FDA Approves Dupixent® (dupilumab) as First Biologic Medicine for Children Aged 6 Months to 5 Years with Moderate-to-severe Atopic Dermatitis

Post to Reddit

Children treated with Dupixent and topical corticosteroids (TCS) achieved clearer skin, experienced significantly improved overall disease severity and significantly reduced itch compared to TCS alone at week 16 in a Phase 3 trial

Long-term safety data from a 52-week open-label extension trial in this age group reinforce the well-established safety profile of Dupixent observed across all other approved age groups

Dupixent is the first and only biologic medicine approved to treat moderate-to-severe atopic dermatitis from infancy through adulthood

TARRYTOWN, N.Y. and PARIS, June 7, 2022 -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the U.S. Food and Drug Administration (FDA) has approved Dupixent® (dupilumab) for children aged 6 months to 5 years with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. Regulatory filings for this age group are underway by the European Medicines Agency and regulatory authorities in additional countries.

"Moderate-to-severe atopic dermatitis in babies and young children is more than just a rash – the intense itch can make them scratch uncontrollably throughout the day and night and cause their skin to crack and bleed," said Julie Block, President and Chief Executive Officer at National Eczema Association. "Caregivers do their best to manage skincare routines multiple times a day, but for many, topical treatments are not enough. We're pleased to see how scientific innovation and research continues to address unmet needs for the atopic dermatitis community, and we're hopeful for the positive impact Dupixent can have for these children and their families."

Atopic dermatitis is a chronic type 2 inflammatory skin disease. Eighty-five to ninety percent of patients first develop symptoms before 5 years of age, which can often continue through adulthood. Symptoms include intense, persistent itch and skin lesions that cover much of the body, resulting in skin dryness, cracking, pain, redness or darkening, and crusting and oozing. In the U.S., more than 75,000 children aged 5 years and younger have uncontrolled moderate-to-severe disease and are most in need of new treatment options. Moderate-to-severe atopic dermatitis may also significantly impact the quality of life of a young child and their caregivers.

"Young children with moderate-to-severe atopic dermatitis are a significantly underserved population of patients, who spend vulnerable years of their lives suffering through the relentless and far-reaching effects of this chronic disease," said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer at Regeneron, and a principal inventor of Dupixent. "Dupixent has changed the atopic dermatitis treatment paradigm – significantly clearing skin and reducing itch – by targeting an underlying cause of this disease without broadly suppressing the immune system. Today's approval brings the proven efficacy and, importantly, well-established safety profile of Dupixent to these young children, making it the first of its kind to be approved for any U.S. patient aged six months or older living with this debilitating disease."

"Until today, treatment options in the U.S. for infants and children under the age of 6 suffering from moderate-to-severe atopic dermatitis have been limited to topical steroids – which may be associated with significant safety risks when used long-term. This has left patients and their caregivers in desperate need of medicines that can better address the chronic, long-term nature of the disease," Naimish Patel, M.D, Senior Vice President, Head of Global Development, Immunology and Inflammation at Sanofi. "These young people, and their families, often struggle to cope with the significant impact itch can have not only on the body, but on many other facets of daily life. This approval means that Dupixent, with its well-established safety and efficacy profile, is now available to some of the youngest people living with this disease."

The FDA evaluated Dupixent under Priority Review, which is reserved for medicines that represent potentially significant improvements in efficacy or safety in treating serious conditions. The approval is based on data that include a Phase 3 trial evaluating Dupixent every four weeks (200 mg or 300 mg, based on body weight) plus low-potency topical corticosteroids (TCS) or TCS alone. The trial met the primary and all secondary endpoints. At 16 weeks, patients who received Dupixent with TCS experienced the following, compared to TCS alone (placebo):

  • 28% achieved clear or almost-clear skin compared to 4% with placebo, the primary endpoint.
  • 53% achieved 75% or greater improvement in overall disease severity from baseline compared to 11% with placebo, the co-primary endpoint outside of the U.S.
  • 48% achieved clinically meaningful reduction in itch compared to 9% with placebo.

The safety profile of Dupixent observed through 16 weeks in children aged 6 months to 5 years was similar to the safety profile in patients 6 years and older with atopic dermatitis. The long-term safety profile of Dupixent in children aged 6 months to 5 years through 52 weeks was also similar to the safety profile observed in the pivotal trial and consistent with what was observed in older patients with atopic dermatitis. Hand-foot-and-mouth disease and skin papilloma were, respectively, reported in 5% and 2% of Dupixent patients aged 6 months to 5 years, and none of these cases led to treatment discontinuation.

About the Dupixent Trial  
The Phase 3 randomized, double-blind, placebo-controlled trial evaluated the efficacy and safety of Dupixent added to standard-of-care low-potency TCS compared to low-potency TCS alone (placebo) in 162 children aged 6 months to 5 years with uncontrolled moderate-to-severe atopic dermatitis.

The primary endpoints assessed the proportion of patients achieving an Investigator's Global Assessment (IGA) score of 0 (clear) or 1 (almost clear) and 75% improvement in Eczema Area and Severity Index (EASI-75) at week 16. Additional outcome measures included itch reduction, which was assessed using a caregiver-reported 0 to 10 Numerical Rating Scale, with a clinically meaningful improvement defined as ≥4-point improvement at week 16.

Children who completed the trials were eligible to enroll in an open-label extension trial to assess the safety and efficacy of long-term treatment with Dupixent in this age group.

About Dupixent  
Dupixent is administered as an injection under the skin (subcutaneous injection) at different injection sites. In patients aged 6 months to 5 years, Dupixent is administered with a pre-filled syringe every four weeks based on weight (200 mg for children ≥5 to <15 kg and 300 mg for children ≥15 to <30 kg). 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 younger than 12 years of age, Dupixent should be administered by a caregiver if given at home. Dupixent does not require initial lab testing or ongoing lab monitoring.

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 approved for use in certain patients with atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis (CRSwNP) or eosinophilic esophagitis in different age populations in a number of countries around the world. Dupixent is currently approved across these indications in the U.S. and for one or more of these indications in the European Union and Japan and more than 60 countries. More than 400,000 patients have been treated globally.

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, CRSwNP and eosinophilic esophagitis, as well as investigational diseases such as prurigo nodularis.

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 approximately one in five of all original, FDA-approved fully human monoclonal antibodies currently available. This includes REGEN-COV® (casirivimab and imdevimab), Dupixent® (dupilumab), Libtayo® (cemiplimab-rwlc), Praluent® (alirocumab), Kevzara® (sarilumab), Evkeeza® (evinacumab-dgnb) and Inmazeb™ (atoltivimab, maftivimab and odesivimab-ebgn).

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 prurigo nodularis, pediatric eosinophilic esophagitis, hand and foot atopic dermatitis, chronic inducible urticaria-cold, chronic spontaneous urticaria, chronic pruritis of unknown origin, chronic obstructive pulmonary disease with evidence of type 2 inflammation, chronic rhinosinusitis without nasal polyposis, allergic fungal rhinosinusitis, allergic bronchopulmonary aspergillosis 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 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 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 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.
  • 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, cold sores in your mouth or on your lips, and high count of a certain white blood cell (eosinophilia).
  • 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 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.

About Regeneron  
Regeneron is a leading biotechnology company that invents, develops and commercializes 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 numerous FDA-approved treatments and 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 more information, please visit www.Regeneron.com or follow @Regeneron on Twitter.

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 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

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. These statements concern, and these risks and uncertainties include, among others, the impact of SARS-CoV-2 (the virus that has caused the COVID-19 pandemic) on Regeneron's business and its employees, collaborators, and suppliers and other third parties on which Regeneron relies, Regeneron's and its collaborators' ability to continue to conduct research and clinical programs, Regeneron's ability to manage its supply chain, net product sales of products marketed or otherwise commercialized by Regeneron and/or its collaborators or licensees (collectively, "Regeneron's Products"), and the global economy; the nature, timing, and possible success and therapeutic applications of Regeneron's Products and product candidates being developed by Regeneron and/or its collaborators or licensees (collectively, "Regeneron's Product Candidates") and research and clinical programs now underway or planned, including without limitation Dupixent® (dupilumab) for the treatment of moderate-to-severe atopic dermatitis in children aged 6 months to 5 years; uncertainty of the utilization, market acceptance, and commercial success of Regeneron's Products (such as Dupixent) and Regeneron's Product Candidates and the impact of studies (whether conducted by Regeneron or others and whether mandated or voluntary), including the studies discussed or referenced in this press release, on any of the foregoing or any potential regulatory approval of Regeneron's Products (such as Dupixent) and Regeneron's Product Candidates; the likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron's Product Candidates and new indications for Regeneron's Products, such as Dupixent for the treatment of hand and foot atopic dermatitis, prurigo nodularis, eosinophilic esophagitis, chronic inducible urticaria-cold, chronic spontaneous urticaria, bullous pemphigoid, chronic obstructive pulmonary disease with evidence of type 2 inflammation, chronic rhinosinusitis without nasal polyposis, allergic fungal rhinosinusitis, allergic bronchopulmonary aspergillosis, peanut allergy, and other potential indications; the ability of Regeneron's collaborators, licensees, suppliers, or other third parties (as applicable) to perform manufacturing, filling, finishing, packaging, labeling, distribution, and other steps related to Regeneron's Products and Regeneron's Product Candidates; the ability of Regeneron to manage supply chains for multiple products and product candidates; safety issues resulting from the administration of Regeneron's Products (such as Dupixent) and Regeneron's Product Candidates in patients, including serious complications or side effects in connection with the use of Regeneron's Products and Regeneron's Product Candidates in clinical trials; determinations by regulatory and administrative governmental authorities which may delay or restrict Regeneron's ability to continue to develop or commercialize Regeneron's Products and Regeneron's Product Candidates, including without limitation Dupixent; ongoing regulatory obligations and oversight impacting Regeneron's Products, research and clinical programs, and business, including those relating to patient privacy; the availability and extent of reimbursement of Regeneron's Products from third-party payers, including private payer healthcare and insurance programs, health maintenance organizations, pharmacy benefit management companies, and government programs such as Medicare and Medicaid; coverage and reimbursement determinations by such payers and new policies and procedures adopted by such payers; competing drugs and product candidates that may be superior to, or more cost effective than, Regeneron's Products and Regeneron's Product Candidates; the extent to which the results from the research and development programs conducted by Regeneron and/or its collaborators or licensees may be replicated in other studies and/or lead to advancement of product candidates to clinical trials, therapeutic applications, or regulatory approval; unanticipated expenses; the costs of developing, producing, and selling products; the ability of Regeneron to meet any of its financial projections or guidance and changes to the assumptions underlying those projections or guidance; the potential for any license, collaboration, or supply agreement, including Regeneron's agreements with Sanofi, Bayer, and Teva Pharmaceutical Industries Ltd. (or their respective affiliated companies, as applicable) to be cancelled or terminated; and risks associated with intellectual property of other parties and pending or future litigation relating thereto (including without limitation the patent litigation and other related proceedings relating to EYLEA® (aflibercept) Injection, Dupixent, Praluent® (alirocumab), and REGEN-COV® (casirivimab and imdevimab)), other litigation and other proceedings and government investigations relating to the Company and/or its operations, the ultimate outcome of any such proceedings and investigations, and the impact any of the foregoing may have on Regeneron's business, prospects, operating results, and financial condition. A more complete description of these and other material risks can be found in Regeneron's filings with the U.S. Securities and Exchange Commission, including its Form 10-K for the year ended December 31, 2021 and its Form 10-Q for the quarterly period ended March 31, 2022. Any forward-looking statements are made based on management's current beliefs and judgment, and the reader is cautioned not to rely on any forward-looking statements made by Regeneron. Regeneron does not undertake any obligation to update (publicly or otherwise) any forward-looking statement, including without limitation any financial projection or guidance, whether as a result of new information, future events, or otherwise.

Regeneron uses its media and investor relations website and social media outlets to publish important information about the Company, including information that may be deemed material to investors. Financial and other information about Regeneron is routinely posted and is accessible on Regeneron's media and investor relations website (http://newsroom.regeneron.com) and its Twitter feed (http://twitter.com/regeneron).

Sanofi Disclaimers or Forward-Looking Statements   
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates regarding the marketing and other potential of the product, or regarding potential future revenues from the product. Forward-looking statements are generally identified by the words "expects", "anticipates", "believes", "intends", "estimates", "plans" and similar expressions. Although Sanofi's management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, unexpected regulatory actions or delays, or government regulation generally, that could affect the availability or commercial potential of the product, the fact that product may not be commercially successful, the uncertainties inherent in research and development, including future clinical data and analysis of existing clinical data relating to the product, including post marketing, unexpected safety, quality or manufacturing issues, competition in general, risks associated with intellectual property and any related future litigation and the ultimate outcome of such litigation, and volatile economic and market conditions, and the impact that COVID-19 will have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole.  Any material effect of COVID-19 on any of the foregoing could also adversely impact us. This situation is changing rapidly and additional impacts may arise of which we are not currently aware and may exacerbate other previously identified risks. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under "Risk Factors" and "Cautionary Statement Regarding Forward-Looking Statements" in Sanofi's annual report on Form 20-F for the year ended December 31, 2021. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.

Regeneron Contacts:

Media Relations

Hannah Kwagh

Tel: +1 914-847-6314

Hannah.Kwagh@regeneron.com

Investor Relations

Vesna Tosic

Tel: +1 914-847-5443

Vesna.Tosic@regeneron.com 



Sanofi Contacts:

Media Relations

Sally Bain

Tel: +1 617-834-6026

Sally.Bain@sanofi.com

Investor Relations

Eva Schaefer-Jansen

Tel: +33 7 86 80 56 39

eva.schaefer-jansen@sanofi.com

  
Arnaud Delepine

Tel: +33 (0)6 73 69 36 93

arnaud.delepine@sanofi.com

  
Corentine Driancourt

Tel: +33 (0)6 40 56 92

corentine.driancourt@sanofi.com

  
Felix Lauscher

Tel: +1 908-612-7239

felix.lauscher@sanofi.com

  
Priya Nanduri

Tel: +1 617-764-6418

priya.nanduri@sanofi.com

  
Nathalie Pham

Tel: +33 (0)7 85 93 30 17

nathalie.pham@sanofi.com

Cision View original content:https://www.prnewswire.com/news-releases/fda-approves-dupixent-dupilumab-as-first-biologic-medicine-for-children-aged-6-months-to-5-years-with-moderate-to-severe-atopic-dermatitis-301563406.html

SOURCE Regeneron Pharmaceuticals