Drug Discovery and Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Women in Pharma and Biotech
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • Resources
    • Video features
    • Podcast
    • Voices
    • Views
    • Webinars
  • Pharma 50
    • 2025 Pharma 50
    • 2024 Pharma 50
    • 2023 Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50
  • Advertise
  • SUBSCRIBE

Rare diseases, immense needs: J&J’s mission to change the landscape

By Brian Buntz | February 27, 2024

Johnson & JohnsonRare diseases may seem niche, but their impact is far from small. An estimated 7,000 rare diseases exist, collectively affecting a 300 million people worldwide.  This immense burden of disease, coupled with a profound lack of treatment options, underscores the urgent need for innovation. “Actually, the total burden of disease and unmet medical need [for rare disease] is really high,” emphasizes Dr. Katie Abouzahr, vice president, autoantibody portfolio and maternal fetal disease area leader at Johnson & Johnson Innovative Medicine. In recognition of Rare Disease Day on February 29 in 2024, we spoke with Abouzahr to explore how Johnson & Johnson Innovative Medicine aims to tackle these challenges.

The profound scarcity of treatments for the thousands of known rare diseases drives Johnson & Johnson’s commitment to this area. Abouzahr highlights that many of these diseases still lack advanced therapies, emphasizing the “incredibly high” unmet medical need. She explains that her return to her physician roots fuels her desire to impact patients’ lives. “A lot of my motivation is in areas where there isn’t an approved advanced therapy,” she said, which drives a quest to do “something really new to address unmet need.”

Addressing the unmet need in rare diseases

Katie Abouzahr

Katie Abouzahr

Autoantibodies, antibodies that mistakenly target the body’s own tissues, play a critical role in various autoimmune diseases, including myasthenia gravis (MG) and hemolytic disease of the fetus and newborn (HDFN). These misguided immune components can cause significant damage, leading to a diverse range of debilitating symptoms. Consequently, the conditions are also a pillar of J&J’s strategy of targeting rare disease.

At the heart of J&J’s autoantibody-based rare disease approach is nipocalimab, a therapeutic candidate designed to reduce the harmful effects of autoantibodies. Its unique mechanism may offer potential benefits across a spectrum of autoantibody-driven diseases.  “One of the unique things about autoantibody-driven diseases is that they do share a common underlying mechanism,” Abouzahr said. This mechanism is driven by IgG antibodies, the primary type responsible for these harmful autoimmune responses. This commonality means that by targeting IgG antibodies, “you can use this one approach to impact many different diseases,” she added. 

Targeting autoantibodies: A common thread in rare diseases

J&J is now actively developing nipocalimab to tackle a range of autoantibody-related conditions, tapping its ability to modulate the detrimental effects of IgG antibodies. For instance, nipocalimab is in a phase 3 trial for warm autoimmune hemolytic anemia. It is also undergoing phase 2 trials for hemolytic disease of the fetus and newborn, Sjögren’s syndrome, systemic lupus erythematosus, idiopathic inflammatory myopathy, and as a combination therapy for rheumatoid arthritis.

Abouzahr emphasized the importance of collaboration in tackling the rarity and diversity of rare diseases with both patients and other crucial stakeholders. This focus extends beyond patient communities to include non-governmental organizations (NGOs) and government agencies. Abouzahr describes patients as especially vital in the rare disease space. “These patients often become experts in navigating their condition, from diagnosis to finding appropriate specialists,” she said. By listening to them, J&J can gain insights to inform its clinical trial programs in rare disease. “Involving patients in these discussions is crucial because rare diseases necessitate a more personalized, tailored approach than [prevalent diseases],” Abouzahr concluded.


Filed Under: Uncategorized
Tagged With: autoantibodies, autoimmune diseases, clinical trials, IgG antibodies, Johnson & Johnson, nipocalimab, Rare Diseases
 

About The Author

Brian Buntz

As the pharma and biotech editor at WTWH Media, Brian has almost two decades of experience in B2B media, with a focus on healthcare and technology. While he has long maintained a keen interest in AI, more recently Brian has made making data analysis a central focus, and is exploring tools ranging from NLP and clustering to predictive analytics.

Throughout his 18-year tenure, Brian has covered an array of life science topics, including clinical trials, medical devices, and drug discovery and development. Prior to WTWH, he held the title of content director at Informa, where he focused on topics such as connected devices, cybersecurity, AI and Industry 4.0. A dedicated decade at UBM saw Brian providing in-depth coverage of the medical device sector. Engage with Brian on LinkedIn or drop him an email at bbuntz@wtwhmedia.com.

Related Articles Read More >

Why Regeneron’s Lynozyfic FDA approval validates its extreme R&D thesis
Parallel Bio’s $21M in Series A will drive aim to trim $2B and 9 years from drug development timelines
Glass vial, pipette and woman scientist in laboratory for medical study, research or experiment. Test tube, dropper and professional female person with chemical liquid for pharmaceutical innovation
From 1.5% to 5.9%: Deloitte digs into what’s fueling Big Pharma’s R&D IRR climb
Recce targets A$15.8M to advance anti-infectives into Phase 3 trials
“ddd
EXPAND YOUR KNOWLEDGE AND STAY CONNECTED
Get the latest news and trends happening now in the drug discovery and development industry.

MEDTECH 100 INDEX

Medtech 100 logo
Market Summary > Current Price
The MedTech 100 is a financial index calculated using the BIG100 companies covered in Medical Design and Outsourcing.
Drug Discovery and Development
  • MassDevice
  • DeviceTalks
  • Medtech100 Index
  • Medical Design Sourcing
  • Medical Design & Outsourcing
  • Medical Tubing + Extrusion
  • Subscribe to our E-Newsletter
  • Contact Us
  • About Us
  • R&D World
  • Drug Delivery Business News
  • Pharmaceutical Processing World

Copyright © 2025 WTWH Media LLC. All Rights Reserved. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media
Privacy Policy | Advertising | About Us

Search Drug Discovery & Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Women in Pharma and Biotech
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • Resources
    • Video features
    • Podcast
    • Voices
    • Views
    • Webinars
  • Pharma 50
    • 2025 Pharma 50
    • 2024 Pharma 50
    • 2023 Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50
  • Advertise
  • SUBSCRIBE