How likely is tvtx new drug will be approved

The likelihood of Travere Therapeutics' new drug, FILSPARI (sparsentan), being approved for the treatment of IgA nephropathy (IgAN) is currently high. Here's an analysis based on the latest information:

1. FDA Approval Status: FILSPARI has already received accelerated approval from the U.S. Food and Drug Administration (FDA) in February 2023, based on reduction in proteinuria. The continued approval of FILSPARI may be contingent upon confirmation of a clinical benefit in the ongoing Phase 3 PROTECT Study, which is designed to demonstrate whether FILSPARI slows kidney function decline.
2. Clinical Trial Results: The PROTECT Study showed that FILSPARI demonstrated long-term kidney function preservation and achieved a clinically meaningful difference in estimated glomerular filtration rate (eGFR) total and chronic slope versus the active comparator, irbesartan. Although the trial narrowly missed statistical significance on a secondary endpoint called estimated glomerular filtration rate (eGFR) total slope, the results were generally considered clinically meaningful.

3. Market Opportunity: IgA nephropathy is a leading cause of kidney failure, affecting an estimated 150,000 people in the U.S., with approximately 30,000 to 50,000 of such patients addressable under the indication approved via accelerated approval. The potential to serve a significant patient population bolsters the market opportunity for FILSPARI.
4. Regulatory Path: Travere Therapeutics plans to submit a supplemental New Drug Application (sNDA) in the first half of 2024 for full approval in the U.S., which is based on 2-year confirmatory results from the Phase 3 PROTECT Study. The FDA has granted priority review to this sNDA, with a PDUFA target action date of September 5, 2024.
5. Market Dynamics: The approval of FILSPARI would provide a new treatment option for patients with IgA nephropathy, addressing a significant unmet need. The drug's non-immunosuppressive nature differentiates it from existing therapies and could offer a more favorable safety profile.
6. Financial Outlook: Travere Therapeutics has reported strong financial performance, with net product sales of FILSPARI totaling $15 million in the fourth quarter of 2023, and expectations of approximately $128 million in net product sales for the fiscal year 2023. This financial stability supports the company's ability to invest in drug development and potential regulatory submissions.

In conclusion, while there have been setbacks in clinical development for FILSPARI, the overall clinical trial results, regulatory progress, and market potential suggest a high likelihood of approval for the full FDA approval of FILSPARI in the treatment of IgA nephropathy.