All posts by Mary Jo Strobel

Dupixent® Receives FDA Approval to Treat EoE in People Ages 12 and Older

Announcements, APFED Blog, In the News, Press Releases, Research, Uncategorized, What's New

Dupixent® Receives FDA Approval to Treat EoE in People Ages 12 and Older

Injection is the first indicated treatment for EoE

 

May 21, 2022:  APFED (The American Partnership for Eosinophilic Disorders) is pleased to share the exciting news that the first treatment for eosinophilic esophagitis (EoE) has been approved by the U.S. Food and Drug Administration (FDA) during National Eosinophil Awareness Week.

The FDA approved Dupixent® (dupilumab), jointly developed by pharmaceutical companies Sanofi and Regeneron, for treatment of adults and children 12 years and older with EoE. This chronic inflammatory condition is characterized by the presence of white blood cells called eosinophils in the esophagus.  Eosinophils cause inflammation in the esophagus and damage to the tissue, leading to symptoms of difficulty swallowing, food getting stuck in the esophagus, pain, vomiting, and malnutrition.  Treatments to date have included eliminating allergy-causing food from the diet, proton pump inhibitors, and off-label use of swallowed steroids.

APFED congratulates Sanofi and Regeneron for their commitment to developing Dupixent® and for funding the LIBERTY EoE TREET clinical studies, which demonstrated significant decrease of the number of eosinophils in the esophagus as well as improved symptoms in patients with EoE treated with Dupixent® compared to placebo. APFED would also like to thank the FDA for its priority review for this breakthrough treatment that has the potential to improve the lives of people living with EoE, and to all those who selflessly participated in research and clinical trials so that developments such as dupilumab could advance.

“The fact that this news comes during National Eosinophil Awareness Week is incredible,” said APFED’s Executive Director Mary Jo Strobel. “Fifteen years ago, APFED set out to establish an official awareness week, which was formally recognized by the U.S. House of Representatives through House Bill 296. The purpose of the week is to create awareness and educate the general public and the medical community about eosinophil-associated diseases. With greater awareness, a supportive community develops, and the need for research and improved therapies is amplified. The FDA’s decision to grant approval to Dupixent® to treat EoE is very welcome news to our community.”

“APFED was founded in 2001 with the mission to support patients and families affected by EoE and other eosinophil-associated disorders by educating patients, providers, and the public about these conditions, advocating for patients’ needs, and supporting research,” said APFED’s President Dr. Wendy Book. “Since 2006, thanks to community donations, APFED has awarded nearly $2 million in grants to investigators to advance research in eosinophilic disorders through a rigorous application process.” Many of the research projects APFED funded involved research of the immune and inflammatory pathways involved in the development of EoE. Having the pathways to inflammation identified helps guide potential future therapies.

Dr. Prad Tummala, Chairman of APFED’s Board of Directors, adds, “APFED hopes that the current success of Dupixent® is the first of many therapies that will be discovered as a result of ongoing research in the field of eosinophilic disorders. APFED depends on the generosity of donors to continue to support research and hopes that this exciting announcement of the first FDA-approved therapy indicated for EoE treatment will inspire more members of the community to donate to research, as it demonstrates the value of investing in it.”

Ryan Piansky, a young adult who was diagnosed with EoE in early childhood, shares the excitement patients and families feel about the FDA approval. “APFED has always provided hope to patients and families living with eosinophilic diseases. As a patient with EoE who met with members of Congress to help pass the National Eosinophil Awareness Week resolution, I am thrilled to hear of the first FDA approved treatment indicated for EoE. APFED’s commitment to finding treatments for these rare diseases provides a beacon of hope.”

To learn more about APFED’s HOPE on the Horizon Research Program and to make a donation, please visit apfed.org.

Read More

  • FDA Press Release: FDA approves first treatment for eosinophilic esophagitis, a chronic immune disorder
  • Sanofi Press Release: FDA approves Dupixent® (dupilumab) as first treatment for adults and children aged 12 and older with eosinophilic esophagitis

2022 Annual Meeting of AAAAI

APFED Blog, What's New

AAAAI represents allergists, asthma specialists, clinical immunologists, allied health professionals and others with a special interest in the research and treatment of allergic and immunologic diseases. AAAAI has more than 7,000 members around the globe.

APFED attended the AAAAI 2022 annual meeting held in Phoenix, AZ, Feb. 25-28 as an invited speaker. We also awarded two awards for the best research abstracts for EGID, attended advocacy meetings, and had a booth in the exhibit hall where we could share patient friendly material with clinicians.

Breakthroughs in Understanding and Treating EGID

An all-day symposium to present EGID information and research was held on February 24. The program was organized by the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR) and the International Gastrointestinal Eosinophil Researchers (TIGERS).

Attendees learned information about the diagnosis, epidemiology, and the role of the allergy in EGID, and pipeline treatments. There was also discussion on research that is shedding light onto cellular and molecular processes with these conditions, monitoring the progression of EGID through time, dietary aspects, the patient experience, and new therapies and treatments on the horizon.

As an active member of CEGIR, APFED’s Executive Director, Mary Jo Strobel, gave a presentation during this symposium to highlight ways that doctors and patients can work in partnership with one another to advance education, awareness, advocacy and research, as well as resources available to help people who have EGID.

There were presentations and discussions regarding EGIDs and associations with allergic disease.  EoE is highly associated with allergic diseases and elimination diets seem to work for many with EoE. On the other hand, there are differences at the cellular and molecular level between EoE and other EGID, which may make dietary therapy less successful.

Attendees were also informed of recent international consensus surrounding what subsets of eosinophilic gastrointestinal diseases are called for both clinical and research purposes.

Throughout AAAAI sessions, scientists discussed current knowledge and research of the etiology, pathology, and genetic and environmental factors that might affect the development of EoE, as well as data related to therapies in various stages of clinical trial for eosinophil-associated disease.

Dr. Benjamin Wright, an APFED HOPE Pilot Grant recipient who is studying the association of environmental factors and its impact on cells involved in EoE, presented his latest findings in the “Featured Poster Session” at AAAAI. His poster presentation was “Oral Detergent Exposure Induces Eosinophilic Inflammation in the Esophagus.” These early findings show that an ingredient in detergents, called SDS, reduces the functionality of epithelial cells and may be a factor to the development of EoE.

Special Committees

APFED attended special interest EGID Task Force committee meeting to participate in discussions on ways the medical community can support those with eosinophil-associated disease, and also met with patient advocacy organizations serving people with allergic conditions to discuss advocacy and policy work, and aligning initiatives to help our communities, including the Medical Nutrition Equity Act and communicating patient needs and perspectives to the FDA.

2022 AAAAI/APFED Best Abstract on EGIDs Awards

APFED offered two $750 awards to recognize outstanding research abstracts on eosinophilic gastrointestinal disease presented at the AAAAI meeting this year. The awards are intended to help defray travel costs to the meeting so that the researchers may present their findings to their peers. Abstract award winners may go on to successfully compete for grants from APFED or from other funding mechanisms.

  • Marc E. Rothenberg, MD PhD FAAAAI Cincinnati Children’s Hospital Medical Center “Dupilumab Improves Clinical and Histologic Aspects of Disease in Adult and Adolescent Patients with Eosinophilic Esophagitis at Week 24: Results from Part B of the 3-Part LIBERTY EoE TREET Study”
  • Gencer Kurt, MD Aarhus University Hospital “Perinatal Factors Increase Risk of Eosinophilic Esophagitis – A Nationwide Case Control Study”

The awards were made possible thanks to the generous community donations to APFED’s HOPE on the Horizon Research Program.

Additional highlights surrounding presentations and research presented at AAAAI will be published in the Spring 2022 issue of EoSolutions newsletters for APFED subscribers.

International Consensus Recommendations EGID Nomenclature

APFED Blog, Research, What's New

International consensus surrounding what subsets of eosinophilic gastrointestinal diseases (EGIDs) should now be called for both clinical and research purposes was recently published in the Journal of Gastroenterology and Hepatology.

Eosinophilic gastrointestinal disease (EGID) will continue be the umbrella term for eosinophilic inflammation in the GI tract when there is not another known cause.

The consensus indicated more specific terms should be used to describe has previously been called “eosinophilic gastroenteritis.”

These changes to the terms used to describe subsets of patients and conditions help characterize the specific areas of the GI tract that are affected. These include:

  • Eosinophilic Gastritis (EoG) = stomach
  • Eosinophilic Enteritis (EoN) = small bowel
  • Eosinophilic Colitis (EoC) = colon

“Eosinophilic Gastroenteritis” was previously used as a “catch-all” term for eosinophilia that was found in the stomach and/or small intestine, and should not be primarily used, with the terms above being used instead. The new international consensus also more granularly identifies areas of the GI tract that are affected:

  • Eosinophilic Duodenitis (EoD) = duodenum
  • Eosinophilic Jejunitis (EoJ) = jejunum
  • Eosinophilic Ileitis (EoI)  = ileum

This groundbreaking publication was spearheaded by CEGIR, a federally-funded research consortia in which APFED is a member.

Response to FDA denial of topical oral viscous formulation of budesonide

Advocacy, What's New

In December 2021, APFED reported that the U.S. Food and Drug Administration (FDA) denied the TAK-721 New Drug Application, indicating the drug – a budesonide oral suspension –  would not be approved in its present form. According to the drug developer, Takeda, the FDA had also recommended additional clinical study to address their feedback.

TAK-721 is a novel, topical oral viscous formulation of budesonide, specifically formulated to treat localized inflammation in the esophagus caused by EoE. The drug sponsor has not made public the Complete Response Letter generated by the FDA regarding its application for TAK-721.  Given the pathway laid out by FDA regarding drug approval for EoE, the fact that oral budesonide has good safety profile and is approved for use to treat EoE in other countries and approved in the U.S. to treat other GI inflammatory conditions, APFED, along with other patient advocacy groups, sent a joint communication to the FDA to express our disappointment and confusion regarding the denial, and requested the FDA reconsider their decision regarding TAK 721 and make public the explanation for its decision not to approve.

We will continue to advocate and share information as it becomes available. In the interim, we encourage you to continue to share with us your experiences with mixing budesonide slurry for treatment of eosinophilic esophagitis, so that we may share compiled feedback with the FDA.

 

Industry News: Drug Development

Announcements, In the News, Research, What's New

 

FDA denies approval of TAK-721 (budesonide oral suspension) for EoE

Takeda, the pharmaceutical company developing TAK-721 (budesonide oral suspension) for the treatment of EoE, announced that the U.S. Food and Drug Administration (FDA) responded to the TAK-721 New Drug Application, indicating the drug cannot be approved in its present form.

Takeda reports that the FDA recommended an additional clinical study to help resolve their feedback.

“We are disappointed by the outcome of the FDA’s review of TAK-721, and that EoE patients will still be without a treatment option that the FDA has approved as safe and effective,” said Ramona Sequeira, President, U.S. Business Unit and Global Portfolio Commercialization, Takeda, in a press release issued by the company. “Takeda is assessing the details of the CRL and evaluating a regulatory path forward.”

 

Lirentelimab met histologic co-primary endpoints but missed symptomatic co-primary endpoints in both ENIGMA and KRYPTOS studies

Allakos Inc., a biotechnology company developing lirentelimab (AK002) for the treatment of eosinophil and mast cell-related diseases, reported Phase 3 data from ENIGMA 2 (a study of lirentelimab in patients with eosinophilic gastritis and/or eosinophilic duodenitis) and Phase 2/3 data from KRYPTOS (a study of lirentelimab in patients with eosinophilic esophagitis.

Both studies met their histologic endpoints, but did not achieve statistical significance on the patient reported symptoms endpoints.

In a company press release, Dr. Craig Paterson, MD, Chief Medical Officer of Allakos states, “Although the EGID results are surprising and disappointing, we will continue to analyze the data to understand the results and to determine the path forward for lirentelimab in EGIDs.”

 

Tezspire approved for severe asthma in ages 12+

Tezspire (tezepelumab) has been approved in the U.S. for people aged 12 years and older with severe asthma. Tezspire is the first biologic approved for severe asthma without phenotype (e.g., eosinophilic or allergic) or biomarker limitations. This therapy is also in development for other potential indications, including eosinophilic esophagitis. Learn more.