SpringWorks Therapeutics received approval from the U.S. Food and Drug Administration (FDA) on Nov. 27, 2023, for its first drug, Ogsiveo^™ (nirogacestat) tablets. A gamma-secretase inhibitor, it is the first drug that is indicated for treating adults who have desmoid tumors that are worsening and that need to be treated systemically. The recommended dose is 150mg (three tablets) twice daily. Because taking Ogsiveo may affect electrolytes and damage the liver, patients should be tested frequently for blood levels of electrolytes and liver enzymes. The launch is planned within the next five to 10 days. Pricing is not yet available. For complete prescribing information, look here.

At a Glance

• Brand (Generic) Name: Ogsiveo (nirogacestat)
• Manufacturer: SpringWorks Therapeutics
• Date Approved: Nov. 27, 2023
• Indication: to treat adult patients who have progressing desmoid tumors and who require systemic treatment
• Dosage Forms Available: 50mg oral tablets
• Launch Date: By mid-December, 2023
• Estimated Annual Cost: Unavailable
• Desmoid tumors are nonmalignant growths that do not metastasize to other sites. Occurring in fibrous tissues, they are most common in the abdomen, arms, and legs; but they can affect any part of the body. Although they typically grow slowly, desmoid tumors can become painful and disrupt normal movements.
• In the U.S., desmoid tumors are diagnosed for between 900 and 1,500 patients, annually – mostly among older teens and adults. After the tumors are removed, about three-quarters will come back.
• By interfering with the enzyme, gamma-secretase, Ogsiveo blocks the activity of Notch, a protein that promotes desmoid tumor growth.
• A key phase III clinical trial included 142 patients. The risk of tumor advancement or death decreased by 71% for those taking Ogsiveo versus participants taking a placebo. The actively-treated patients also reported less pain and better ability to perform activities. About two-thirds of the patients had been treated for desmoid tumors, previously.
• In the clinical trials, 15% or more of patients taking Ogsiveo experienced altered electrolytes and/or elevated liver enzymes. Other common side effects include cough, diarrhea, fatigue, hair loss and upper respiratory infections.
• Present therapy for desmoid tumors includes surgery, radiation, and off-label treatment with chemotherapy (chemo) or tyrosine kinase-inhibiting drugs.
• Currently, no other drugs are FDA-approved for the treatment of desmoid tumors.
• A breakthrough therapy that was approved under the FDA’s Fast-Track, Priority Review and Real-Time Oncology Review processes, Ogsiveo also has Orphan Drug status for treating desmoid tumors.