An Open Letter to the Hemophilia Community Regarding Patient Access to Hemlibra (emicizumab-kxwh)

Update for the Hemophilia Community on Hemlibra

On February 1, 2019, a U.S. district court issued a final judgment in favor of Genentech and against Shire, through its wholly-owned subsidiaries Baxalta Inc. and Baxalta GmbH. The judgment states that Hemlibra® (emicizumab-kxwh) does not infringe Shire’s patent (U.S. Patent, 7,033,590) based on the court’s definition of key terms related to the patent. This follows the court’s ruling in August 2018 denying the request from Shire for a preliminary injunction that intended to prevent certain patients in the U.S. from receiving Hemlibra. Since the case has been resolved in favor of Genentech, there is no longer a trial scheduled for September 2019.

This outcome protects the rights of people with hemophilia A, their families and doctors to decide if Hemlibra is the right medicine for them. We are confident that patient access to Hemlibra in the U.S. will not be affected as a result of this legal matter.

At the Roche Group—Genentech in the U.S., Chugai in Japan, and Roche in the rest of the world—we look forward to continuing to focus on the hemophilia community and making Hemlibra available to people all over the world who are living with hemophilia A and may benefit from this therapy.

South San Francisco, CA — January 11th, 2018 —

This is an open letter to the hemophilia community to clarify how recent legal actions could impact patient access to Hemlibra® (emicizumab-kxwh, formerly ACE910)1. As of now, there are no limitations on the ability of physicians to prescribe Hemlibra in the U.S., and we assure you that we are doing everything we can to protect patients’ rights to access this important new medicine.

At the Roche Group—Genentech in the U.S., Chugai in Japan, and Roche in the rest of the world—we are dedicated to improving the lives of patients through innovation and scientific discoveries. Hemlibra represents one of our most important scientific achievements, with over 20 years of research and development invested in bringing this therapy to patients with hemophilia.

We are writing this letter because we value transparency. The hemophilia community has fought hard to preserve access to healthcare and to treatment choice, and deserves more information about recent legal actions and patent infringement claims. We strongly believe that patients should not be put in the middle of these legal disputes, and we are committed to preventing any impact on patients throughout the ongoing litigation. It is understandable that companies may disagree about their intellectual property or patents, but it is never acceptable for a company to try to keep breakthrough medicines from patients.

The most recent legal actions began in May 2017 when Shire sued Genentech and Chugai for alleged patent infringement in the U.S. We do not believe that Shire’s patent is valid, nor that Hemlibra infringes this patent. We intend to vigorously defend our case in court during a trial, which is scheduled for September 2019. In the meantime, on December 14, 2017, Shire filed a motion for a preliminary injunction against Genentech that attempts to limit patient access to this medicine before the scheduled patent trial. In its motion, Shire asked the court to issue an order that prevents us from providing Hemlibra to certain patients in the U.S. We believe the injunction is unfounded and will oppose it. The court would need to grant Shire’s request before it can be implemented.

If granted as proposed, Shire’s preliminary injunction would prevent certain patients in the U.S. from receiving Hemlibra. Specifically, Shire has proposed that the court prohibit Genentech from selling Hemlibra to the following patients:  

  • Hemophilia A patients with inhibitors (defined as those with an inhibitor titer of greater than 5 Bethesda units who cannot be treated effectively with Factor VIII replacement therapy), unless (i) they have already started Hemlibra before the injunction is granted by the court, or (ii) they have previous experience with on-demand or prophylactic bypassing agents and their needs are not currently being met, as defined by Shire using criteria that include experiencing certain life- or limb-threatening bleeds or venous access issues.
  • Hemophilia A patients who have an inhibitor titer less than or equal to 5 Bethesda units, or who can be effectively treated with Factor VIII replacement therapy, regardless of whether they have already started Hemlibra.
  • Hemophilia A patients without an inhibitor, regardless of whether they have already started Hemlibra.

The FDA approved Hemlibra for routine prophylaxis for adult and pediatric patients with hemophilia A with inhibitors. The data that supported the FDA approval have also been submitted to the European Medicines Agency and are currently under review for approval consideration. We believe it is inappropriate for Shire to dictate which patients should receive Hemlibra. That decision rests with treating physicians and patients. Over the last two years, we have met with Shire to try to come to an agreement in the best interest of patients. It is now up to the court to make a ruling and we are prepared to defend our case. We are disappointed that Shire would attempt to limit patient access to Hemlibra in advance of the full trial before the court.

In addition to these legal actions in the U.S., Shire has taken other actions over the last two years that attempt to limit patient access to this medicine worldwide or block scientific exchange about this medicine, including:

  1. Shire attempts to block global patient access and manufacturing of Hemlibra in Japan: In April 2016, Shire, through its wholly-owned subsidiaries Baxalta Inc. and Baxalta GmbH (“Shire”), filed a lawsuit against Chugai for alleged patent infringement, and asked the Japanese court to stop the global exportation, sale and manufacture of Hemlibra. Again, we do not believe that Shire’s patent is valid, nor that Hemlibra infringes this patent. In addition, Shire asked the court to order the destruction of the existing inventory of medicine. Chugai is pursuing a vigorous defense through the appropriate legal channels. This lawsuit is ongoing at this time.
  2. Shire attempts to block scientific exchange at a key medical congress: On July 9, 2017, Shire obtained a preliminary injunction in Germany against Roche alleging that Roche made “incomplete and misleading statements” about Hemlibra in an abstract of the pivotal HAVEN 1 data. This may have been an effort to prevent the data from being presented on July 10, 2017 at the International Society on Thrombosis and Haemostasis (ISTH) Congress in Germany. In September, a German court reviewed the evidence and concluded that Roche had not engaged in any inappropriate promotional activities. Following those comments from the judges, Shire chose to withdraw their injunction. As a result, the case was formally closed and Shire incurred all related court and legal fees for Roche.

While the lawsuits are currently active in Japan and the U.S., similar actions could follow in other geographies where similar patents exist, such as Germany, France, UK, Spain, Italy and Australia.

We hope this letter provides transparency into a matter that has the potential to affect patients. As a reminder, there are currently no limitations on the ability of physicians to prescribe Hemlibra in the U.S. We ask you to join us in advocating for access to Hemlibra for all appropriate patients so there are no limitations in the future.

Thank you for your consideration of this important matter and for your partnership. We will continue to share as much information as possible.

Sincerely,

The Hemlibra Team

 

Hemlibra U.S. Indication

Hemlibra is a prescription medicine used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with factor VIII inhibitors.

Important Safety Information

Hemlibra increases the potential for blood to clot. Discontinue prophylactic use of bypassing agents the day before starting Hemlibra prophylaxis. Carefully follow the healthcare provider’s instructions regarding when to use an on-demand bypassing agent, and the dose and schedule one should use. Cases of thrombotic microangiopathy and thrombotic events were reported when on average a cumulative amount of >100 U/kg/24 hours of activated prothrombin complex concentrate (aPCC) was administered for 24 hours or more to patients receiving Hemlibra prophylaxis.

Hemlibra may cause the following serious side effects when used with 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:

    • 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 one’s 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:

    • swelling in arms or legs
    • pain or redness in the arms or legs
    • shortness of breath
    • chest pain or tightness
    • fast heart rate
    • cough up blood
    • feel faint
    • headache
    • numbness in the face
    • eye pain or swelling
    • trouble seeing

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. 

How should patients use Hemlibra?

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.

What are the other possible side effects of Hemlibra?

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 call their doctor for medical advice about side effects.

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 the Medication Guide, including Serious Side Effects, for more important safety information.


Reference:

 1. Hemlibra is currently approved in the U.S. for routine prophylaxis for adults and pediatric patients with hemophilia A with inhibitors. It is not currently approved for hemophilia A patients without an inhibitor. It has not yet been approved for use outside of the U.S.


About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.