NAFLD in Children and Alternative Development Paths in NASH

Sponsored by: Covance

Focused on:

  • Drug Development
  • Chronic Liver Disease

Date: 30 October


Time: 3PM London/11AM New York

Part 1: Lessons Learned in Pediatric NAFLD Clinical Development: Clinical and Regulatory Considerations Part 2: Repurposing Drugs for a NASH Indication: Alternative Development Paths

Non-alcoholic fatty liver disease (NAFLD) is the most common chronic liver disease in children and adults in industrialized countries. Children with NAFLD are particularly at risk of complications in their lifetime. Besides liver-related morbidity, NAFLD is also associated with an increased risk of cardiovascular disease, type 2 diabetes and mortality at adult age. However, despite the high prevalence and serious complications, diagnosing and staging of disease remains complicated due to a lack of accurate screening tools and non-invasive methods to detect fibrosis.

In the last 5 years, a number of clinical trials have assessed the safety and efficacy of different novel medications to treat NAFLD in the adult population. Several drugs are in phase III development for adult patients with NAFLD and may receive approval by Regulatory Agencies within the next few years. Pharmaceutical companies have already started planning for trials of new agents in children with NAFLD. Some trial design issues are important to consider for the successful development of future treatments in the paediatric population, including optimal primary endpoints beyond improvement in surrogate biomarkers
such as progression to cirrhosis and end-stage liver disease, the effect of confounding variables such as puberty and paediatric-specific safety-related adverse events such as effects on growth and development.

The 505(b)(2) drug development path is an abbreviated or hybrid path of regulatory approval in the U.S. It offers a unique opportunity for rapid approval of drugs since it eliminates most nonclinical studies as well as extensive safety and efficacy tests. Examples of ideal candidate compounds are: drugs with potential new indications; drugs with changes in dosage form, strength, formulation, dosing regimen or route of administration; new combination products or prodrugs of an existing drug. The products approved under the 505(b)(2) pathway can also sometimes qualify for several types of market exclusivity such as orphan drug exclusivity, new chemical entity exclusivity and pediatric exclusivity.

Join our 90-minute educational webinar to gain an in-depth overview of pediatric NAFLD and get insights on developing strategies that help navigate today’s challenging clinical trial landscape. You will learn about the epidemiology of this progressive liver disease and its common phenotypes, such as nonalcoholic steatohepatitis (NASH), along with the near-term prospects in current NAFLD/NASH clinical trials. The presenters will also share valuable lessons learned in NAFLD clinical development in children and adolescents along with important regulatory considerations for pediatric trials in NAFLD.
Finally, join this webinar to learn about a faster, less expensive path to market, a commercially attractive path to approval under certain specific circumstances

Presented by

Miriam Vos, MD, MSPH,

Assistant Professor of Pediatrics, Emory University School of Medicine Pediatric Hepatologist, Children’s Healthcare of Atlanta

Dr. Vos is an assistant professor of Pediatrics in the School of Medicine at Emory University as well as the director of Graduate Studies for the PhD Nutrition and Health Sciences Program in the Laney Graduate School and the director of the Pediatric Fatty Liver Program, Children’s Healthcare of Atlanta.
Dr. Vos’ research focuses on pediatric nonalcoholic fatty liver disease (NAFLD), a chronic liver disease closely associated with insulin resistance and obesity. Her research has demonstrated the importance of dietary sugar in the pathogenesis of NAFLD in children and the benefit of sugar reduction as a primary therapy for it. Ongoing work includes investigations of effective ways to prevent NAFLD, better non-invasive diagnostics, combination therapies and long term outcomes of pediatric NAFLD.

Johannes Taminiau , MD,

Pediatric Committee (PDCO), European Medicines Agency (EMA) Pediatric gastroenterologist, University Hospital Antwerp/Paola Children's Hospital (Belgium)

Dr. Jan Taminiau was a pediatric gastroenterologist and emeritus associate professor in the Department of Pediatric Gastroenterology, Hepatology and Nutrition at Emma Childrens Hospital, Amsterdam and a pediatric gastroenterologist at the University Hospital Antwerp and Paola Children’s Hospital in Belgium. He is a consultant in the Department of Pediatrics at the University of Antwerp in Antwerp, Belgium and a member of the pediatric committee at the European Medicines Evalutation Agency in Amsterdam, Netherlands.

Donna Griebel, MD,

Regulatory Drug Consultant, Griebel and Rosebraugh Consulting LLC.

Donna Griebel, MD is a regulatory drug consultant in Griebel and Rosebraugh Consulting LLC. Dr. Griebel served as director of the Division of Gastroenterology and Inborn Errors Products (DGIEP) in FDA's Center for Drug Evaluation and Research (CDER) for 10 years, where she managed regulatory review of a diverse portfolio of programs, encompassing small molecule and biologic drugs (including biosimilars) intended to treat a broad range of conditions, including liver and biliary diseases (e.g., nonalcoholic steatohepatitis, primary sclerosing cholangitis, pancreatic insufficiency and hepatic encephalopathy).

Dr. Griebel has extensive regulatory experience in drug development issues associated with new molecular entities, 505(b)(2) pathway, pediatric drug development, patient reported outcome instrument development, expedited programs for drugs intended to treat serious conditions and combination product development. She served on CDER's Medical Policy Council (MPC) and the MPC's Subcommittee on Safety Outcome Trials. She led the CDER team that revised a CDER MAPP on review of informed consent documents.

Claudia Filozof, MD, PhD,

Vice President, Liver Therapeutic Area Head, Global Clinical Development, Covance

Dr. Claudia Filozof joined Covance in 2014 and consults on nonalcoholic fatty liver disease and steatohepatitis (NAFLD/NASH) research projects, as well as provides medical oversight. She is board-certified in nutrition and metabolism and has broad expertise in diabetes, obesity and related metabolic disorders, including in NAFLD/NASH. Her career spans almost 20 years in academic research, as well as 15 years of experience in the pharmaceutical industry.

Dr. Filozof led multiple drug development programs in obesity and other metabolic diseases and has been actively involved in NASH clinical development since 2011. She is an active member of the Liver Forum, member of the American Association for the Study of Liver Diseases steatohepatitis working group, and the European Association for the Study of Diabetes NAFLD group. She has published multiple manuscripts on obesity, lipids, diabetes and 4 recent review articles in clinical development in NASH.

Key Learning Objectives

  • Understand the current trends, trials and epidemiology of NAFLD/NASH
  • Hear about valuable lessons learned in NAFLD pediatric clinical development
  • Get insights on key regulatory considerations for pediatric trials in NAFLD
  • Learn how the 505(b)(2) drug approval pathway can provide an advantage for sponsors


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