Also known as: FSGS
Focal segmental glomerulosclerosis is a kidney disorder characterized by scarring in some glomeruli, leading to proteinuria and progressive decline in kidney function. It can be primary or secondary to other conditions.
BioMarin reported Q2 2026 revenues of $990 million, up 20% Y/Y, and raised guidance. It filed an sNDA for VOXZOGO in hypochondroplasia and advanced BMN 333, BMN 820, and BMN 351.
January and March 2026 drug pipeline updates included Phase 3 results, Fast Track designations and Priority Review decisions across multiple disease areas. Mavacamten, teplizumab-mzwv, anifrolumab, AXS-05, gedatolisib and AFTX-201 were among the programs with notable milestones.
The FDA granted full approval to Filspari to reduce proteinuria in patients aged eight and older with FSGS without nephrotic syndrome. The decision was based on Phase III DUPLEX data versus irbesartan.
The FDA is set to make decisions on four drug applications in April 2026, including Orca-T for hematological malignancies, orforglipron for obesity, sparsentan for kidney disease, and a doravirine/islatravir combination for HIV-1.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07268638 |
A Study of Praliciguat in Participants With Focal Segmental Glomerulosclerosis (FSGS) |
RECRUITING | PHASE2 |
| NCT07220083 |
A Study to Find Out if BI 764198 Helps Adults and Adolescents With a Kidney Condition Called Focal Segmental Glomerulosclerosis (FSGS) |
RECRUITING | PHASE3 |
| NCT07219121 |
Sparsentan in Posttransplant Immunoglobulin A Nephropathy or Focal Segmental Glomerulosclerosis |
ACTIVE_NOT_RECRUITING | PHASE4 |
| NCT06664814 |
An Open-Label Phase 2 Study of N-Acetyl-D-Mannosamine (ManNAc) in Subjects With Primary Focal Segmental Glomerulosclerosis |
RECRUITING | PHASE2 |
| NCT06500702 |
A Study to Evaluate the Efficacy and Safety of Frexalimab, Brivekimig, or Rilzabrutinib in Participants Aged 16 to 75 Years With Primary Focal Segmental Glomerulosclerosis or Minimal Change Disease |
RECRUITING | PHASE2 |
| NCT05650619 |
Recurrence Post-transplant Observational Study in Focal Segmental Glomerulosclerosis and Minimal Change Disease |
RECRUITING | |
| NCT05003986 |
Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases |
RECRUITING | PHASE2 |
| NCT04573920 |
Atrasentan in Patients With Proteinuric Glomerular Diseases |
ACTIVE_NOT_RECRUITING | PHASE2 |
| NCT04571658 |
NEPTUNE Match Study |
RECRUITING | NA |
| NCT02194582 |
Genetic Causes of FSGS, Nephrotic Syndrome, or Kidney Failure |
ACTIVE_NOT_RECRUITING |
