protein in the complement system known as C5. By attaching to C5, Soliris prevents it from breaking down into smaller pieces that would otherwise trigger the final, destructive stage of the complement cascade.

How It Helps Specific Conditions

By blocking the complement pathway, Soliris helps manage different diseases in the following ways:

• Paroxysmal Nocturnal Hemoglobinuria (PNH): It stops the immune system from rupturing red blood cells, which reduces the need for blood transfusions and lowers the risk of blood clots.
• Atypical Hemolytic Uremic Syndrome (aHUS): It prevents the immune system from damaging small blood vessels, which helps protect vital organs like the kidneys from blood clots and inflammation.
• Generalized Myasthenia Gravis (gMG) and Neuromyelitis Optica Spectrum Disorder (NMOSD): It prevents the complement system from attacking the junctions between nerves and muscles or the central nervous system, helping to reduce muscle weakness and neurological damage.

Common Side Effects

The most frequently reported side effects of Soliris are often mild and may include:

• Headache
• Nausea or vomiting
• Diarrhea or stomach pain
• Joint, muscle, or back pain
• Dizziness or fatigue
• Cold symptoms such as a sore throat, cough, or stuffy nose
• Swelling in the arms or legs

Serious Side Effects

Soliris can cause severe or life-threatening side effects. Contact a healthcare provider immediately if you experience any of the following:

Meningococcal Infection

This is a rare but very serious infection of the lining of the brain and spinal cord or the blood. Symptoms include:

• High fever and severe headache
• Stiff neck or back
• Confusion
• Nausea and vomiting
• Sensitivity to light
• A rash

Other Serious Concerns

• Infusion-related reactions: Symptoms like chest pain, shortness of breath, or swelling of the face and throat can occur during the injection.
• High blood pressure: Some patients experience an increase in blood pressure during treatment.
• Blood cell changes: Soliris may cause a decrease in red blood cells or other blood-related issues.
• Kidney problems: Monitoring is often required to ensure the kidneys are functioning correctly during and after treatment.

Meningococcal Infection Warning

Soliris increases the risk of serious, life-threatening, or fatal meningococcal infections. These infections can progress rapidly if not recognized and treated early. Because of this risk, the medication is only available through a restricted program called the Soliris REMS.

• Vaccination: Patients must be vaccinated against meningococcal infections at least two weeks before receiving the first dose. If urgent treatment is needed and the patient is not vaccinated, antibacterial drugs should be given immediately and continued until two weeks after vaccination.
• Monitoring: Healthcare providers monitor patients for early signs of meningitis, such as headache with nausea or vomiting, fever, stiff neck, or skin rash.

Other Serious Infections

The medication may increase susceptibility to other infections, particularly those caused by encapsulated bacteria like Streptococcus pneumoniae and Haemophilus influenzae. Patients should be up to date on all recommended vaccinations before starting therapy.

Infusion-Related Reactions

Administration of Soliris can cause infusion reactions. If a reaction occurs, the infusion may be slowed or stopped depending on the severity of the reaction.

Stopping Treatment

Discontinuing Soliris can lead to serious complications depending on the condition being treated. For patients with paroxysmal nocturnal hemoglobinuria (PNH), stopping treatment may cause the destruction of red blood cells (hemolysis). For those with atypical hemolytic uremic syndrome (aHUS), stopping treatment may lead to blood clotting in small vessels (thrombotic microangiopathy). Monitoring is required for several weeks after the last dose.

Pregnancy

Data on the use of Soliris in pregnant women are limited. While animal studies using similar molecules have not shown a high risk of harm to the developing fetus, there is not enough evidence to confirm its safety in humans. Because Soliris is a monoclonal antibody, it may cross the placenta and potentially affect the fetus.

If you are pregnant or planning to become pregnant, your doctor will help you weigh the risks of the untreated condition against the potential risks of the medication. It is generally recommended to use Soliris during pregnancy only if the benefits clearly outweigh the potential risks to the fetus.

Breastfeeding

It is currently unknown whether Soliris passes into human breast milk. Because many medications can be excreted in milk and have the potential to cause side effects in a nursing infant, caution is advised.

Healthcare providers typically recommend deciding between breastfeeding or continuing treatment with Soliris based on the mother's medical need for the drug. You should consult with your doctor to determine the best course of action for your specific situation.

Dosage Forms

Soliris is supplied in the following form:

• Intravenous Solution: A concentrated, preservative-free liquid provided in single-dose vials for administration into a vein.

This medication is not available in oral forms such as tablets or capsules. It must be prepared and diluted by a healthcare professional before it is administered as an infusion.

FDA Approval Status

Soliris (eculizumab) received its initial FDA approval in March 2007. It is currently approved for the treatment of the following conditions:

• Paroxysmal nocturnal hemoglobinuria (PNH)
• Atypical hemolytic uremic syndrome (aHUS)
• Generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) antibody-positive
• Neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4 (AQP4) antibody-positive

Boxed Warning

The FDA has issued a boxed warning for Soliris due to the increased risk of serious and life-threatening meningococcal infections. These infections can progress rapidly and may become fatal if not recognized and treated early.

Risk Evaluation and Mitigation Strategy (REMS)

Because of the risk of meningococcal infection, Soliris is only available through a restricted distribution program called the Soliris REMS. Regulatory requirements for this program include:

• Prescriber Certification: Healthcare providers who prescribe Soliris must be enrolled and certified in the REMS program.
• Vaccination: Patients must be vaccinated against meningococcal disease at least two weeks before receiving the first dose. If the treatment must start immediately, the patient must receive appropriate preventive antibiotics.
• Patient Education: Patients must be informed of the risks and provided with a Medication Guide and a Patient Safety Card, which they are instructed to carry at all times during treatment and for three months after the last dose.

Factors Influencing Cost

The price of Soliris can vary significantly depending on several factors, including your insurance plan, the dosage required for your condition, and where you receive your infusions. Because it is a specialty drug administered by a healthcare professional, costs typically include both the medication itself and the clinical fees for the infusion procedure.

Insurance Coverage

Most private insurance plans and government programs like Medicare or Medicaid provide coverage for Soliris, but they often require prior authorization. This means your doctor must prove the medication is medically necessary before the insurance company will agree to pay for it. Patients may still be responsible for deductibles, copays, or coinsurance amounts.

Financial Assistance Programs

The manufacturer offers a patient support program that provides personalized assistance, including help navigating insurance hurdles and identifying potential financial aid options for eligible patients. There may also be independent charitable foundations that offer grants to help cover treatment costs for specific rare diseases.

Biosimilar Options

Lower-cost versions of eculizumab, known as biosimilars, may be available. These medications are highly similar to Soliris and are intended to provide the same clinical benefits at a potentially lower price point. You should discuss with your healthcare provider or insurance company if a biosimilar is an option for your treatment plan.