Dupixent® (dupilumab) Recommended for EU Approval by the CHMP to Treat Eosinophilic Esophagitis (EoE) in Children as Young as 1 Year Old

Rhea-AI Summary

Regeneron Pharmaceuticals and Sanofi announced that the European Medicines Agency's CHMP has recommended the expanded approval of Dupixent® (dupilumab) in the EU for eosinophilic esophagitis (EoE) in children aged 1 to 11 years. The recommendation is based on a Phase 3 trial showing significantly greater histological remission in children treated with Dupixent compared to placebo. If approved, Dupixent would be the first and only medicine in the EU indicated for EoE in this age group.

The trial demonstrated sustained results for up to one year, with caregivers observing improvements in EoE signs and symptoms. The safety profile was consistent with that observed in adolescents and adults. The European Commission is expected to make a final decision in the coming months.

Positive

• CHMP recommended expanded approval of Dupixent for EoE in children aged 1-11 years
• Phase 3 trial showed significantly greater histological remission in children treated with Dupixent vs placebo
• Results were sustained for up to one year in the trial
• If approved, Dupixent would be the first and only medicine in the EU for EoE in this age group

Negative

• None.

Insights

Medical Research Analyst

The CHMP's positive recommendation for Dupixent in treating EoE in children as young as 1 year old is a significant development. The Phase 3 trial results showing histological remission in children are particularly noteworthy. This could potentially expand the market for Dupixent, as it would be the first and only medicine in the EU for this indication in this age group.

The consistent safety profile with adult and adolescent populations is reassuring. However, investors should note that COVID-19 was among the more common adverse events, which might warrant further investigation. The long-term efficacy demonstrated in Part B of the trial is a strong point, potentially leading to sustained revenue if approved.

Financial Analyst

This development could have a positive impact on Regeneron (NASDAQ: REGN) and Sanofi's financials. Expanding Dupixent's indications to younger patients could increase the drug's market size significantly. EoE is a chronic condition, potentially leading to long-term prescriptions and recurring revenue.

However, investors should consider that the European Commission's final decision is still pending. While CHMP recommendations are often followed, it's not guaranteed. Additionally, the pricing strategy for this new indication will be crucial, balancing market penetration with profitability. The competitive landscape should also be monitored, as this approval could attract new entrants to the pediatric EoE market.

Market Research Analyst

The potential approval of Dupixent for EoE in children as young as 1 year old represents a significant market opportunity. Being the first and only medicine in this category gives Regeneron and Sanofi a first-mover advantage in a potentially underserved market. The unmet medical need in pediatric EoE could drive rapid adoption if approved.

However, market penetration may face challenges such as healthcare provider education and parental concerns about using biologics in young children. The companies will need to invest in awareness campaigns and support programs to maximize uptake. Long-term market success will depend on real-world efficacy data and the potential entry of competitors in this space.

Recommendation based on a Phase 3 trial showing a significantly greater proportion of children on Dupixent achieved histological remission compared to placebo, consistent with improvements seen in adults and adolescents

If approved, Dupixent would be the first and only medicine in the EU indicated for EoE in this age group

TARRYTOWN, N.Y. and PARIS, Sept. 20, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the expanded approval of Dupixent^® (dupilumab) in the European Union (EU) for eosinophilic esophagitis (EoE) in children down to 1 year of age. The recommendation is for 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. The European Commission is expected to announce a final decision in the coming months. Dupixent is already approved in the EU for certain adults and adolescents aged 12 years and older with EoE.

The positive CHMP opinion is supported by a two-part (Part A and B) EoE KIDS Phase 3 trial in children aged 1 to 11 years. In Part A, a significantly greater proportion of children receiving weight-based doses of Dupixent achieved histological disease remission at week 16, compared to placebo, with results sustained for up to one year in Part B. At week 16, caregivers of children treated with Dupixent also observed improvements in the frequency and severity of EoE signs, and fewer days with at least one sign of EoE, compared to placebo. These data established a bridge showing the response to Dupixent in children with EoE is similar to that of the approved adult and adolescent EoE populations.

The safety results in the EoE KIDS trial were generally consistent with the known safety profile of Dupixent in adolescents and adults with EoE. AEs 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.

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

The use of Dupixent in children aged 1 to 11 years with EoE is investigational in the EU and is not yet approved.

About EoE
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 and there are 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 Dupixent
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.

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), EoE, prurigo nodularis, chronic spontaneous urticaria and chronic obstructive pulmonary disease (COPD) in different age populations. More than 1,000,000 patients are being treated with Dupixent globally.

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. 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 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.
  

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, or prurigo nodularis 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:

• 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.
  

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 http://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.

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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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FAQ

What did the CHMP recommend for Dupixent (REGN) in treating eosinophilic esophagitis?

The CHMP recommended expanded approval of Dupixent for eosinophilic esophagitis (EoE) in children aged 1 to 11 years in the European Union.

What were the key results of the Phase 3 trial for Dupixent (REGN) in children with EoE?

The Phase 3 trial showed a significantly greater proportion of children receiving Dupixent achieved histological disease remission at week 16 compared to placebo, with results sustained for up to one year.

When is the European Commission expected to make a final decision on Dupixent (REGN) for EoE in children?

The European Commission is expected to announce a final decision on Dupixent for EoE in children in the coming months following the CHMP recommendation.

What is the current approval status of Dupixent (REGN) for EoE in the EU?

Dupixent is currently approved in the EU for certain adults and adolescents aged 12 years and older with EoE. The new recommendation is for children aged 1 to 11 years.