People with severe hemophilia A are at risk for prolonged bleeding events that can cause serious complications. To prevent these bleeding events, most people with this condition get injections of a blood protein called factor VIII. In most cases, these injections must be given multiple times each week.
In 2023, Altuviiio became the first once-weekly factor VIII product to be approved for reducing the risk of bleeding events in people with hemophilia A.
How Does Altuviiio Work for Hemophilia A?
Hemophilia A is a rare, inherited condition that is passed down from parents to their children. People with this condition have low levels of a blood protein called factor VIII, which keeps the blood from clotting the way that it normally would. Due to these low levels, the body has trouble stopping the flow of blood once a person has started bleeding. This is referred to as a prolonged bleeding event.
This condition can be mild, moderate, or severe depending on a person’s factor VIII levels. Because of the way that this condition is inherited (“X-linked”), males are most often affected and usually have more severe forms of the condition. Females can also be affected but tend to have milder symptoms.
People with mild hemophilia A may have prolonged bleeding events after a surgical procedure, dental work, or a serious injury. They may also have frequent nosebleeds or bleeding from the gums. People with more severe forms of this condition may have prolonged bleeding events in other situations, such as after small cuts or injuries. Some people with severe hemophilia A may also bleed into their vital organs, joints, or muscles even when there is no clear cause. These events, called “spontaneous bleeds,” can cause long-term damage and can become life-threatening if they are not treated.
Most people with hemophilia A experience symptoms as children or teenagers. In fact, they may experience symptoms more often at a younger age than they do as adults. Young children with severe hemophilia A who are not receiving treatment can have as many as two to five bleeding episodes each month.
Treatment for this condition usually involves replacing factor VIII with a form of the protein that is made in a lab, called “recombinant” factor VIII. These injections can be used in two ways:
- They help to stop a prolonged bleed, and people with all forms of hemophilia A can use these injections to treat a bleeding event. This is called “on-demand” treatment.
- In people with more severe forms of hemophilia A, regular injections can raise factor VIII levels high enough to help prevent prolonged and spontaneous bleeding events. As a result, many people with severe hemophilia A will receive regular, scheduled injections of factor VIII.
Many different factor VIII products have been approved for use in people with hemophilia A. When they are used on a regular schedule to prevent bleeding events, most of these products must be given two to three times each week.
Altuviiio is a factor VIII product that is given once weekly. It contains a unique fusion protein (Fc-VWF-XTEN) that makes factor VIII more stable, allowing it to stay active in the body for a longer time after each infusion.
How Do I Use It?
Altuviiio is a liquid given as an infusion into the vein (IV). This infusion is often given by a health care professional in a health care facility. If you are interested in giving the medicine at home, you or a caregiver will need special training on how to prepare the dose and place a needle into the vein. You should speak with your health care provider about whether this may be a good option for you.
Each dose of Altuviiio comes in a box that contains a vial of powder and a syringe of liquid (diluent), as well as other supplies that will help you to prepare your dose. Your prescription will also come with detailed “Instructions for Use,” which provide step-by-step guidance on how to use the medicine. It is important to follow each step in the process to ensure that the medicine remains sterile and that you get the full dose each time. A general overview of this process is provided here.
First you will need to reconstitute the medicine, which involves mixing the liquid in the syringe with the powder in the vial. This is done by connecting a vial adapter to the vial. You will then connect the plunger rod to the syringe and remove the cap from the top of the syringe. Connect the syringe to the vial adapter and slowly push the liquid into the vial. Once all of the liquid has entered the vial, you will need to swirl the vial gently until the powder has completely dissolved. Then, you will turn the vial upside down and draw the liquid back out of the vial and into the syringe.
It is important that you inspect the medicine before using it. The reconstituted medicine should be clear and either colorless or slightly yellowish. If it is cloudy or there are any particles in the liquid, you should not use the dose. Once a dose is reconstituted, it must be used within 3 hours.
Some people may need to reconstitute more than one vial of medicine to get their full dose. In this case, you will follow the steps above to reconstitute as many vials as needed for your dose. Then you will combine the liquid in the smaller syringes into one, larger syringe supplied by your health care provider or pharmacy.
When your full dose has been prepared, you will attach a special type of tubing to the syringe. This tubing connects to a needle that must be placed into the vein and secured. Once this is done, you can begin to give the dose of medicine by slowly pushing down on the syringe plunger. This will push the medicine through the tube and into the vein. Your health care provider will tell you how slowly to push the medicine into the vein. It typically takes about 1 to 10 minutes to give the full dose.
Are There Any Other Unique Considerations With This Medicine?
Some people with hemophilia A have developed antibodies, or “inhibitors,” against other factor VIII replacement products. These inhibitors bind to and destroy factor VIII, making these products much less effective for preventing or treating bleeding events. It isn’t clear how often this may happen in people who are using Altuviiio.
Your health care provider will monitor your blood for these inhibitors. If they detect low levels of inhibitors in your blood, they may recommend a higher dose of Altuviiio. If they detect high levels of inhibitors in your blood, you may not be able to use Altuviiio anymore. In this case, they will discuss some of the other treatment options that have been developed specifically for people with inhibitors in their blood.
How Was It Studied for Preventing Bleeding Events?
One study was done to see if Altuviiio could prevent prolonged or spontaneous bleeding events in adults and adolescents with hemophilia A. This study included people who were at least 12 years old, with an average age of about 35. Almost every person in the study (99%) was male. About 61% were White, about 18% were Asian, 2% were Black, and the race of the other people in the study was not reported.
Everyone in this study had severe hemophilia A and was already receiving regular injections of a factor VIII product before the study started. These people had experienced about eight prolonged bleeding episodes in the past year, on average, and about 6 episodes of bleeding into the joints in the past year, on average.
When the study started, everyone stopped using their previous factor VIII product. During the study, most people received an infusion of Altuviiio once per week. If they experienced a prolonged bleeding event, they were told to use another dose of Altuviiio to treat their bleeding.
This study measured efficacy by counting the number of bleeding episodes each person had during the time in the study.
What Did These Studies Find?
Number of bleeding episodes. In the group who used Altuviiio once weekly for a year, the average rate of bleeds during that year was 0.71 (less than one). During the year of the study, about two-thirds (65%) of people had no bleeding events and almost everyone (93%) had two or fewer bleeding events.
Since everyone in this study was already using a factor VIII product before the study started, this means that Altuviiio may work better for reducing the number of prolonged bleeding events in some people.
Treating bleeding episodes. If a person had a prolonged bleeding event during the study, they were told to treat it with Altuviiio. One injection of Altuviiio was enough to effectively treat almost every bleeding event (97%) that happened during the study.
How Was It Studied in Children?
One clinical study was done in children under 12 years old. Everyone (100%) in this study was male, and the average age was 5. About 74% were White, about 11% were Asian, about 4% were Black, and the race of the other people was not reported. All of these children had severe hemophilia A and were receiving regular injections of factor VIII before the study started.
When the study started, everyone stopped using their previous factor VIII product and started using Altuviiio once weekly. To evaluate safety, the study counted the number of children who developed an inhibitor to Altuviiio. It also recorded the number and type of side effects that people experienced. To evaluate efficacy, the study counted the number of bleeding events that happened during the year in the study.
None of the children in this study developed an inhibitor to Altuviiio. Most of the side effects that happened during the study were relatively mild, with the most common side effects being fever and infections of the airways. During the year in the study, about two-thirds (64%) of the children had no bleeding events. When a bleeding event did happen, almost all of them (95%) were effectively treated with one injection of Altuviiio.
How Quickly Will Altuviiio Work for Hemophilia A?
Altuviiio will raise your levels of factor VIII right away. If you are using Altuviiio every week, these higher levels should reduce your risk of a prolonged or spontaneous bleeding event. Do not stop using Altuviiio without talking to your health care provider first, even if your condition has improved. Altuviiio must be given every week in order to keep factor VIII levels up and reduce the risk of bleeding. If you miss a dose, it is important to use a dose as soon as possible.
Are There Any Interactions With Other Medicines?
There are no known interactions between Altuviiio and other medicines. But it is still important for your health care provider to be aware of all the prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, or other supplements you take or have recently taken.
Are There Any Special Steps Required to Get Altuviiio?
Altuviiio is a type of medicine called a “specialty” medicine. This means that you may need to get it from a specialty pharmacy and that it may require prior authorization from your insurance company.
Is There a Coupon Card Available?
There is a savings coupon available from the manufacturer that may allow you to pay as little as $0 for your prescription. Whether you are eligible depends on whether you have prescription insurance and what type of insurance you have. You can find out more at portal.trialcard.com/sanofi/hem.