FDA Approves Dupixent® (dupilumab) as First Treatment for Adults and Children Aged 12 and Older with Eosinophilic Esophagitis
TARRYTOWN, N.Y. and PARIS, May 20, 2022 --
Dupixent is the first and only medicine indicated to treat eosinophilic esophagitis in the United States; approval granted more than two months ahead of FDA's Priority Review action date
Dupixent 300 mg weekly significantly improved signs and symptoms of eosinophilic esophagitis compared to placebo in a Phase 3 trial, underscoring the role of type 2 inflammation in this complex disease
Eosinophilic esophagitis is a chronic, progressive inflammatory disease driven by type 2 inflammation that damages the esophagus and prevents it from working properly
Approval represents first indication for Dupixent in a gastrointestinal disease and fourth disease indicated overall
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the U.S. Food and Drug Administration (FDA) has approved Dupixent® (dupilumab) 300 mg weekly to treat patients with eosinophilic esophagitis (EoE) aged 12 years and older, weighing at least 40 kg. With this approval, Dupixent becomes the first and only medicine specifically indicated to treat EoE in the United States. A regulatory filing for EoE is under review by the European Medicines Agency, and submissions to regulatory authorities in additional countries are planned by the end of 2022.
"We have waited a long time for an FDA-approved treatment option for eosinophilic esophagitis – an underdiagnosed and misunderstood disease of the esophagus that can make it extremely challenging and uncomfortable to eat and swallow," said Mary Jo Strobel, Executive Director at the American Partnership for Eosinophilic Disorders (APFED). "Before today, there were no approved treatments specifically for eosinophilic esophagitis, resulting in many people needing to maintain a strict diet and live in constant fear of food getting stuck in their throat. We welcome therapeutic options that can provide much-needed relief for these patients."
EoE is a chronic inflammatory disease driven by type 2 inflammation that damages the esophagus and prevents it from working properly. For people with EoE, swallowing even small amounts of food can be a painful and worrisome choking experience. They are often left to contend with the frustration and anxiety of a constantly evolving list of foods to avoid, a poor quality of life and a higher risk of depression. In cases where EoE causes the esophagus to narrow, forced and potentially painful dilation (physical expansion) of the esophagus may be needed. In severe cases, a feeding tube may be the only option to ensure proper caloric intake and adequate nutrition. About 160,000 patients are living with EoE in the U.S. These individuals are currently treated with therapies not specifically approved for the disease, of whom approximately 48,000 continue to experience symptoms despite multiple treatments.
"It is gratifying that Dupixent, a medicine that we invented in our laboratories, is now approved in yet another disease marked by allergic or type 2 inflammation, namely eosinophilic esophagitis," said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer at Regeneron, and a principal inventor of Dupixent. "Eosinophilic esophagitis can be debilitating for patients by inflaming and damaging the esophagus and limiting the ability to eat normally. Dupixent is the first and only medicine specifically indicated to treat eosinophilic esophagitis in the United States, and today's approval marks the fourth disease for which Dupixent is now indicated, reinforcing the promise of targeting IL-4 and IL-13 to effectively treat diseases with underlying type 2 inflammation."
"Eating regularly throughout the day is essential, yet significant difficulty swallowing food is a common symptom for people living with eosinophilic esophagitis. This can be incredibly upsetting and often leads to fear of pain or choking with every meal, every day," said John Reed, M.D., Ph.D., Global Head of Research and Development at Sanofi. "A large unmet need exists for treatment options that can provide meaningful symptom relief. Our Phase 3 clinical program showed that Dupixent weekly improved the ability to swallow and reduced inflammation in the esophagus, underscoring the role of type 2 inflammation in this complex disease. This is a landmark FDA approval for patients and their caregivers who now have a new option for treating this devastating disease."
The FDA approval is based on data from a Phase 3 trial with two parts (Part A and Part B) evaluating the efficacy and safety of Dupixent 300 mg weekly, compared to placebo, in patients aged 12 years and older with EoE, weighing at least 40 kg. After 24 weeks, patients treated with Dupixent 300 mg weekly experienced the following changes in Part A and Part B, respectively:
- 69% and 64% reduction in disease symptoms from baseline compared to 32% and 41% for placebo. Disease symptoms were measured by the Dysphagia Symptom Questionnaire (DSQ), where patients receiving Dupixent experienced a 21.9- and 23.8-point clinically meaningful improvement compared to 9.6- and 13.9-point improvement for placebo.
- Approximately 10 times as many patients achieved histological disease remission (peak esophageal intraepithelial eosinophil count of ≤6 eos/high power field [hpf]) compared to placebo: 60% and 59% compared to 5% and 6% of patients receiving placebo.
The safety results were generally consistent with the known safety profile of Dupixent in its approved indications. Pooled adverse events from Parts A and B that were more commonly (≥2%) observed with Dupixent than placebo were injection site reactions (38% Dupixent, 33% placebo), upper respiratory tract infections (18% Dupixent, 10% placebo), arthralgia (2% Dupixent, 1% placebo) and herpes viral infections (2% Dupixent, 1% placebo).
The FDA evaluated the Dupixent application under Priority Review, which is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions.
The Phase 3 randomized, double-blind, placebo-controlled trial evaluated the efficacy and safety of Dupixent in patients aged 12 years and older with EoE. Part A enrolled 81 patients and evaluated Dupixent 300 mg weekly (42 treated with Dupixent and 39 with placebo). Part B enrolled 159 patients and evaluated Dupixent 300 mg weekly (80 with Dupixent and 79 with placebo).
At 24 weeks, the co-primary endpoints in Parts A and B assessed patient-reported measures of difficulty swallowing (change from baseline in the DSQ on a 0-84 scale) and esophageal inflammation (proportion of patients achieving histological disease remission, defined as peak esophageal intraepithelial eosinophil count of ≤6 eos/hpf).
Dupixent is administered as an injection under the skin (subcutaneous injection) at different injection sites. In patients aged 12 years and older, weighing at least 40 kg, with EoE, Dupixent is administered as a 300 mg dose with a pre-filled syringe or pen every week. Dupixent is intended for use under the guidance of a healthcare professional and can be given in a clinic or at home by self-administration after training by a healthcare professional. In children aged 12 to 17 years, Dupixent should be administered under the supervision of an adult.
Dupixent, which was invented using Regeneron's proprietary VelocImmune® technology, is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways and is not an immunosuppressant. The Dupixent development program has shown significant clinical benefit and a decrease in type 2 inflammation in Phase 3 trials, establishing that IL-4 and IL-13 are key and central drivers of the type 2 inflammation that plays a major role in multiple related and often co-morbid diseases. These diseases include approved indications for Dupixent such as asthma, atopic dermatitis, chronic rhinosinusitis with nasal polyposis (CRSwNP) and eosinophilic esophagitis, as well as investigational diseases such as prurigo nodularis.
Regeneron and Sanofi are committed to helping patients in the U.S. who are prescribed Dupixent gain access to the medicine and receive the support they may need with the DUPIXENT MyWay® program. For more information, please call 1-844-DUPIXENT (1-844-387-4936) or visit www.DUPIXENT.com.
Dupixent is also approved for use in certain patients with atopic dermatitis, asthma or CRSwNP in different age populations in a number of countries around the world, including the U.S., European Union and Japan. Dupixent is currently approved for one or more of these indications in more than 60 countries, and more than 400,000 patients have been treated globally.
Regeneron's VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron's President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite® technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create approximately a quarter of all original, FDA-approved or authorized fully human monoclonal antibodies currently available. This includes Dupixent, REGEN-COV® (casirivimab and imdevimab), Libtayo® (cemiplimab-rwlc), Praluent® (alirocumab), Kevzara® (sarilumab), Evkeeza® (evinacumab-dgnb) and InmazebTM (atoltivimab, maftivimab, and odesivimab-ebgn).
Dupilumab is being jointly developed by Regeneron and Sanofi under a global collaboration agreement. To date, dupilumab has been studied across more than 60 clinical trials involving more than 10,000 patients with various chronic diseases driven in part by type 2 inflammation.
In addition to the currently approved indications, Regeneron and Sanofi are studying dupilumab in a broad range of diseases driven by type 2 inflammation or other allergic processes including pediatric atopic dermatitis (6 months to 5 years of age, Phase 3), hand and foot atopic dermatitis (Phase 3), chronic obstructive pulmonary disease with evidence of type 2 inflammation (Phase 3), pediatric eosinophilic esophagitis (Phase 3), bullous pemphigoid (Phase 3), prurigo nodularis (Phase 3), chronic spontaneous urticaria (Phase 3), chronic pruritis of unknown origin (Phase 3), chronic inducible urticaria-cold (Phase 3), chronic rhinosinusitis without nasal polyposis (Phase 3), allergic fungal rhinosinusitis (Phase 3) and allergic bronchopulmonary aspergillosis (Phase 3). These potential uses of dupilumab are currently under clinical investigation, and the safety and efficacy in these conditions have not been fully evaluated by any regulatory authority.
DUPIXENT is a prescription medicine used:
- to treat adults and children 6 years of age and older with moderate-to-severe atopic dermatitis (eczema) that is not well controlled with prescription therapies used on the skin (topical), or who cannot use topical therapies. DUPIXENT can be used with or without topical corticosteroids. It is not known if DUPIXENT is safe and effective in children with atopic dermatitis under 6 years of age.
- with other asthma medicines for the maintenance treatment of moderate-to-severe eosinophilic or oral steroid dependent asthma in adults and children 6 years of age and older whose asthma is not controlled with their current asthma medicines. DUPIXENT helps prevent severe asthma attacks (exacerbations) and can improve your breathing. DUPIXENT may also help reduce the amount of oral corticosteroids you need while preventing severe asthma attacks and improving your breathing. DUPIXENT is not used to treat sudden breathing problems. It is not known if DUPIXENT is safe and effective in children with asthma under 6 years of age.
- with other medicines for the maintenance treatment of chronic rhinosinusitis with nasal polyposis (CRSwNP) in adults whose disease is not controlled. It is not known if DUPIXENT is safe and effective in children with chronic rhinosinusitis with nasal polyposis under 18 years of age.
- to treat adults and children 12 years of age and older, who weigh at least 88 pounds (40 kg), with eosinophilic esophagitis (EoE). It is not known if DUPIXENT is safe and effective in children with eosinophilic esophagitis under 12 years of age and who weigh at least 88 pounds (40 kg).
IMPORTANT SAFETY INFORMATION
Do not use if you are allergic to dupilumab or to any of the ingredients in DUPIXENT®.
Before using DUPIXENT, tell your healthcare provider about all your medical conditions, including if you:
- have eye problems
- have a parasitic (helminth) infection
- are scheduled to receive any vaccinations. You should not receive a "live vaccine" right before and during treatment with DUPIXENT.
- are pregnant or plan to become pregnant. It is not known whether DUPIXENT will harm your unborn baby.
- A pregnancy registry for women who take DUPIXENT during pregnancy collects information about the health of you and your baby. To enroll or get more information call 1-877-311-8972 or go to https://mothertobaby.org/ongoing-study/dupixent/.
- are breastfeeding or plan to breastfeed. It is not known whether DUPIXENT passes into your breast milk.
Tell your healthcare provider about all the medicines you take, including prescription and over-the- counter medicines, vitamins and herbal supplements.
Especially tell your healthcare provider if you are taking oral, topical, or inhaled corticosteroid medicines; have asthma and use an asthma medicine; or have atopic dermatitis or CRSwNP, and also have asthma. Do not change or stop your corticosteroid medicine or other asthma medicine without talking to your healthcare provider. This may cause other symptoms that were controlled by the corticosteroid medicine or other asthma medicine to come back.
DUPIXENT can cause serious side effects, including:
- Allergic reactions. DUPIXENT can cause allergic reactions that can sometimes be severe. Stop using DUPIXENT and tell your healthcare provider or get emergency help right away if you get any of the following signs or symptoms: breathing problems or wheezing, swelling of the face, lips, mouth, tongue or throat, fainting, dizziness, feeling lightheaded, fast pulse, fever, hives, joint pain, general ill feeling, itching, skin rash, swollen lymph nodes, nausea or vomiting, or cramps in your stomach-area.
- Eye problems. Tell your healthcare provider if you have any new or worsening eye problems, including eye pain or changes in vision, such as blurred vision. Your healthcare provider may send you to an ophthalmologist for an exam if needed.
- Inflammation of your blood vessels. Rarely, this can happen in people with asthma who receive DUPIXENT. This may happen 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 DUPIXENT. Tell your healthcare provider right away if you have: rash, chest pain, worsening shortness of breath, a feeling of pins and needles or numbness of your arms or legs, or persistent fever.
- Joint aches and pain. Some people who use DUPIXENT have had trouble walking or moving due to their joint symptoms, and in some cases needed to be hospitalized. Tell your healthcare provider about any new or worsening joint symptoms. Your healthcare provider may stop DUPIXENT if you develop joint symptoms.
The most common side effects include:
- Atopic dermatitis: injection site reactions, eye and eyelid inflammation, including redness, swelling, and itching, sometimes with blurred vision, and cold sores in your mouth or on your lips.
- Asthma: injection site reactions, pain in the throat (oropharyngeal pain), high count of a certain white blood cell (eosinophilia), and parasitic (helminth) infections.
- Chronic rhinosinusitis with nasal polyposis: injection site reactions, eye and eyelid inflammation, including redness, swelling, and itching, sometimes with blurred vision, high count of a certain white blood cell (eosinophilia), trouble sleeping (insomnia), toothache, gastritis, and joint pain (arthralgia).
- Eosinophilic esophagitis: injection site reactions, upper respiratory tract infections, cold sores in your mouth or on your lips, and joint pain (arthralgia).
Tell your healthcare provider if you have any side effect that bothers you or that does not go away.
These are not all the possible side effects of DUPIXENT. Call your doctor for medical advice about side
effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Use DUPIXENT exactly as prescribed by your healthcare provider. It's an injection given under the skin (subcutaneous injection). Your healthcare provider will decide if you or your caregiver can inject DUPIXENT. Do not try to prepare and inject DUPIXENT until you or your caregiver have been trained by your healthcare provider. In children 12 years of age and older, it's recommended DUPIXENT be administered by or under supervision of an adult. In children under 12 years of age, DUPIXENT should be given by a caregiver.
Please see accompanying full Prescribing Information including Patient Information.
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for nearly 35 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to nine FDA-approved treatments and numerous product candidates in development, almost all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, pain, hematologic conditions, infectious diseases and rare diseases.
Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite technologies, such as VelocImmune, which uses unique genetically humanized mice to produce optimized fully human antibodies and bispecific antibodies, and through ambitious research initiatives such as the Regeneron Genetics Center, which is conducting one of the largest genetics sequencing efforts in the world.
For additional information about the company, please visit www.regeneron.com or follow @Regeneron on Twitter.
We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people's lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY.
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