Nirogacestat Application Scope

For the treatment of adults with progressive desmoid tumors requiring systemic therapy.

First approved targeted therapy specifically for sclerofibroblastoma.

Nirogacestat Characteristics

Ingredients: Nirogacestat (PF-03084014), a selective gamma-secretase inhibitor.
Properties:

Oral small molecule drug that inhibits tumor growth by inhibiting the Notch signaling. pathway.

Specification: 150 mg per tablet, recommended dose is 150 mg (3 tablets) twice daily.
Packaging Specification:

No specific package quantity is mentioned, but 6 tablets (150 mg × 2) should be taken daily.

Storage: Store at room temperature away from children.
Expiry Date: Not explicitly mentioned, please refer to the package labeling of the drug.
Executive Standard: Comply with FDA cGMP.
Approval Number:

FDA approval date: November 27, 2023, NDA number not disclosed.

Recommended Dose: 150 mg orally twice daily (either on an empty stomach or with a meal) until disease progression or intolerable toxicity.

Dosage Adjustment: Diarrhea (≥3 days duration): suspend dosing and reduce to 100 mg twice daily upon recovery.

Hepatotoxicity (elevated ALT/AST): suspend at ≥3x ULN and reduce to 100 mg twice daily upon recovery; permanently discontinue at ≥5x ULN.

Hypophosphatemia/hypokalemia: suspend and replenish electrolytes, reduce dosage after recovery.

Common (≥20%): diarrhea (84%), ovarian toxicity (75% of female patients), nausea (54%), fatigue (51%), hypophosphatemia (42%), rash (32%).

Others: headache, abdominal pain, alopecia, upper respiratory tract infection, etc.

Hypersensitivity to nirogacestat or its components.

Prohibited in pregnancy (may cause fetal damage).

Ovarian toxicity: ovarian dysfunction (e.g., amenorrhea) occurs in 75% of female patients and is reversible in 74%.

Hepatotoxicity: regular monitoring of liver function is required.

Contraceptive requirements: patients of childbearing age need to use effective contraception during treatment and for 1 week after stopping the drug.

CYP3A4 inhibitors/inducers: avoid combination (e.g., ketoconazole, rifampin), may affect nirogacestat blood levels.

Gastric acid modulators (PPI/H2 receptor antagonists): need to be taken at 2-hour intervals (nirogacestat is less soluble at pH ≥ 6).

Note:

If there is a new packaging for the drug, the new packaging shall prevail. The above information is sourced from DengYue Medicine.
It is only for internal discussion among medical staff and does not serve as a basis for medication. For specific medication guidelines, please consult the attending physician.