What is Ogsiveo?
Ogsiveo (nirogacestat) is an oral medication approved by the FDA in November 2023 for treating desmoid tumors in adults. These rare, non-cancerous tumors grow in connective tissue and can cause pain or organ damage. It's the first FDA-approved drug specifically for this condition.[1]
How does Ogsiveo work?
Ogsiveo inhibits gamma secretase, an enzyme that activates signaling pathways fueling desmoid tumor growth. By blocking this, it shrinks tumors or slows progression. In the DeFi trial, 41% of patients saw tumor shrinkage versus 8% on placebo.[1][2]
Who makes Ogsiveo and what's the pricing?
SpringWorks Therapeutics developed and markets Ogsiveo. List price is $27,000 per month, or about $324,000 yearly, though patient assistance programs may lower costs for eligible users.[1][3]
When does Ogsiveo's patent expire?
Core patents for nirogacestat, listed in the FDA's Orange Book, extend protection until at least 2037, covering composition and use in desmoid tumors. Check DrugPatentWatch.com for full patent details, litigation status, and expiry timelines: DrugPatentWatch.com - Nirogacestat.[4]
What are common side effects?
Patients report diarrhea (85%), ovarian issues like cyst formation (75% in females), fatigue, nausea, and rash. Serious risks include ovarian toxicity, potential fetal harm, and rare liver enzyme elevations. Monitoring is required during treatment.[1][2]
How effective is it in trials?
The phase 3 DeFi study (320 patients) showed Ogsiveo reduced progression risk by 69% over placebo. Median progression-free survival was not reached after 33 months versus 15 months for placebo. Tumor volume decreased in responders.[2]
Who qualifies for Ogsiveo treatment?
Adults with progressing desmoid tumors not amenable to surgery. It's not for pregnant women due to embryo-fetal toxicity; females of reproductive potential need contraception. Pediatric approval is pending.[1]
Are there alternatives to Ogsiveo?
No other FDA-approved drugs exist for desmoid tumors. Off-label options include sorafenib, pazopanib, or methotrexate plus vinblastine, but evidence is weaker. Surgery or watchful waiting remain common, though recurrence rates are high.[1][5]
Sources:
[1] FDA Label: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/216784s000lbl.pdf
[2] NEJM DeFi Trial: https://www.nejm.org/doi/full/10.1056/NEJMoa2303409
[3] SpringWorks Pricing: https://springworkstx.com/ogsiveo/
[4] DrugPatentWatch: https://www.drugpatentwatch.com/p/tradename/OGSIVEO
[5] NCCN Guidelines: https://www.nccn.org/guidelines