Hemophilia is a rare genetic condition that keeps your blood from clotting, leading to bleeding problems. Bleeding can happen at any time (spontaneous) or as a result of trauma (injury, cut, or fall). People with hemophilia A do not have enough of a protein called factor VIII, and people with hemophilia B do not have enough of a protein called factor IX. These factors are needed to make clots and stop bleeding. Regular treatment to prevent bleeding, called “prophylaxis,” may be needed, and if bleeding happens, you may need “on-demand” therapy. Factor replacement therapy given into a vein (intravenous) with factor VIII or factor XI is used to control hemophilia. In some people, the body develops inhibitors (antibodies against factor replacement products), which reduces the effectiveness of factor products.
Alhemo (concizumab-mtci) is approved to prevent or reduce bleeding problems in people with hemophilia A with or without factor VIII inhibitors or hemophilia B with or without factor IX inhibitors. It is given once a day as a shot under the skin.
How Does Alhemo Work?
Alhemo is a biologic medicine that blocks a protein called tissue factor pathway inhibitor (TFPI). This substance is normally made by your body and prevents blood from clotting. Blocking this substance allows your body to make more factor Xa, which can help you form clots and reduce bleeding problems.
How Do I Use Alhemo?
Alhemo is a subcutaneous (SQ) injection that is available as a prefilled pen. If your health care provider decides you can give your injections at home, they will give you instructions and teach you how to give yourself or your child the injection. Read the Medication Guide and the Patient Instructions for Use.
In adults and children aged 12 and older, the medicine is used once every day. Your dose will be larger (loading dose) on your first day of treatment. Follow directions from your health care provider about the exact dose that you need to use. You will need to have a blood test after four weeks to make sure you have the right amount of medicine in your blood. Your health care provider may change your dose based on the results of the blood test. Your dose is based on your weight, so if you have a change in your weight, let your health care provider know.
Alhemo is injected into the thighs or stomach area by you or a caregiver. Do not inject into areas of the skin that are tender, bruised, red, or hard, or in an area that has moles, scars, or stretch marks. Each injection should be given at a different site with a new needle. Do not inject within 2 inches around your belly button.
It is very important to use your dose every day to protect against bleeding. Do not stop using Alhemo without talking to your health care provider. You may need other medicines called bypassing agents during treatment with Alhemo. Follow your health care provider’s directions for how to use on-demand bypassing agents to treat any bleeding that happens while you are using Alhemo.
If you miss a dose of Alhemo during the first four weeks of treatment, tell your health care provider right away to find out how much medicine to give yourself. After your normal (maintenance) dose has been established, if you miss one dose, take it as soon as possible and then continue taking at your usual time. If you miss two to six doses, take two doses as soon as possible, then continue taking your usual dose the next day. If you miss seven or more doses, tell your health care provider right away because you will need a new loading dose before continuing your normal daily dose.
How Was Alhemo Studied?
Two clinical studies, EXPLORER 7 and EXPLORER 8, were done to evaluate the effectiveness and safety of Alhemo in males with hemophilia. Both studies focused on the treatment of bleeding episodes.
The EXPLORER 7 trial included 91 adults and 42 children 12 years of age and older with hemophilia A or B with inhibitors who used bypassing agents. The average age was 29 years old and the study included White people (59%), Asian people (28%), Black people (7%), Hispanic or Latino people (5%), and American Indian or Alaska Native people (2%). People with a history of blood clots or who were at high risk for blood clots, were using or planned to use immune tolerance induction treatment, or had planned major surgery were not included in the study. People were assigned to two groups to continue on-demand treatment (treatment only when bleeding happens) for 24 or more weeks (group 1) or daily prophylaxis with Alhemo for 32 or more weeks (group 2).
The EXPLORER 8 trial included 118 adults and 38 children 12 years of age and older with hemophilia A or B without inhibitors. The mean (middle) age was 32 years (range: 12 to 72) and the study included White people (66%), Asian people (28%), Black people (3%), Hispanic or Latino people (8%), and American Indian or Alaska Native people (2%). Similar to the EXPLORER 7 trial, people were assigned to receive either Alhemo prophylaxis or no prophylaxis (on-demand treatment).
What Benefits Were Seen?
In both studies, people who used daily prophylaxis with Alhemo had a lower rate of treated spontaneous and traumatic bleeding episodes. In the EXPLORER 7 study, Alhemo reduced the annualized bleeding rate (ABR) by 86%. In EXPLORER 8, Alhemo reduced the ABR by 86% in patients with hemophilia A and by 79% in patients with hemophilia B.
Your results may differ from what was seen in clinical studies.
What Are the Side Effects and How Can I Manage Them?
Because Alhemo comes as an injection, a common side effect from the medicine is an injection site reaction. This can include bruising, redness, bleeding, itching, or hives where the needle goes in the skin. Applying a cool compress to the area may help. Your health care provider or pharmacist may recommend an over-the-counter (OTC) pain reliever, such as acetaminophen, or an OTC antihistamine for itching. Talk to your health care provider if you experience an injection site reaction and before taking any medicines to help with your symptoms. Rotating your injection sites can also help minimize injection site reactions.
Alhemo may cause serious side effects such as blood clots to form in blood vessels like your arms, legs, heart, lung, brain, eyes, kidneys, or stomach. You may be at higher risk if you use high or frequent doses of factor replacement products or bypassing agents to treat bleeding episodes or if you have certain medical conditions. Contact your health care provider right away if you have symptoms of heart problems or blood clots such as fast heartbeat, pain in your chest, feeling short of breath, or pain or swelling in your legs. Tell your health care provider if you plan to have surgery or an invasive procedure.
Is There Any Cost Assistance Available?
There is a patient support program, NovoCare Bleeding Disorders, that may help you afford Alhemo. Your health care provider will help you get started. For questions or more information, you can contact the drugmaker at 844-668-6732 or visit www.novocare.com/bleeding-disorders/home.html.
Getting a Specialty Medicine: What to Expect
Your health care provider will prescribe Alhemo. It is a specialty medicine, which is a high-cost medication that is taken for rare, complex, or chronic (long-term) diseases. It will require a different process than picking up a prescription at your local pharmacy. You will use a specialty pharmacy for getting this medicine. The specialty pharmacy will reach out to you to discuss the details about how to ship Alhemo to your house.
Your insurance may require approval for using Alhemo, also called prior authorization. The insurance company reviews the prescription from your health care provider to make sure it is covered and determines the process that needs to be followed.
You may also be able to work with a representative from the drugmaker that can help you through the process of insurance coverage and with details around the specialty pharmacy shipping the medicine.