Approval based on Phase 3 data showing significantly more children aged 1 to 11 years on Dupixent achieved histological disease remission at 16 weeks compared to placebo, which was sustained up to one year

Dupixent is the first-ever medicine in the EU indicated to treat these young patients, who persistently struggle to eat at a critical stage in life where growth is crucial

TARRYTOWN, N.Y. and PARIS, Nov. 06, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the European Commission (EC) has approved Dupixent^® (dupilumab) to treat eosinophilic esophagitis (EoE) in children as young as 1 year of age. Specifically, the approval covers children aged 1 to 11 years who weigh at least 15 kg and who are inadequately controlled by, intolerant to, or who are not candidates for conventional medicinal therapy. This expands the initial approval in the European Union (EU) for EoE in adults and adolescents and makes Dupixent the first and only medicine indicated to treat these young patients. Dupixent is also approved in this young age group in the U.S. and Canada.

“Young children with eosinophilic esophagitis are at the beginning of their life-long journey with a disease that challenges their ability to eat,” said Roberta Giodice, President, ESEO Italia. “Parents of these children have often relied on restrictive diets that do not specifically address the disease and can stunt their growth at a critical time in development that could impact them for years to come. We are pleased that research continues and offers new treatment options to improve the quality of their care.”

“Eosinophilic esophagitis presents a unique challenge in young children, who struggle with their basic ability to eat during a time in their lives where proper nutrition is essential for growth and development,” said George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer at Regeneron, and a principal inventor of Dupixent. “This approval will bring the proven efficacy and demonstrated safety profile of Dupixent to this vulnerable, young population that has already been established in older EoE patients and has the potential to transform the standard of care for children with EoE who previously had no therapies specifically approved for them.”

The approval is based on the two-part (Part A and B) EoE KIDS Phase 3 trial in children aged 1 to 11 years, which established a bridge showing the response to Dupixent in children with EoE is similar to that of the approved adult and adolescent populations. In Part A, children who received a higher dose of Dupixent (n=37) based on a weight-based dosing regimen experienced the following outcomes, compared to placebo (n=34) at 16 weeks:

• 68% achieved histological disease remission (≤6 eosinophils/high power field) compared to 3%, the primary endpoint. These results were sustained for up to one year in Part B of the trial.
• 86% reduction in peak esophageal intraepithelial eosinophil count from baseline compared to a 21% increase.
• Reductions in abnormal endoscopic findings and disease severity and extent (as measured at the microscopic level).
• Nominally significant improvement in the frequency and severity of EoE signs, and numerical reduction in days with at least one sign of EoE, based on caregiver-reported outcomes.

The safety results in the EoE KIDS trial were generally consistent with the known safety profile of Dupixent in adolescents and adults with EoE. The most common adverse reactions for Dupixent overall are injection site reactions, conjunctivitis, conjunctivitis allergic, arthralgia, oral herpes and eosinophilia. In addition, the adverse reaction of injection site bruising was reported in EoE. In patients aged 1 to 11 years, adverse events more commonly observed with Dupixent (≥10%) in either weight-based dosing regimen compared to placebo during Part A were COVID-19, nausea, injection site pain and headache. The long-term safety profile of Dupixent evaluated in Part B was similar to that observed during Part A.

“Up to half of all children in the EU with eosinophilic esophagitis remain uncontrolled despite existing standard of care treatment options, and, as a result, many of these young patients struggle to maintain weight due to serious symptoms such as difficulty swallowing and vomiting,” said Houman Ashrafian, M.D., Ph.D., Executive Vice President, Head of Research and Development at Sanofi. “This milestone provides an important new treatment for pediatric patients who were previously without options specifically approved for their disease. With this novel approach to addressing an underlying cause of eosinophilic esophagitis, Dupixent has the potential to give these young children a better chance to thrive.”

About Eosinophilic Esophagitis
EoE is a chronic, progressive disease associated with type 2 inflammation that is thought to be responsible for damaging the esophagus and impairing its function. Diagnosis is difficult, as symptoms can be mistaken for other conditions leading to delays in diagnosis. EoE can severely impact a child’s ability to eat and may also cause vomiting, abdominal pain, difficulty swallowing, decreased appetite and challenges thriving. Continuous management of EoE may be needed to reduce the risk of complications and disease progression.

About the Dupixent Pediatric Eosinophilic Esophagitis Trial
The EoE KIDS Phase 3 trial was a randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of Dupixent in children aged 1 to 11 years with EoE. Part A enrolled 71 patients and evaluated Dupixent at a weight-based dose regimen, compared to placebo, for 16 weeks. Part B was a 36-week extended active treatment period in which eligible children from Part A in the Dupixent group continued treatment, while those in the placebo group switched to Dupixent. Patients included in this trial were previously treated and did not respond to conventional medicinal therapies, including proton pump inhibitors and/or swallowed topical corticosteroids.

The primary endpoint was histologic remission at 16 weeks, and secondary endpoints included assessments of endoscopic and histopathologic measures of the severity of disease along with caregiver-reported clinical signs and symptoms of EoE. The 108-week open-label extension period (Part C) to evaluate longer-term outcomes was recently completed.

Results from the trial were published in The New England Journal of Medicine.

About Dupixent
Dupixent is an injection administered under the skin (subcutaneous injection) at different injection sites. In patients aged 1 to 11 years with EoE, Dupixent is administered every other week (200 mg for children ≥15 to <30 kg, 300 mg for children ≥30 to <40 kg) or every week (300 mg for children ≥40 kg), based on weight. Dupixent is intended for use under the guidance of a healthcare professional and can be given in a clinic or at home administered by a caregiver after training by a healthcare professional.

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 two of the key and central drivers of the type 2 inflammation that plays a major role in multiple related and often co-morbid diseases.

Dupixent has received regulatory approvals in more than 60 countries in one or more indications including certain patients with atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyps (CRSwNP), eosinophilic esophagitis (EoE), prurigo nodularis, chronic spontaneous urticaria (CSU), and chronic obstructive pulmonary disease (COPD) in different age populations. More than 1,000,000 patients are being treated with Dupixent globally.¹

About Regeneron’s VelocImmune Technology 
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 co-Founder, 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 a substantial proportion of all original, FDA-approved or authorized fully human monoclonal antibodies. This includes REGEN-COV^® (casirivimab and imdevimab), Dupixent, Libtayo^® (cemiplimab-rwlc), Praluent^® (alirocumab), Kevzara^® (sarilumab), Evkeeza^® (evinacumab-dgnb), Inmazeb^® (atoltivimab, maftivimab and odesivimab-ebgn) and Veopoz™ (pozelimab-bbfg).

Dupilumab Development Program 
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 in Phase 3 trials, including chronic pruritus of unknown origin and bullous pemphigoid. 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. 

U.S. INDICATIONS 
DUPIXENT is a prescription medicine used:

• to treat adults and children 6 months of age and older with moderate-to-severe eczema (atopic dermatitis or AD) 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 months 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. 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 polyps (CRSwNP) in adults and children 12 years of age and older whose disease is not controlled. It is not known if DUPIXENT is safe and effective in children with chronic rhinosinusitis with nasal polyps under 12 years of age.
• to treat adults and children 1 year of age and older with eosinophilic esophagitis (EoE), who weigh at least 33 pounds (15 kg). It is not known if DUPIXENT is safe and effective in children with eosinophilic esophagitis under 1 year of age, or who weigh less than 33 pounds (15 kg).
• to treat adults with prurigo nodularis (PN). It is not known if DUPIXENT is safe and effective in children with prurigo nodularis under 18 years of age.
• with other medicines for the maintenance treatment of adults with inadequately controlled chronic obstructive pulmonary disease (COPD) and a high number of blood eosinophils (a type of white blood cell that may contribute to your COPD). DUPIXENT is used to reduce the number of flare-ups (the worsening of your COPD symptoms for several days) and can improve your breathing. It is not known if DUPIXENT is safe and effective in children with chronic obstructive pulmonary disease under 18 years of age.

DUPIXENT is not used to relieve sudden breathing problems and will not replace an inhaled rescue medicine.

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, chronic rhinosinusitis with nasal polyps, eosinophilic esophagitis, prurigo nodularis, or chronic obstructive pulmonary disease and also have asthma. Do not change or stop your other medicines, including corticosteroid medicine or other asthma medicine, without talking to your healthcare provider. This may cause other symptoms that were controlled by those medicines 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:

• Eczema: injection site reactions, eye and eyelid inflammation, including redness, swelling, and itching, sometimes with blurred vision, dry eye, cold sores in your mouth or on your lips, and high count of a certain white blood cell (eosinophilia).
• Asthma: injection site reactions, high count of a certain white blood cell (eosinophilia), pain in the throat (oropharyngeal pain), and parasitic (helminth) infections.
• Chronic Rhinosinusitis with Nasal Polyps: 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), gastritis, joint pain (arthralgia), trouble sleeping (insomnia), and toothache.
• Eosinophilic Esophagitis: injection site reactions, upper respiratory tract infections, cold sores in your mouth or on your lips, and joint pain (arthralgia).
• Prurigo Nodularis: eye and eyelid inflammation, including redness, swelling, and itching, sometimes with blurred vision, herpes virus infections, common cold symptoms (nasopharyngitis), dizziness, muscle pain, and diarrhea.
• Chronic Obstructive Pulmonary Disease: injection site reactions, common cold symptoms (nasopharyngitis), high count of a certain white blood cell (eosinophilia), viral infection, back pain, inflammation inside the nose (rhinitis), diarrhea, gastritis, joint pain (arthralgia), toothache, headache, and urinary tract infection.

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 6 months to less than 12 years of age, DUPIXENT should be given by a caregiver.

Please see accompanying full Prescribing Information including Patient Information.

About Regeneron
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most 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, neurological diseases, hematologic conditions, infectious diseases, and rare diseases.

Regeneron pushes the boundaries of scientific discovery and accelerates drug development using our proprietary technologies, such as VelociSuite^®, which produces optimized fully human antibodies and new classes of bispecific antibodies. We are shaping the next frontier of medicine with data-powered insights from the Regeneron Genetics Center^® and pioneering genetic medicine platforms, enabling us to identify innovative targets and complementary approaches to potentially treat or cure diseases.

For more information, please visit www.Regeneron.com or follow Regeneron on LinkedIn, Instagram, Facebook or X.

About Sanofi 
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 the world, 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.

Regeneron Forward-Looking Statements and Use of Digital Media 
This press release includes forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Regeneron Pharmaceuticals, Inc. (“Regeneron” or the “Company”), and actual events or results may differ materially from these forward-looking statements. Words such as “anticipate,” “expect,” “intend,” “plan,” “believe,” “seek,” “estimate,” variations of such words, and similar expressions are intended to identify such forward-looking statements, although not all forward-looking statements contain these identifying words. 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Sanofi Disclaimers or Forward-Looking Statements 
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¹ Data on File