Highly Anticipated New Medicine for Hemophilia A

(BPT) - Growing up, Roberto Novelli wore a helmet every day until he was 4 years old. His parents were very protective after he was diagnosed with a bleeding disorder called hemophilia A at just 2 months old. To manage his disorder, he received three infusions of treatment into his veins each week to try to prevent bleeds.

Living with hemophilia A means a person’s blood does not clot properly, which can result in bleeds in the muscles and joints that may lead to joint damage and chronic pain.^[1] For some, even a single joint bleed can cause long-term damage.^[2] Roberto isn’t alone — about 20,000 Americans have hemophilia, with hemophilia A being the most common form.^[3]

“Before I got to high school, my parents didn’t want me playing sports or being too active. They were so worried about me getting a serious bleed,” he said. “It really impacted my life growing up.”

Roberto, now 26, is an academic advisor at the University of Michigan-Dearborn, a graduate student and an avid outdoor enthusiast who spends much of his free time kayaking.

In 2017, Dr. Michael Callaghan, a hematologist at Children’s Hospital of Michigan, recommended Roberto join a clinical trial for Hemlibra^® (emicizumab-kxwh), which was being studied as a prophylactic (preventative) treatment for people with hemophilia A.

“When Dr. Callaghan mentioned trying Hemlibra to me, I’ll admit I was nervous — why mess with what you’ve been doing for so long?” Roberto said. “But I’m glad I took the opportunity to join the clinical trial. With Hemlibra, I’ve had fewer bleeds, and I’m proud to have played a role in making this medicine available for other people with hemophilia A.”

In October 2018, the U.S. Food and Drug Administration (FDA) approved Hemlibra for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A without factor VIII inhibitors. Hemlibra is now the first and only approved medicine for hemophilia A, with or without factor VIII inhibitors, that can be self-administered under the skin (subcutaneously) and only once weekly, every two weeks or every four weeks.

“The approval of Hemlibra is an important advancement for the entire hemophilia A community,” Dr. Callaghan said. “For the first time in nearly 20 years, people with hemophilia A, like Roberto, have a new class of medicine. Hemlibra can reduce bleeds, while offering a new subcutaneous administration and multiple dosing options.”

Positive results from the clinical trial Roberto joined showed Hemlibra prophylaxis significantly reduced treated bleeds compared to no prophylaxis treatment in people with hemophilia A without factor VIII inhibitors, and 55.6 percent of people taking Hemlibra every week had zero bleeds after 24 weeks compared to 0 percent of people receiving no prophylaxis treatment. Additionally, Hemlibra is the first medicine to significantly reduce treated bleeds compared to prior factor VIII prophylaxis, which has been the recommended standard of care for people with hemophilia A without factor VIII inhibitors, as demonstrated by a statistically significant reduction of 68 percent in treated bleeds in an intra-patient comparison.

Hemlibra increases the potential for blood to clot. Patients should carefully follow their healthcare provider’s instructions regarding when to use an on-demand bypassing agent or factor VIII, and the dose and schedule to use for breakthrough bleed treatment. Hemlibra may cause serious side effects when used with activated prothrombin complex concentrate (aPCC; FEIBA^®), including thrombotic microangiopathy (TMA) and blood clots (thrombotic events). If aPCC (FEIBA^®) is needed, patients should talk to their healthcare provider in case they feel they need more than 100 U/kg of aPCC (FEIBA^®) total. Hemlibra may interfere with laboratory tests that measure how well a patient’s blood is clotting and may cause a false reading.^[4]

“Taking Hemlibra once a week reduced the amount of time I spent treating my hemophilia A — it was a meaningful milestone,” Roberto said. “Because of that, I have more time to do other things. This summer I went to Michigan’s Pictured Rocks and Kensington Metropark, and I spent more time outdoors and kayaking and less time taking my medicine.”

Everyone is different and each individual's experience may vary. For more information about hemophilia A with or without factor VIII inhibitors and Hemlibra, visit www.hemlibra.com and talk to your doctor.

Please see the Hemlibra full Prescribing Information and Medication Guide for more important safety information including Serious Side Effects.

Patient Indication and Safety Information

Hemlibra is a prescription medicine used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A with or without factor VIII inhibitors.

What is the most important information to know about Hemlibra?

Hemlibra increases the potential for blood to clot. Patients should carefully follow their healthcare provider’s instructions regarding when to use an on-demand bypassing agent or factor VIII, and the dose and schedule to use for breakthrough bleed treatment. Hemlibra may cause the following serious side effects when used with activated prothrombin complex concentrate (aPCC; FEIBA^®), including:

• Thrombotic microangiopathy (TMA). This is a condition involving blood clots and injury to small blood vessels that may cause harm to one’s kidneys, brain, and other organs. Patients should get medical help right away if they have any of the following signs or symptoms during or after treatment with Hemlibra:

• Blood clots (thrombotic events). Blood clots may form in blood vessels in the arm, leg, lung, or head. Patients should get medical help right away if they have any of these signs or symptoms of blood clots during or after treatment with Hemlibra:

If aPCC (FEIBA^®) is needed, patients should talk to their healthcare provider in case they feel they need more than 100 U/kg of aPCC (FEIBA^®) total.

Before using Hemlibra, patients should tell their healthcare provider about all of their medical conditions, including if they:

• are pregnant or plan to become pregnant. It is not known if Hemlibra may harm an unborn baby. Females who are able to become pregnant should use birth control (contraception) during treatment with Hemlibra.
• are breastfeeding or plan to breastfeed. It is not known if Hemlibra passes into breast milk.

Patients should tell their healthcare provider about all the medicines they take, including prescription medicines, over-the-counter medicines, vitamins, or herbal supplements. Patients should keep a list of them to show their healthcare provider and pharmacist when they get a new medicine.

How should patients use Hemlibra?

Patients should see the detailed “Instructions for Use” that comes with Hemlibra for information on how to prepare and inject a dose of Hemlibra, and how to properly throw away (dispose of) used needles and syringes.

• Stop (discontinue) prophylactic use of bypassing agents the day before starting Hemlibra prophylaxis.
• Patients may continue prophylactic use of factor VIII for the first week of Hemlibra prophylaxis.

What should patients know about lab monitoring?

Hemlibra may interfere with laboratory tests that measure how well blood is clotting and may cause a false reading. Patients should talk to their healthcare provider about how this may affect their care.

The most common side effects of Hemlibra include: redness, tenderness, warmth, or itching at the site of injection; headache; and joint pain.

These are not all of the possible side effects of Hemlibra. Patients should speak to their healthcare provider for medical advice about side effects.

Medicines are sometimes prescribed for purposes other than those listed in a Medication Guide. Patients should not use Hemlibra for a condition for which it was not prescribed. Patients should not give Hemlibra to other people, even if they have the same symptoms that they have. It may harm them. Patients can ask their pharmacist or healthcare provider for information about Hemlibra that is written for health professionals.

Side effects may be reported to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch. Side effects may also be reported to Genentech at (888) 835-2555.

Please see the Hemlibra full Prescribing Information and Medication Guide for more important safety information including Serious Side Effects.