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

Rare diseases may seem niche, but their impact is far from small. An estimated 7,000 rare diseases exist, collectively affecting a staggering 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 and 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 “incred…

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Nipocalimab shows promise in RA subgroups in phase 2a IRIS-RA study

Johnson & Johnson’s nipocalimab, which works by targeting the neonatal Fc receptor (FcRn), has the potential to treat an array of autoimmune conditions. But the antibody recently hit a snag in the first-ever clinical study of an FcRn inhibitor in rheumatoid arthritis (RA), missing its primary endpoint. The development has sparked debate within the rheumatology community. “Now, there are some highly respected physicians who’ve said, ‘This is a failed trial,’” said Dr. Peter Taylor, the Norman Collisson Professor of Musculoskeletal Sciences at the University of Oxford. “I think that’s an incorrect interpretation.”

Johnson & Johnson is also pursuing a host of other autoimmune indications for nipocalimab. The FDA has granted it Fast Track designation for hemolytic disease of the fetus and newborn (HDFN) and orphan drug status for HDFN while EMA granted it orphan medicinal product designation for HDFN.

Breaking down the data from IRIS-RA

Johnson …

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