FDA Grants Breakthrough Therapy Designation for Nipocalimab to Treat Sjögren Disease

The FDA granted breakthrough therapy designation for nipocalimab (Johnson & Johnson) as treatment of moderate-to-severe Sjögren disease (SjD), making it the first and only therapy granted this designation for this disease state. SjD is a chronic autoantibody disease, typically identified by dry eyes and dry mouths. According to Mayo Clinic, the condition accompanies rheumatoid arthritis, lupus, and other immune system disorders.^1,2

Sjogren Disease, FDA, Nipocalimab, Lupus, Rheumatoid Arthritis | Image Credit: © fizkes | stock.adobe.com

"[The] announcement marks an important step forward in the continued research and development of nipocalimab, the first investigational FcRn blocker to demonstrate positive results in a phase 2 study in adult patients with moderate-to-severe Sjögren disease," Terence Rooney, MD, vice president and rheumatology and immunology disease area leader at Johnson & Johnson Innovative Medicine, said in a news release. "With no treatments currently approved that may directly address the underlying cause(s) of the disease, innovation is critically needed to improve patient outcomes in Sjögren disease."¹

SjD is more common in women, and most individuals are older than 40 years at the time of diagnosis. Although dry eyes and dry mouth are the primary symptoms, individuals may also experience joint pain, swelling, and stiffness, swollen salivary glands, skin rashes or dry skin, vaginal dryness, persistent dry cough, and prolonged fatigue. Other parts of the body can be damaged by SjD, including joints, thyroid, kidneys, liver, lungs, skin, and nerves.²

The designation is supported by data from the phase 2 DAHLIAS (NCT04968912) study, evaluating the efficacy and safety of the drug for patients with moderate-to-severe SjD. The data supports further investigation through a phase 3 study, which the company reports is currently underway. Investigators included a total of 163 individuals who received randomized treatment of the drug at 5 mg/kg, 15 mg/kg, or the placebo every 2 weeks through week 22.^1,3

According to the data, nipocalimab demonstrated statistically significant and clinically meaningful improvement in Clinical European League Against Rheumatism Sjögren’s Syndrome Disease Activity Index score compared with the placebo at 24 weeks. This response was evident as early as week 4, increasing throughout the treatment period. Additionally, there were improvements in the secondary outcomes at week 24, including multiple organ assessments, physician assessments, and composite tools for clinical trial end point as well as improvements in symptoms, which included mouth, eye, and vaginal dryness. Safety and tolerability remained consistent with other clinical trials.⁴

Serious adverse events were reported in 7.5% of patients for the 5 mg/kg group, 7.4% for the 15 mg/kg group, and 5.4% for the placebo group. Severe infections or infections requiring intravenous anti-infectives occurred in 3.8%, 1.9%, and 1.8% of individuals, respectively.³

The drug previously received breakthrough therapy designation for the treatment of alloimmunized pregnant individuals with high risk of early-onset severe hemolytic disease of the fetus and newborn and fast track designation for fetal neonatal alloimmune thrombocytopenia in pregnant adults who are alloimmunized.^5,6

REFERENCES

1. Nipocalimab is the first and only investigational therapy granted U.S. FDA Breakthrough Therapy Designation for the treatment of adults living with moderate-to-severe Sjögren's disease. News release. Johnson and Johnson. November 11, 2024. Accessed November 18, 2024. https://www.prnewswire.com/news-releases/nipocalimab-is-the-first-and-only-investigational-therapy-granted-us-fda-breakthrough-therapy-designation-for-the-treatment-of-adults-living-with-moderate-to-severe-sjogrens-disease-302301655.html

2. Mayo Clinic. Sjogren’s syndrome. August 2, 2022. Accessed November 18, 2024. https://www.mayoclinic.org/diseases-conditions/sjogrens-syndrome/symptoms-causes/syc-20353216

3. Gottenberg JE, Sivils K, Campbell K, Idokogi J, et al. LBA0010 efficacy and safety of nipocalimab, an anti-FcRn monoclonocal antibody, in primary sjogren’s disease: results from a phase 2, multicenter, randomized, placebo-controlled, double-blind study (DAHLIAS). Ann Rheum Dis. 2024. doi:https://doi.org/10.1136/annrheumdis-2024-eular.LBA90

4. Late-breaking results show nipocalimab significantly improves Sjögren’s disease activity in a phase 2 study. News release. Johnson and Johnson. June 15, 2024. Accessed November 18, 2024. https://www.jnj.com/media-center/press-releases/late-breaking-results-show-nipocalimab-significantly-improves-sjogrens-disease-activity-in-a-phase-2-study

5. Hunter E. FDA Grants Breakthrough Therapy Designation to Nipocalimab for the Treatment of Rare Disease in Pregnancy. Pharmacy Times. February 9, 2024. Accessed November 18, 2024. https://www.pharmacytimes.com/view/fda-grants-breakthrough-therapy-designation-to-nipocalimab-for-the-treatment-of-rare-disease-in-pregnancy

6. Gallagher A. FDA Grants Fast Track Designation to Nipocalimab for Fetal Neonatal Alloimmune Thrombocytopenia. Pharmacy Times. March 26, 2024. Accessed November 18, 2024. https://www.pharmacytimes.com/view/fda-grants-fast-track-designation-to-nipocalimab-for-fetal-neonatal-alloimmune-thrombocytopenia