Full Press Release Details
Dupixent (dupilumab) Breakthrough Therapy designation for eosinophilic esophagitis
PARIS and TARRYTOWN, N.Y., September 14, 2020 The U.S. Food and Drug Administration (FDA) has granted
Breakthrough Therapy designation to Dupixent (dupilumab) for the treatment of patients 12 years and older with eosinophilic esophagitis (EoE). The designation for this investigational use is
based on positive results from Part A of a Phase 3 trial in patients with EoE.
There are currently no
FDA-approved medicines for EoE, a chronic and progressive type 2 inflammatory disease that damages the esophagus and prevents it from working properly. Over time, excessive type 2 inflammation causes scarring
and narrowing of the esophagus, making it difficult to swallow. If left untreated, EoE can affect a patient s ability to eat and cause food to become stuck after being swallowed (food impaction), which can lead to a medical emergency.
In the U.S. alone, there are approximately 160,000 patients with EoE who are currently being treated with various unapproved therapies or diet modification.
Of these patients, approximately 50,000 have failed multiple treatments.
Sanofi and Regeneron previously reported positive results from Part A of
the pivotal Phase 3 trial evaluating Dupixent in patients 12 years and older with EoE. Part A of the randomized, double-blind, placebo-controlled trial of 81 patients met both of its co-primary endpoints, as
well as all key secondary endpoints. Patients treated weekly with Dupixent 300 mg over a 24-week treatment period experienced a reduction in symptoms, esophageal inflammation and abnormal endoscopic findings
Part A of the trial also demonstrated safety results consistent with the known safety profile of Dupixent in its approved indications.
The EoE trial is ongoing, with additional patients enrolling in Part B as well as patients continuing in a 28-week extended active treatment period (Part C) after completing either Part A or Part B.
Breakthrough Therapy designation is designed to expedite the development and review of drugs in the U.S.
that target serious or life-threatening conditions. Drugs qualifying for this designation must show preliminary clinical evidence that the drug may demonstrate a substantial improvement on clinically significant endpoints over available therapies,
or over placebo if there are no available therapies. In 2017, Dupixent also was granted Orphan Drug designation for the potential treatment of EoE. This is given to investigational medicines intended for the treatment of rare diseases that affect
fewer than 200,000 people in the U.S.
The potential use of Dupixent in eosinophilic esophagitis is currently under clinical development, and its safety
and efficacy for this indication have not been evaluated by any regulatory authority.
Dupixent is approved in the U.S. to treat patients aged 6 years and older with
moderate-to-severe atopic dermatitis that is not well controlled with prescription therapies used on the skin (topical), or who cannot use topical therapies; for use
with other asthma medicines for the maintenance treatment of moderate-to-severe eosinophilic or oral steroid dependent asthma in patients aged 12 years and older whose
asthma is not controlled with their current asthma medicines; and for use with other medicines for the maintenance treatment of chronic rhinosinusitis with nasal polyposis (CRSwNP) in adults whose disease is not controlled. In adolescents 12 years
of age or older, it is recommended that Dupixent be administered by or under the supervision of an adult. In children younger than 12 years of age, Dupixent should be administered by a caregiver.
Outside of the U.S., Dupixent is approved for specific patients with
moderate-to-severe atopic dermatitis and certain patients with asthma in a number of other countries around the world, including the EU and Japan. Dupixent is also
approved in the EU and Japan to treat certain adults with severe CRSwNP.
Dupixent is a fully-human monoclonal antibody that inhibits the signaling of the
interleukin-4 (IL-4) and interleukin-13 (IL-13) proteins. Data from Dupixent clinical
trials have shown that IL-4 and IL-13 are key drivers of the type 2 inflammation that plays a major role in asthma, CRSwNP and atopic dermatitis.
Dupilumab Development Program
To date, dupilumab has
been studied in more than 10,000 patients across 50 clinical trials in various chronic diseases driven by type 2 inflammation. In addition to the currently approved indications, Sanofi and Regeneron are studying dupilumab in a broad range of
diseases driven by type 2 inflammation or other allergic processes, including pediatric asthma (6 to 11 years of age, Phase 3), pediatric atopic dermatitis (6 months to 5 years of age, Phase 3), eosinophilic esophagitis (Phase 3), chronic
obstructive pulmonary disease (Phase 3), bullous pemphigoid (Phase 3), prurigo nodularis (Phase 3), chronic spontaneous urticaria (Phase 3), and food and environmental allergies (Phase 2). These potential uses are investigational, and the safety and
efficacy of dupilumab in these conditions have not been evaluated by any regulatory authority. Dupilumab is being jointly developed by Regeneron and Sanofi under a global collaboration agreement.
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for over
30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to seven FDA-approved treatments and numerous product candidates in development, 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, 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.
Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness
with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.
With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.
Sanofi, Empowering Life
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limitation Dupixent (dupilumab) for the treatment of patients 12 years and older with eosinophilic esophagitis; the likelihood, timing, and scope of possible regulatory approval and
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