Ultomiris

Ultomiris is a prescription medication used to treat rare blood disorders including Paroxysmal Nocturnal Hemoglobinuria (PNH) and atypical Hemolytic Uremic Syndrome (aHUS). These diseases are characterized by destruction of red blood cells and low levels of other blood components.

Ultomiris, a complement inhibitor, belongs to a group of drugs called humanized monoclonal antibodies. Ultomiris works on certain proteins to prevent the destruction of red blood cells.

This medication is available in an injectable form to be given directly into a vein by intravenous (I.V.) infusion. The infusion usually lasts about 2 hours in adults and up to 4 hours in children.

Common side effects of Ultomiris include upper respiratory infections, headache, diarrhea, and nausea.

Ultomiris is used to treat:

• adults with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). 
• adults and children 1 month of age and older with a disease called atypical Hemolytic Uremic Syndrome (aHUS). Ultomiris is not used in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS). 

It is not known if Ultomiris is safe and effective in children with PNH.
It is not known if Ultomiris is safe and effective in children younger than 1 month of age.

This medication may be prescribed for other uses. Ask your doctor or pharmacist for more information.

Serious side effects have been reported with Ultomiris. See the “Ultomiris Precautions” section.

• Infusion reactions. Infusion reactions may happen during your Ultomiris infusion. Symptoms of an infusion reaction with Ultomiris may include lower back pain, pain with the infusion, feeling faint or discomfort in your arms or legs. Tell your doctor or nurse right away if you develop these symptoms, or any other symptoms during your Ultomiris infusion that may mean you are having a serious infusion reaction, including:
  • chest pain
  • trouble breathing or shortness of breath
  • swelling of your face, tongue, or throat
  • feel faint or pass out

Your doctor will treat your symptoms as needed.

The most common side effects of Ultomiris in people treated for PNH are upper respiratory infection and headache.

The most common side effects of Ultomiris in people with aHUS are:

• upper respiratory infections
• diarrhea
• nausea
• vomiting
• headache
• high blood pressure
• fever

Tell your doctor about any side effect that bothers you or that does not go away. These are not all the possible side effects of Ultomiris. For more information, ask your doctor or pharmacist.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

No drug interactions have been determined by the manufacturer. However, tell your doctor about all the medicines you take including prescription and non-prescription medicines, vitamins, and herbal supplements. Not all drug interactions are known or reported and new drug interactions are continually being reported.

Serious side effects have been reported with Ultomiris including the following: 

Ultomiris increases your chance of getting serious and life-threatening meningococcal infections. Meningococcal infections may quickly become life-threatening and cause death if not recognized and treated early.

• You must receive meningococcal vaccines at least 2 weeks before your first dose of Ultomiris if you have not already had this vaccine.
• If your doctor decided that urgent treatment with Ultomiris is needed, you should receive meningococcal vaccination as soon as possible.
• If you have not been vaccinated and Ultomiris therapy must be initiated immediately, you should also receive 2 weeks of antibiotics with your vaccinations.
• If you had a meningococcal vaccine in the past, you might need additional vaccination before starting Ultomiris. Your doctor will decide if you need additional meningococcal vaccination.
• Meningococcal vaccines reduce the risk of meningococcal infection but do not prevent all meningococcal infections. Call your doctor or get emergency medical care right away if you get any of these signs and symptoms of a meningococcal infection:
  • headache with nausea or vomiting
  • headache and fever
  • headache with a stiff neck or stiff back
  • fever
  • fever and a rash
  • confusion
  • muscle aches with flu-like symptoms
  • eyes sensitive to light

Your doctor will give you a Patient Safety Card about the risk of meningococcal infection. Carry it with you at all times during treatment and for 8 months after your last Ultomiris dose. Your risk of meningococcal infection may continue for several months after your last dose of Ultomiris. It is important to show this card to any doctor or nurse who treats you. This will help them diagnose and treat you quickly. 

Ultomiris is only available through a program called the Ultomiris REMS. Before you can receive Ultomiris, your doctor must:

• enroll in the Ultomiris REMS program
• counsel you about the risk of meningococcal infection
• give you information about the 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 a meningococcal vaccine

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

• People who take Ultomiris may have an increased risk of getting infections caused by Streptococcus pneumoniae and Haemophilus influenzae.
• Certain people may also have an increased risk of gonorrhea infection. Talk to your doctor to find out if you are at risk for gonorrhea infection, about gonorrhea prevention, and regular testing.

Call your doctor right away if you have any new signs or symptoms of infection.

Do not receive Ultomiris if you:

• have a meningococcal infection
• Have not been vaccinated against meningococcal infection unless your doctor decides that urgent treatment with Ultomiris is needed.

Medications can interact with certain foods. In some cases, this may be harmful and your doctor may advise you to avoid certain foods. In the case of Ultomiris, there are no specific foods that you must exclude from your diet when receiving this medication.

Before taking Ultomiris, tell your doctor about all of your medical conditions. Especially tell your doctor if you:

• are allergic to Ultomiris or to any of its ingredients

• if you have a meningococcal infection (bloodstream or brain infections from meningococcal organisms) or any other infection

• are pregnant or plan to become pregnant

• are breastfeeding or plan to breastfeed

Tell your doctor about all the medicines you take including prescription and non-prescription medicines, vitamins, and herbal supplements.

Tell your doctor if you are pregnant or plan to become pregnant.

There are no well-controlled studies that have been done in pregnant women. Animal studies have shown a higher rate of developmental abnormalities and a higher rate of dead offspring. Patients who are pregnant or who may become pregnant should talk to their doctor before starting on this medication.

Tell your doctor if you are breastfeeding or plan to breastfeed.

It is not known if Ultomiris crosses into human milk. However, products similar to Ultomiris are secreted into human milk. Because of the potential for adverse effects, breastfeeding should be stopped during treatment and 8 months after the medication has been completed.

• Ultomiris is given through a vein by intravenous (I.V.) infusion usually over about 2 hours in adults and up to 4 hours in children. 
• If you are an adult with PNH or aHUS, you will usually receive:
  • a starting dose of Ultomiris as an infusion by your doctor, and then
  • 2 weeks later, you will start to receive an infusion of Ultomiris every 8 weeks.
• Children 1 month of age and older with aHUS will usually receive:
  • a starting dose of Ultomiris as an infusion by your doctor, and then
  • your doctor will decide how often your child will receive Ultomiris, either every 4 weeks or every 8 weeks, depending on their weight, starting 2 weeks after the starting dose.
• Your doctor will decide how long you need to take Ultomiris for your aHUS.
• If you are changing treatment from Soliris to Ultomiris, you should receive your starting dose of Ultomiris 2 weeks after your last dose of Soliris.
• After each infusion, you should be monitored for at least 1 hour for infusion reactions. 
• If you have PNH and you stop receiving Ultomiris, your doctor will need to monitor you closely for at least 16 weeks after you stop Ultomiris. Stopping Ultomiris may cause breakdown of your red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: 
  • drop in your red blood cell count
  • tiredness
  • blood in your urine
  • stomach-area (abdomen) pain
  • shortness of breath
  • blood clots
  • trouble swallowing
  • erectile dysfunction (ED) in males
• If you have aHUS, your doctor will need to monitor you closely for at least 12 months after stopping treatment for signs of worsening aHUS symptoms or problems related to a type of abnormal clotting and breakdown of your red blood cells called thrombotic microangiopathy (TMA). Symptoms or problems that can happen with TMA may include:
  • confusion or loss of consciousness
  • seizures
  • chest pain (angina)
  • difficulty breathing
  • blood clots or stroke
• If you miss an Ultomiris infusion, call your doctor right away

Take this medication exactly as prescribed by your doctor. Follow the directions on your prescription label carefully.

The dose your doctor recommends may be based on the following:

• the condition being treated

• other medical conditions you have

• other medications you are taking

• how you respond to this medication

• your weight

• your height

• your age

• your gender

The dose of the medication is based on weight and time of treatment and ranges from 2400 mg to 3600 mg. The medication is typically given every 8 weeks after 2 weeks of the original administration. Your doctor will work with you on establishing a proper dose and schedule of the medication.

If Ultomiris is administered by a healthcare provider in a medical setting, it is unlikely that an overdose will occur. However, if overdose is suspected, seek emergency medical attention.

Life-threatening meningococcal infections/sepsis have occurred in patients treated with ULTOMIRIS. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early.

• Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients with complement deficiencies.
• Immunize patients with meningococcal vaccines at least 2 weeks prior to administering the first dose of ULTOMIRIS, unless the risks of delaying ULTOMIRIS therapy outweigh the risk of developing a meningococcal infection [see Warnings and Precautions (5.1) for additional guidance on the management of the risk of meningococcal infection].
• Vaccination reduces, but does not eliminate, the risk of meningococcal infections. Monitor patients for early signs of meningococcal infections and evaluate immediately if infection is suspected.

ULTOMIRIS is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the ULTOMIRIS REMS, prescribers must enroll in the program [see Warnings and Precautions (5.1)]. Enrollment in the ULTOMIRIS REMS program and additional information are available by telephone: 1-844-259-6783 or at www.ultomirisrems.com.