Zaltenibart Receives FDA Rare Pediatric Disease Designation for C3 Glomerulopathy

The FDA granted rare pediatric disease designation for zaltenibart (Omeros Corporation; OMS906) for the treatment of complement 3 glomerulopathy (C3G), which is caused by dysregulation of the alternative pathway of complement. The rare and progressive renal disorder commonly impacts children and young adults and there is no approved treatment to prevent and aid C3G.¹

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“C3G is devastating for children as well as for adults, and our receipt of FDA’s rare pediatric disease designation is a welcome acknowledgment of zaltenibart as a potential therapeutic for this disease that has no approved treatment,” Gregory A Demopulos, chairman and CEO of Omeros, said in a news release.¹

According to the National Kidney Foundation, C3G is a kidney disease that has 2 forms: dense deposit disease (DDD) and C3 glomerulonephritis (C3GN). Symptoms of DDD typically appear earlier than C3GN signs, but both initially begin in adulthood. The most common symptoms of C3G include hematuria, proteinuria, edema, gout, recurrent infections, oliguria, hypertension, and fatigue, according to study authors.²

If C3G is not treated, about 70% of children with the disease and 30% to 50% of impacted adults are estimated to develop worsening of proteinuria and advance to end-stage renal disease in 10 years following the diagnosis.³

Zaltenibart could be an effective treatment option for C3G as the most proximal inhibitor of the alternative pathway. The study authors noted that the role of zaltenibart is to “block mannan-binding lectin-associated serine protease-3 (MASP-3), the key activator of the alternative pathway, stopping the conversion of pro-complement factor D (pro-CFD) to mature CFD.”¹

Researchers anticipate a phase 3 clinical trial to begin next year assessing zaltenibart for the treatment of C3G. However, the drug was previously granted FDA organ drug designation for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disease.¹ The blood disorder occurs when the immune system attacks and damages red blood cells and platelets. Study authors noted that without proper treatment the disease will result in hemolytic anemia, chronic kidney disease or thrombosis.⁴ A phase 3 trial is also underway for zaltenibart and its role in treating PNH, according to study authors.¹

“With zaltenibart clinical studies ongoing in both PNH and C3G and preparations underway to begin Phase 3 trials, we look forward to bringing zaltenibart to market, expanding its list of targeted indications and demonstrating its advantages over other alternative pathway inhibitors,” Demopulos siad in a news release.¹

REFERENCES

1. FDA Grants Rare Pediatric Disease Designation to Omeros’ MASP-3 Inhibitor Zaltenibart for Treatment of C3 Glomerulopathy. Omeros. News release. October 24, 2024. Accessed October 25, 2024. https://www.businesswire.com/news/home/20241024637899/en/FDA-Grants-Rare-Pediatric-Disease-Designation-to-Omeros%E2%80%99-MASP-3-Inhibitor-Zaltenibart-for-Treatment-of-C3-Glomerulopathy

2. Complement 3 Glomerulopathy (C3G): Knowing the Signs and Symptoms. National Kidney Foundation. Accessed October 25, 2024. https://www.kidney.org/kidney-topics/complement-3-glomerulopathy-c3g-knowing-signs-and-symptoms#:~:text=Complement%203%20glomerulopathy%20(C3G)%20is,be%20inherited%20or%20not%20inherited.

3. Complete Renal Recovery in Pediatric Patient with C3 Glomerulonephritis: A Case Report. Karger. September 2, 2021. Accessed October 25, 2024. https://karger.com/cnd/article/11/3/261/827723/Complete-Renal-Recovery-in-Pediatric-Patient-with.

4. Paroxysmal Nocturnal Hemoglobinuria. April 25, 2022. Accessed October 25, 2024. https://my.clevelandclinic.org/health/diseases/22871-paroxysmal-nocturnal-hemoglobinuria.