The FDA has indicated that it is not currently planning to hold an advisory committee meeting to discuss the application and has assigned a Prescription Drug User Fee Act (PDUFA) target action date of Nov. 17 of this year.
‘New Standard of Care’
“For decades people living with IgA nephropathy have had limited treatment options while facing a progression toward end-stage kidney disease. If approved, sparsentan could become the first non-immunosuppressive steroid therapy for this condition,” said Eric Dube, Ph.D., president and chief executive officer of Travere Therapeutics. “We aspire to ultimately position sparsentan as a new standard of care.”
The NDA submission for sparsentan is supported by results from the ongoing Phase 3 PROTECT Study, one of the largest interventional studies to date in IgAN.
The PROTECT Study evaluating sparsentan in 404 patients with persistent proteinuria met its pre-specified interim primary efficacy endpoint.
After 36 weeks of treatment, patients receiving sparsentan achieved a mean reduction in proteinuria from baseline of 49.8 percent, compared to 15.1 percent for irbesartan-treated patients.
Preliminary results at the time of the interim assessment suggested that sparsentan had been generally well-tolerated in the study and consistent with its overall observed safety profile.
“There is a clear need for a non-immunosuppressive treatment option that helps to delay disease progression to dialysis or the need for a transplant for these patients – and as a healthcare provider it’s exciting to see this innovation is potentially on the horizon,” Rovin added.
Market Reaction and Promising Pipeline
The NDA approval announcement was good news to investors.
In addition to the possible FDA approval of sparsentan for IgAN, which is a leading cause of kidney failure affecting more than 100,000 people in the U.S., Travere is actively working on approval in the EU and initiated work needed to support potential future registration in Japan where the disease is one of the leading causes of acute nephritis.
“We’re hoping to submit our first file with European regulators in the middle of this year. We’re on track for that, and we’re excited to have partnered with a leader in Europe for kidney disease, Vifor Pharma,” Dube said.
Travere is also investigating sparsentan’s safety and efficacy in a Phase 3 study for the treatment of focal segmental glomerulosclerosis (FSGS) – a rare proteinuric kidney disorder that is defined by progressive scarring of the kidney and often leads to end-stage kidney disease.
Sparsentan has been granted Orphan Drug Designation for the treatment of IgAN and FSGS in the U.S. and Europe.
Dedicated to Rare Diseases
Travere Therapeutics has a history of success in rare diseases. In fact, the company’s motto is: “We are in Rare for Life.”
Travere currently has four approved medications available in the U.S. for treating rare kidney and liver diseases – including diseases that only affect a few hundred patients.
“For 95% of the patients with a rare disease, they do not have an approved therapy,” Dube said, whose personal dedication to rare disease stems from his own experience being diagnosed with two rare cancers over 20 years ago.
“My oncologist told me he only knew of seven other cases ever with this type of tumor and it doesn’t respond to treatment. So basically, the strategy was ‘I hope the surgery caught it early enough.’ And that still resonates with me,” he said. “So many patients with a rare disease don’t have that luxury and they don’t have an approved therapy. So for us to say that we are in rare for life really communicates our commitment to this community.”
Travere Therapeutics, Inc.
CEO: Eric Dube
HEADQUARTERS: San Diego
BUSINESS: Researcher, maker of therapies for rare diseases.
REVENUE: $227.5 million (2021)
STOCK: TVTX (NASDAQ)
NOTABLE: Travere currently has four approved medications for treating rare kidney and liver diseases.