dm+d

Unassigned

New Medicines

IgA nephropathy in adults with persistent proteinuria, at high risk of disease progression despite being on a stable dose of an ACEI/ARB

Information

New molecular entity
Travere Therapeutics
Travere Therapeutics

Development and Regulatory status

Phase III Clinical Trials
Pre-registration (Filed)
Pre-registration (Filed)
Yes
Yes
Oct 22 FDA have requested changes to proposed risk evaluation mitigation strategy (REMS) to include liver monitoring. This is expected to delay the decision date by three months, a decision was previously anticipated in November [10].
Aug 22EMA accept conditional MAA for treatment of IgAN [9].
May 22FDA grants Priority Review with PDUFA target action date 17 Nov 2022 [8]
Mar 22Travere file NDA with US FDA for accelerated approval of sparsentan for treatment of IgAN. Submission is supported by positive interim results from PIII PROTECT trial [4].
Aug 21Travere plan to file for accelerated approval in US H1 2022, and to submit an application for conditional marketing authorisation in Europe [3].
Feb 21Granted Orphan Drug designation in EU [7].
Jan 21Granted Orphan Drug designation in US [6].

Category

Combined angiotensin receptor blocker and endothelin receptor type A blocker
IgAN is characterised by the deposition of IgA-containing immune complexes in glomeruli of the kidney, causing an inflammatory response that leads to proteinuria and haematuria. IgAN is the most common type of primary glomerulonephritis worldwide. Estimated incidence of IgAN is 2.5 per 100,000 individuals per year among adults [1].
IgA nephropathy in adults with persistent proteinuria, at high risk of disease progression despite being on a stable dose of an ACEI/ARB
Oral

Trial or other data

Mar 22Travere announce PIII PROTECT trial has met a pre-specified interim primary efficacy endpoint, demonstrating a > 3-fold reduction of proteinuria from baseline at 36 weeks vs. irbesartan (p<0.0001) [3].
Mar 22Estimated primary completion date of PIII PROTECT trial is March 23 [5].
Jan 19Recruitment to PIII PROTECT trial (NCT03762850) evaluating the effect and safety of sparsentan in the treatment of adults with IgA nephropathy (IgAN) begins. Approximately 380 adults with IgAN who have persistent overt proteinuria and remain at high risk of disease progression despite being on a stable dose of an ACEI/ARB will be enrolled in the study globally. They will be randomly assigned 1:1 to either sparsentan 200mg or irbesartan 150 mg as a single oral morning dose. Dose will be increased to 400mg/300mg after 2 weeks and continued to week 110 [2].

Focal segmental glomerulosclerosis

Information

Licence extension / variation
Travere Therapeutics
Travere Therapeutics

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Combined angiotensin receptor blocker and endothelin receptor type A blocker
Focal segmental glomerulosclerosis, or FSGS, is a rare disorder it is defined by progressive scarring of the kidney associated with proteinuria and often leads to end-stage renal disease and renal failure. In the absence of standard treatment, patients are treated with angiotensin receptor blockers, angiotensin converting enzyme inhibitors, calcineurin inhibitors, and steroids [1].
Focal segmental glomerulosclerosis
Oral

Further information

Yes

Evidence based evaluations