FDA Approves New Prophylactic Treatment For Hemophilia A Without Factor VIII Inhibitors

October 4th, 2018

On October 4, 2018, the FDA approved Hemlibra® (emicizumab-kxwh) 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 only medicine approved to treat people of all ages with hemophilia A with and without factor VIII inhibitors.

News in brief

• FDA approval based on positive results from Phase III studies, including the HAVEN 3 study showing Hemlibra prophylaxis significantly reduced treated bleeds compared to no prophylaxis in people with hemophilia A without factor VIII inhibitors
• First medicine to significantly reduce treated bleeds compared to prior factor VIII prophylaxis based on a prospective intra-patient comparison
• Only approved medicine that can be self-administered subcutaneously once weekly, every two weeks or every four weeks for hemophilia A with and without factor VIII inhibitors
• The efficacy and safety of Hemlibra has been demonstrated in one of the largest pivotal clinical trial programs in hemophilia A

Supporting Information

Important Safety Information

What is the most important information I should know about HEMLIBRA?

HEMLIBRA increases the potential for your blood to clot. Carefully follow your 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 your kidneys, brain, and other organs. Get medical help right away if you have any of the following signs or symptoms during or after treatment with HEMLIBRA:
  • confusion
  • weakness
  • swelling of arms and legs
  • yellowing of skin and eyes
  • stomach (abdomen) or back pain
  • nausea or vomiting
  • feeling sick
  • decreased urination

• Blood clots (thrombotic events). Blood clots may form in blood vessels in your arm, leg, lung, or head. Get medical help right away if you have any of these signs or symptoms of blood clots during or after treatment with HEMLIBRA:
  • swelling in arms or legs
  • pain or redness in your arms or legs
  • shortness of breath
  • chest pain or tightness
  • fast heart rate
  • cough up blood
  • feel faint
  • headache
  • numbness in your face
  • eye pain or swelling
  • trouble seeing

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

Your body may make antibodies against HEMLIBRA, which may stop HEMLIBRA from working properly. Contact your healthcare provider immediately if you notice that HEMLIBRA has stopped working for you (eg, increase in bleeds).

Before using HEMLIBRA, tell your healthcare provider about all of your medical conditions, including if you:

• are pregnant or plan to become pregnant. It is not known if HEMLIBRA may harm your 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 your breast milk.

Tell your healthcare provider about all the medicines you take, including prescription medicines, over-the-counter medicines, vitamins, or herbal supplements. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.

How should I use HEMLIBRA?

See the detailed “Instructions for Use” that comes with your 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.
• You may continue prophylactic use of factor VIII for the first week of HEMLIBRA prophylaxis.

What should I know about lab monitoring?

HEMLIBRA may interfere with laboratory tests that measure how well your blood is clotting and may cause a false reading. Talk to your healthcare provider about how this may affect your 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. Speak to your healthcare provider for medical advice about side effects.

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

You may report side effects to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. You may also report side effects 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.