FDA Grants Priority Review to Xolair (omalizumab) for Children and Adults With Food Allergies Based on Positive National Institutes of Health Phase III Study Results

– If approved, Xolair would be the first medicine to reduce allergic reactions to multiple foods following an accidental exposure –

– Interim analysis results from first-of-its-kind Phase III OUtMATCH study showed Xolair significantly increased the amount of peanut, milk, egg and cashew it took to cause an allergic reaction –

– 17 million people in the U.S. have confirmed food allergies and more than 40% of children and more than half of adults with food allergies have experienced a severe reaction at least once –

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has accepted, under Priority Review, the company’s supplemental Biologics License Application (sBLA) for Xolair^® (omalizumab) for the reduction of allergic reactions, including anaphylaxis, that may occur with an accidental exposure to one or more foods in adult and pediatric patients aged 1 year and older with food allergy. If approved, people taking Xolair would still need to avoid foods they are allergic to (commonly referred to as “food avoidance”). The filing acceptance is based on positive interim analysis results from Stage 1 of the National Institutes of Health (NIH)-sponsored pivotal Phase III OUtMATCH study evaluating Xolair in patients allergic to peanuts and at least two other common foods. If approved, Xolair would be the first medicine to reduce allergic reactions to multiple foods following an accidental exposure. The FDA is expected to make a decision on approval in the first quarter of 2024.

“Despite the significant and growing health burden from food allergies, treatment advances have been limited,” said Levi Garraway, M.D., Ph.D., Genentech's chief medical officer and head of Global Product Development. “We are proud to partner with the National Institutes of Health and leading research institutions on this groundbreaking study. The FDA’s Priority Review designation acknowledges the unmet need for these patients, and we hope to make Xolair available to as many people as possible living with food allergies in the U.S.”

At a pre-planned interim analysis, an independent Data and Safety Monitoring Board (DSMB) examined the data on the first 165 children and adolescents aged 1 to 17 years who participated in the first stage of the trial and determined the study met its primary endpoint and key secondary endpoints. These interim results showed that, compared to placebo, Xolair significantly increased the amount of peanut (primary endpoint) and milk, egg and cashew (key secondary endpoints) it took to cause an allergic reaction in children and adolescents with food allergies. Safety findings were consistent with the known benefit-risk profile of Xolair across its approved indications and in previous clinical trials.

The Phase III OUtMATCH study is being sponsored and funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the NIH, and conducted by the NIAID-funded Consortium of Food Allergy Research (CoFAR) across 10 clinical sites throughout the U.S. The study is also supported by Genentech and Novartis Pharmaceuticals Corporation. Detailed results from the OUtMATCH study have been submitted by NIAID and CoFAR to a peer-reviewed journal.

Food allergies affect up to 17 million children and adults in the U.S. and food allergy prevalence has been on the rise for the past 20 years. Allergic reactions can range from mild to moderate, including hives and swelling, to severe and life-threatening, such as anaphylaxis. More than 40% of children and more than half of adults with food allergies have experienced a severe reaction at least once, and it is estimated that food-related anaphylaxis results in 30,000 medical events treated in emergency rooms in the U.S. each year.

In August 2018, the FDA granted Breakthrough Therapy Designation for Xolair for the prevention of severe allergic reactions following accidental exposure to one or more foods in people with allergies. The FDA’s Breakthrough Therapy Designation is designed to expedite the development and review of drugs that are intended to treat serious conditions. Xolair is currently FDA-approved for the treatment of moderate to severe persistent allergic asthma, chronic spontaneous urticaria (CSU) and chronic rhinosinusitis with nasal polyps (CRSwNP). Since its initial approval in 2003, more than 700,000 patients have been treated with Xolair in the U.S.

In the U.S., Genentech and Novartis Pharmaceuticals Corporation work together to develop and co-promote Xolair.

About the OUtMATCH Study

The Omalizumab as Monotherapy and as Adjunct Therapy to Multi-Allergen Oral Immunotherapy in Food Allergic Children and Adults (OUtMATCH; NCT03881696) study is an NIH-sponsored, three-stage, multicenter, randomized, double-blind, placebo-controlled study evaluating Xolair safety and efficacy in patients aged 1 to 55 years who are allergic to peanuts and at least two other common foods. The study includes three stages, of which only Stage 1 has been completed.

Stage 1 patients were randomized to receive placebo or Xolair injections either every two weeks or every four weeks for 16 to 20 weeks. The Xolair dose and dosing interval were determined by total serum immunoglobulin E (IgE) level and body weight.

About Xolair

In the U.S., Xolair is the only approved antibody designed to target and block IgE. By reducing free IgE, down-regulating high-affinity IgE receptors and limiting mast cell degranulation, Xolair minimizes the release of mediators throughout the allergic inflammatory cascade.

Indications and Important Safety Information

What is XOLAIR?

XOLAIR^® (omalizumab) for subcutaneous use is an injectable prescription medicine used to treat:

• moderate to severe persistent asthma in people 6 years of age and older whose asthma symptoms are not well controlled with asthma medicines called inhaled corticosteroids. A skin or blood test is performed to see if you have allergies to year-round allergens. It is not known if XOLAIR is safe and effective in people with asthma under 6 years of age.
• chronic rhinosinusitis with nasal polyps (CRSwNP) in people 18 years of age and older when medicines to treat CRSwNP called nasal corticosteroids have not worked well enough. It is not known if XOLAIR is safe and effective in people with CRSwNP under 18 years of age.
• chronic spontaneous urticaria (CSU, previously referred to as chronic idiopathic urticaria (CIU), chronic hives without a known cause) in people 12 years of age and older who continue to have hives that are not controlled with H1 antihistamine treatment. It is not known if XOLAIR is safe and effective in people with CSU under 12 years of age.

XOLAIR is not used to treat other allergic conditions, other forms of hives, or sudden breathing problems.

IMPORTANT SAFETY INFORMATION

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

Severe allergic reaction. A severe allergic reaction called anaphylaxis can happen when you receive 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. Go to the nearest emergency room right away if you 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

Your healthcare provider will monitor you closely for symptoms of an allergic reaction while you are receiving XOLAIR and for a period of time after treatment is initiated. Your healthcare provider should talk to you about getting medical treatment if you have symptoms of an allergic reaction.

Do not receive and use XOLAIR if you are allergic to omalizumab or any of the ingredients in XOLAIR.

Before receiving XOLAIR, tell your healthcare provider about all of your medical conditions, including if you:

• have a latex allergy or any other allergies (such as food allergy or seasonal allergies). The needle cap on the XOLAIR prefilled syringe contains a type of natural rubber latex.
• 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 your unborn baby.
• are breastfeeding or plan to breastfeed. It is not known if XOLAIR passes into your breast milk. Talk with your healthcare provider about the best way to feed your baby while you receive and use XOLAIR.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

How should I receive and use XOLAIR?

• When starting treatment XOLAIR should be given by your healthcare provider in a healthcare setting.
• If your healthcare provider decides that you or a caregiver may be able to give your own XOLAIR prefilled syringe injections, you should receive training on the right way to prepare and inject XOLAIR.
• Do not try to inject your XOLAIR prefilled syringe until you have been shown the right way to give XOLAIR injections by a healthcare provider. Use XOLAIR exactly as prescribed by your healthcare provider. For children 12 years of age and older, XOLAIR prefilled syringe may be self-injected under adult supervision. For children 6 to 11 years of age, XOLAIR prefilled syringe should be injected by a caregiver.
• See the detailed Instructions for Use that comes with XOLAIR for information on the right way to prepare and inject XOLAIR.
• XOLAIR is given in 1 or more injections under the skin (subcutaneous), 1 time every 2 or 4 weeks.
• In people with asthma and CRSwNP, a blood test for a substance called IgE must be performed before starting XOLAIR to determine the appropriate dose and dosing frequency.
• In people with chronic hives, a blood test is not necessary to determine the dose or dosing frequency.
• Do not decrease or stop taking any of your other asthma, CRSwNP, or hive medicine unless your healthcare providers tell you to.
• You may not see improvement in your symptoms right away after XOLAIR treatment. If your asthma symptoms do not improve or get worse, call your healthcare provider.
• If you inject more XOLAIR than prescribed, call your healthcare provider right away.

What are the possible side effects of XOLAIR?

XOLAIR may cause serious side effects, including:

• Cancer. Cases of cancer were observed in some people who received XOLAIR.
• Inflammation of your blood vessels. Rarely, this can happen in people with asthma who receive XOLAIR. This usually, but not always, happens in people who also take a steroid medicine by mouth that is being stopped or the dose is being lowered. It is not known whether this is caused by XOLAIR. Tell your healthcare provider right away if you have rash; chest pain; shortness of breath; or a feeling of pins and needles or numbness of your arms or legs.
• Fever, muscle aches, and rash. Some people get these symptoms 1 to 5 days after receiving a XOLAIR injection. If you have any of these symptoms, tell your healthcare provider.
• Parasitic infection. Some people who are at a high risk for parasite (worm) infections, get a parasite infection after receiving XOLAIR. Your healthcare provider can test your stool to check if you have a parasite infection.
• Heart and circulation problems. 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 these are caused by XOLAIR.

The most common side effects of XOLAIR:

• In adults and children 12 years of age and older with asthma: joint pain especially in your arms and legs, dizziness, feeling tired, itching, skin rash, bone fractures, and pain or discomfort of your ears.
• In children 6 to less than 12 years of age with asthma: swelling of the inside of your nose, throat, or sinuses, headache, fever, throat infection, ear infection, abdominal pain, stomach infection, and nose bleeds.
• In adults with chronic rhinosinusitis with nasal polyps: headache, injection site reactions, joint pain, upper abdominal pain, and dizziness.
• In people with chronic spontaneous urticaria: nausea, headaches, swelling of the inside of your nose, throat or sinuses, cough, joint pain, and upper respiratory tract infection.

These are not all the possible side effects of XOLAIR. Call your doctor for medical advice about side effects.

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 or Novartis Pharmaceuticals Corporation at (888) 669-6682.

Please see full Prescribing Information, including Medication Guide for additional Important Safety Information and Instructions for Use.

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.

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