Ogsiveo (Nirogacestat) Emerges As First Targeted Therapy For Desmoid Tumors Offering New Hope For Patients
April 12, 2025 — In a landmark advancement for rare cancer treatment, Ogsiveo® (nirogacestat) has solidified its position as the first and only FDA-approved therapy for adults with progressing desmoid tumors, a rare and often debilitating soft-tissue condition.
Developed by SpringWorks Therapeutics, Ogsiveo’s approval in late 2023 marked a turning point for patients who previously had no systemic treatment options beyond surgery or radiation.
A Breakthrough In Desmoid Tumor Management
Desmoid tumors, though non-metastatic, can cause severe pain, functional impairment, and organ compression. Historically, treatment relied on invasive procedures with high recurrence rates.
Ogsiveo, a gamma-secretase inhibitor, disrupts the Notch signaling pathway, a key driver of tumor growth, offering a non-surgical alternative for patients with progressive disease.
Robust Clinical Data From The DeFi Trial
The FDA approval was backed by the Phase 3 DeFi trial (NCT03785964), which demonstrated:
- 72% reduction in risk of progression vs. placebo (HR: 0.29, p < 0.001).
- 41% objective response rate (vs. 8% with placebo), including 7% complete responses.
- Sustained symptom relief, with rapid pain improvement (within weeks) and long-term tumor shrinkage.
Long-term follow-up data presented at the 2024 Connective Tissue Oncology Society meeting further showed deepening responses over time, with 11.4% achieving complete remission by year 4.
Safety And Tolerability: Managing Side Effects
While Ogsiveo is generally well-tolerated, key adverse effects include:
- Diarrhea (84%), managed with dose adjustments or anti-diarrheal agents.
- Ovarian toxicity (75% of female patients), typically reversible upon discontinuation.
- Hypophosphatemia (42%), requiring monitoring and supplementation.
To optimize patient care, SpringWorks has introduced new 100 mg and 150 mg tablets, phasing out the 50 mg formulation by January 2025.
Global Expansion And Future Prospects
With European approval pending and a potential $1 billion peak sales forecast, Ogsiveo is poised for international expansion, starting with Germany in mid-2025.
Meanwhile, SpringWorks is exploring additional indications, including ovarian granulosa cell tumors, with Phase 2 data expected in 2025.
Corporate Developments: Merck KGaA’s Potential Acquisition
SpringWorks’ success has attracted Merck KGaA, currently in advanced acquisition talks.
A deal could accelerate Ogsiveo’s global rollout and bolster SpringWorks’ pipeline, including mirdametinib for neurofibromatosis.
Ogsiveo Patient Support And Advocacy
The Desmoid Tumor Research Foundation (DTRF) continues to advocate for patient access, offering resources through SpringWorks CareConnections™.
Patients are encouraged to consult their healthcare providers regarding the transition to higher-dose tablets.
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