FDA Files Genentech’s Supplemental Biologics License Application of Xolair® (omalizumab) for Pediatric Allergic Asthma

SOUTH SAN FRANCISCO, CA, USA I March 01, 2016 I Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the company’s supplemental Biologics License Application (sBLA) to extend the indication of Xolair^® (omalizumab) in allergic asthma to pediatric patients. The FDA will review Xolair in children from six through 11 years for the treatment of moderate to severe persistent asthma in those patients with a positive skin test or in vitro reactivity to a perennial aeroallergen (airborne allergen) and symptoms that are inadequately controlled with inhaled corticosteroids. Genentech anticipates hearing from the FDA later this year.

“Childhood allergic asthma often remains uncontrolled despite the use of inhaled steroids,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “The disease can significantly impact a child, and this filing acceptance brings us one step closer to addressing this significant unmet need.”Asthma is the leading chronic disease in children¹, affecting about seven million or one in 10 children in the U.S.^2,3. Approximately 25 million people in the U.S. have asthma⁴, with allergic asthma being the most common form of the disease⁵.Xolair was approved in 2003 in the U.S. for moderate to severe persistent asthma in patients 12 years of age and above with a positive skin test or in vitro reactivity to a perennial aeroallergen and symptoms that are inadequately controlled with inhaled corticosteroids. In 2014, the FDA also approved Xolair to treat adults and children 12 years of age and older with chronic idiopathic urticaria (CIU) – chronic hives without a known cause – who continue to have hives that are not controlled by H₁-antihistamine treatment. Xolair is not indicated for the treatment of other allergic conditions, other forms of urticaria (hives), acute bronchospasm (serious and sudden breathing problems) or status asthmaticus (acute, severe, prolonged asthma attack that can be life-threatening). Since the launch of Xolair, more than 200,000 patients 12 years of age and older with allergic asthma in the U.S. have been treated with the medicine⁶.In the U.S., Genentech, Inc. and Novartis Pharmaceuticals Corporation work together to develop and co-promote Xolair. Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or life-threatening medical conditions. Novartis Pharmaceuticals Corporation researches, develops, manufactures and markets innovative medicines aimed at improving patients’ lives.Development Program
The sBLA is supported by multi-center, randomized, double-blind, placebo-controlled Phase III studies that assessed the efficacy and safety of Xolair in children aged six to 11 years with moderate to severe persistent uncontrolled allergic asthma. The primary study is a 52-week trial, with the primary endpoint measured at 24 weeks⁷. Supportive safety and efficacy data comes from a 28-week study⁸. Additional safety data come from a five-year non-randomized observational post-marketing study to evaluate the long-term safety of Xolair in patients12 years⁹ and older.About Xolair
Xolair for subcutaneous use is an injectable prescription medicine used to treat adults and children 12 years of age and older with:

• moderate to severe persistent asthma whose symptoms are not controlled by asthma medicines called inhaled corticosteroids. A skin or blood test is performed to see if you have allergies to year-round allergens.
• chronic idiopathic urticaria (CIU; chronic hives without a known cause) who continue to have hives that are not controlled by H1-antihistamine treatment.

Xolair is not used to treat other allergic conditions, other forms of urticaria, acute bronchospasm or status asthmaticus.Important Safety Information
The most important safety information patients should know about Xolair is that a severe allergic reaction called anaphylaxis can happen when a patient receives Xolair.  The reaction can occur after the first dose, or after many doses.  It may also occur right after a Xolair injection or days later.  Anaphylaxis is a life-threatening condition and can lead to death. Patients must go to the nearest emergency room if they have any of these symptoms of an allergic reaction:

• wheezing, shortness of breath, cough, chest tightness, or trouble breathing
• low blood pressure, dizziness, fainting, rapid or weak heartbeat, anxiety, or feeling of “impending doom”
• flushing, itching, hives, or feeling warm
• swelling of the throat or tongue, throat tightness, hoarse voice, or trouble swallowing

The patient’s healthcare provider will monitor the patient closely for symptoms of an allergic reaction while they are receiving Xolair and for a period of time after the patient’s injection. The patient’s healthcare provider should talk to the patient about getting medical treatment if they have symptoms of an allergic reaction after leaving the healthcare provider’s office or treatment center. Patients must not receive Xolair if they are allergic to omalizumab or any of the ingredients in Xolair.Before receiving Xolair, patients must tell their healthcare provider about all of their medical conditions, including if they:

• have any other allergies (such as food allergy or seasonal allergies)
• have sudden breathing problems (bronchospasm)
• have ever had a severe allergic reaction called anaphylaxis
• have or have had a parasitic infection
• have or have had cancer
• are pregnant or plan to become pregnant. It is not known if Xolair may harm a patient’s unborn baby.
• if a patient becomes pregnant while taking Xolair, they should talk to their healthcare provider. Patients can also call Genentech Drug Safety at 888-835-2555 to report their pregnancy.
• Are breastfeeding or plan to breastfeed. It is not known if Xolair passes into breast milk.

Patients must tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, or herbal supplements.Receiving Xolair

• Xolair should be given by a healthcare provider in a healthcare setting.
• Xolair is given in one or more injections under the skin (subcutaneous), one time every two or four weeks.
• In asthma patients, a blood test for a substance called IgE must be performed prior to starting Xolair to determine the appropriate dose and dosing frequency. In patients with chronic hives, a blood test is not necessary to determine the dose or dosing frequency.
• Patients must not decrease or stop taking any of their other asthma or hive medicine unless their healthcare providers tell them to.
• Patients may not see improvement in their symptoms right away after Xolair treatment.

Possible side effects of Xolair:

Xolair may cause serious side effects, including:

• See “What is the most important information I should know about Xolair?” in the Xolair Medication Guide at http://www.xolair.com regarding the risk of anaphylaxis.
• Cancer. People who receive treatment with Xolair may have a higher chance for getting certain types of cancer.
• Fever, muscle aches, and rash. Some people who take Xolair get these symptoms one to five days after receiving a Xolair injection. If a patient has any of these symptoms, they must tell their healthcare provider.
• Parasitic infection. Some people who are at a high risk for parasite (worm) infections, get a parasite infection after receiving Xolair. The patient’s healthcare provider can test the patient’s stool to check if they have a parasite infection.
• Some people who receive Xolair have had chest pain, heart attack, blood clots in the lungs or legs, or temporary symptoms of weakness on one side of the body, slurred speech, or altered vision. It is not known whether this is caused by Xolair.

The most common side effects of Xolair:

• In people with allergic asthma: pain especially in the arms and legs, dizziness, feeling tired, skin rash, bone fractures, and pain or discomfort of the ears.
• In people with chronic idiopathic urticaria: nausea, headaches, swelling of the inside of the nose, throat or sinuses, cough, joint pain, and upper respiratory tract infection.

These are not all the possible side effects of Xolair. Patients should call their doctor for medical advice about side effects.Report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch. Report side effects to Genentech at (888) 835-2555 or Novartis Pharmaceuticals Corporation at (888) 669-6682.Please see full Prescribing Information, including Medication Guide at http://www.xolair.com for additional Important Safety Information.About Genentech
Founded 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or 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.References
¹ Asthma and Allergy Foundation of America (AAFA) website. Asthma Facts and Figures. August 2015. http://www.aafa.org/page/asthma-facts.aspx. Accessed February 23, 2016.
² U.S. Centers for Disease Control and Prevention. National Institute of Allergy and Infectious Diseases. Asthma Facts. April 2014. http://www.niaid.nih.gov/topics/asthma/understanding/Pages/facts.aspx. Accessed February 23, 2016.
³ U.S. Centers for Disease Control and Prevention. National Institute of Allergy and Infectious Diseases. Asthma in the U.S. June 2012. http://www.niaid.nih.gov/topics/asthma/understanding/Pages/usAsthma.aspx. Accessed February 23, 2016.
⁴ National Institute of Allergy and Infectious Disease. Asthma. http://www.niaid.nih.gov/topics/asthma/Pages/default.aspx. Accessed February 23, 2016.
⁵ American Academy of Allergy, Asthma & Immunology (AAAAI). Allergic Asthma. http://www.aaaai.org/conditions-and-treatments/conditions-a-to-z-search/allergic-asthma.aspx.  Accessed February 23, 2016.
⁶ Data on file. Genentech, Inc, South San Francisco, CA.
⁷ Lanier B et al. Omalizumab for the treatment of exacerbations in children with inadequately controlled allergic (IgE-mediated) asthma. J Allergy Clin Immunol. 2009;124:1210-1216.
⁸ Milgrom H, Berger W, Nayak A, Gupta N, Pollard S, McAlary M, et al. Treatment of childhood asthma with anti-immunoglobulin E antibody (omalizumab). Pediatrics 2001;108:E36.
⁹ Long A, Rahmaroui A, Rothman K, et al. Incidence of malignancy in patients with moderate-to-severe asthma treated with or without omalizumab. J Allergy Clin Immunol. 2014;134:560-7.

SOURCE: Genentech