Building a System to Ensure Awareness of Nirogacestat, a Treatment for Desmoid Tumors

Nirogacestat (Ogsiveo; Springworks Therapeutics) is a therapy used to treat desmoid tumors, rare but aggressive growths that arise in connective tissues such as fascia, ligaments, and tendons.The FDA first approved nirogacestat for use in adult patients in 2023.¹ The product has a broad indication and can be used in adult patients with progressing desmoid tumors that require systemic treatment.Although nirogacestat is used in a rare disease, it is widely available in oncology practices that dispense oral therapies as well as through specialty pharmacies for practices that do not have access to oral therapies.

Utah Cancer Specialists (UCS) has a long history of treating sarcomas in the community and employs a medical oncologist with a primary focus on sarcomas. Sarcomas represent about 1% of all cancer diagnoses in the US,² with desmoid tumors representing about 3% of sarcomas and about 0.03% of neoplasms.³ Because of the rare nature of these diseases, oncologists with sarcoma expertise are rare and usually work within large academic medical centers. Within increasing treatment options, however, there is a growing opportunity for patients to be treated in community settings, as they are at UCS.

Nirogacestat plays an important role in the treatment of desmoid tumors by providing patients with an oral therapy that is highly accessible and well tolerated. The therapy works by inhibiting γ-secretase, which blocks activation of the Notch signaling pathway that drives growth in desmoid tumors. In clinical trials, nirogacestat was compared to placebo and significantly increased the progression-free survival (PFS) of patients. In the phase 3 DeFi trial (NCT03785964), nirogacestat reduced the risk of disease progression or death by approximately 71% compared with placebo and significantly improved PFS.^1,4-5 The most common adverse events associated with treatment are diarrhea, ovarian toxicity, nausea, fatigue, stomatitis, headache, abdominal pain, cough, alopecia, upper respiratory tract infection and dyspnea.^4-5 Patients at Utah Cancer Specialists who have taken nirogacestat have generally been able to tolerate it and have benefited from its efficacy.

Although nirogacestat has been used for several patients at UCS, the rarity of desmoid tumors can make it difficult to maintain clinician awareness of the therapy from both operational and clinical perspectives. To combat this, the practice has engaged the manufacturer to help provide education and answer questions that arise. The clinical care team includes a medical oncologist, an advanced practice provider, nurses, pharmacists, and pharmacy technicians who work together to streamline therapy decisions and remove barriers to patient care. To help the team maintain awareness of rare disease products such as nirogacestat, UCS employs several strategies, including the electronic medical record (EMR) system, the formulary, ongoing manufacturer engagement, and medication management meetings.

The most important of these strategies is to maintain a formulary that stays current within the practice EMR. UCS categorizes its formulary by disease state in the EMR, with designated folders for each disease type. Prescribers then use these folders to select the most appropriate therapy that is currently approved on the formulary.When a patient is diagnosed with a desmoid tumor and systemic therapy is deemed appropriate, the physician can quickly access relevant products and determine whether a product is designated as preferred for the class.

Manufacturers also play an important role in maintaining product awareness for rare diseases. At UCS, manufacturers engage with clinicians and leaders through clinic visits, business meetings, and offsite programs.

Finally, UCS conducts ongoing medication management meetings to raise awareness of clinical developments, formulary updates, and industry news.Meetings are held with the formulary team, the physician group and individual manufacturers to stay informed about drug information and assimilate relevant information into the practice.These efforts are led by the pharmacy team and serve as the hub for industry and clinicians to interface.

Through these efforts, UCS has successfully maintained awareness of nirogacestat, and many patients have benefited from this therapy since its launch in 2023.The challenge of staying abreast of rare disease innovation should not be a barrier for patients receiving the best possible care for their cancer, and extra effort is often warranted. As targeted therapies for rare diseases continue to emerge, community oncology practices must build deliberate systems to maintain awareness of these treatments so that eligible patients can benefit from advances that might otherwise go unnoticed.

Author Information

Kyle Kitchen, PharmD, MBA, is the chief clinical officer with Utah Cancer Specialists. He has more than 20 years of experience in pharmacy and clinical management roles at various organizations, including Intermountain Health and Primary Children’s Hospital in Salt Lake City.

References

1. FDA approves nirogacestat for desmoid tumors. FDA. November 28, 2023. Accessed May 15, 2026. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-nirogacestat-desmoid-tumors
2. Popovich JR, Kashyap S, Gasalberti DP, et al. Sarcoma. In: StatPearls. StatPearls Publishing; 2023. https://www.ncbi.nlm.nih.gov/books/NBK519533/
3. Jenayah AA, Bettaieb H, Saoudi S, et al. Desmoid tumors: clinical features and treatment options: a case report and a review of literature. Pan Afr Med J. 2015;21:93. doi:10.11604/pamj.2015.21.93.7037
4. Gounder M, Ratan R, Alcindor T, et al. Nirogacestat, a γ-secretase inhibitor for desmoid tumors. N Engl J Med. 2023;388(10):898-912. doi:10.1056/NEJMoa2210140.
5. Kasper B, Whiting J, Lawson D, et al. A study to learn if nirogacestat works and is safe for adult participants with desmoid tumors: a plain language summary of the DeFi study. Future Oncol. 2025;21(8):889–901. doi: 10.1080/14796694.2025.2462519