People living with cancer need new advances. Unleash the possibilities.

Patients with cancer are looking to us to develop solutions and create new paths to cancer breakthroughs.

We focus on the science –
and the people

We focus on both solid tumors and hematological malignancies. We know the need is great, which is why we scrutinize promising molecules from every angle to unlock their full potential with the goal of developing transformative therapies that enable patients with cancer to lead better, longer lives.

Desmoid tumors are rare, aggressive soft-tissue tumors characterized by locally invasive growth, significant morbidity, and a high rate of recurrence. OGSIVEO (nirogacestat) is approved in the U.S. for the treatment of adult patients with progressing desmoid tumors who require systemic treatment.

Please see important safety information including link to full Prescribing Information for OGSIVEO at the bottom of this page.

About 30-50% of people with neurofibromatosis type 1 (NF1) develop plexiform neurofibromas (PN), which are peripheral nerve sheath tumors that can cause significant pain, disfigurement, and functional impairment. NF1-PN can transform into malignant peripheral nerve sheath tumors (MPNST), a diagnosis that has a 12-month survival rate of under 50%.

Expanded access & compassionate use

What are the treatment options?

Expanded access, which is also sometimes called “compassionate use,” is a potential pathway for a patient with an immediately life-threatening condition or serious disease to gain access to an investigational medical product for treatment outside of a clinical trial setting, when no comparable or satisfactory alternative therapeutic options are available and prior to its commercial availability.

Does SpringWorks have an expanded access / compassionate use program?

In cases when a patient may not qualify for a clinical trial, there may be no trials available, or a patient has exhausted all available treatment options, SpringWorks may be able to provide access to eligible patients through our Expanded Access Program.

it works

Requests for access to a SpringWorks investigational therapy must be made voluntarily by the patient’s treating physician. Requests cannot be made directly by an individual patient or a patient’s parent/legal guardian or caregiver.

To request expanded access or compassionate use, physicians should send an email to

I was told that a desmoid tumor is ‘benign,’ but the pain it causes has changed my life. It pushes on my ureters and kidneys and has wrapped itself around some of my muscles. The shooting pains sometimes leave me unable to physically move at times, much less take care of my young children.”
Amy, Desmoid Tumor Patient

Patient advocacy

Patient advocacy organizations are instrumental in helping us deepen our connections with and learn from the community. We are committed to building lasting relationships with organizations that share our vision and goals to change the lives of the people we aim to serve.  

The Desmoid Tumor Research Foundation’s (DTRF) mission is to aggressively fund research to accelerate development of therapies for desmoid tumors.
Rein in Sarcoma is a resource for people and families impacted by sarcomas.
SARC (Sarcoma Alliance for Research Through Collaboration), an international collaborative research organization founded by the sarcoma research community itself, is committed to the finding a cure of for all sarcoma patients.
The Children’s Tumor Foundation’s (CTF) mission is to drive research, expand knowledge and advance care for the NF community.
NF Network’s mission is to find treatments and a cure for neurofibromatosis by promoting scientific research. 
National Organization for Rare Disorders’ (NORD) mission is to improve the lives of individuals and families living with rare diseases.

* SpringWorks Therapeutics is providing these links as helpful resources for patients. Inclusion on this list above does not represent an endorsement or a recommendation from SpringWorks for any group or organization. The organizations listed are independent of SpringWorks Therapeutics. 



  • Diarrhea: Diarrhea, sometimes severe, can occur in patients treated with OGSIVEO. Diarrhea occurred in 84% of patients treated with OGSIVEO, and included Grade 3 events in 16% of patients. Median time to first diarrhea event was 9 days (range: 2 to 434 days). Monitor patients and manage using antidiarrheal medications. Modify dose as recommended.
  • Ovarian Toxicity: Female reproductive function and fertility may be impaired in patients treated with OGSIVEO. Impact on fertility may depend on factors like duration of therapy and state of gonadal function at time of treatment. Long-term effects of OGSIVEO on fertility have not been established. Advise patients on the potential risks for ovarian toxicity before initiating treatment. Monitor patients for changes in menstrual cycle regularity or the development of symptoms of estrogen deficiency, including hot flashes, night sweats, and vaginal dryness.
  • Hepatotoxicity: ALT or AST elevations occurred in 30% and 33% of patients, respectively. Grade 3 ALT or AST elevations (>5 × ULN) occurred in 6% and 2.9% of patients. Monitor liver function tests regularly and modify dose as recommended.
  • Non-Melanoma Skin Cancers: New cutaneous squamous cell carcinoma and basal cell carcinoma occurred in 2.9% and 1.4% of patients, respectively. Perform dermatologic evaluations prior to initiation of OGSIVEO and routinely during treatment.
  • Electrolyte Abnormalities: Decreased phosphate (65%) and potassium (22%) occurred in OGSIVEO-treated patients. Phosphate <2 mg/dL occurred in 20% of patients. Grade 3 decreased potassium occurred in 1.4% of patients. Monitor phosphate and potassium levels regularly and supplement as necessary. Modify dose as recommended.
  • Embryo-Fetal Toxicity: OGSIVEO can cause fetal harm when administered to pregnant women. Oral administration of nirogacestat to pregnant rats during the period of organogenesis resulted in embryo-fetal toxicity and death at maternal exposures below human exposure at the recommended dose of 150 mg twice daily. Advise pregnant women of the potential risk to a fetus. Advise females and males of reproductive potential to use effective contraception during treatment with OGSIVEO and for 1 week after the last dose.


  • The most common (≥15%) adverse reactions were diarrhea (84%), ovarian toxicity (75% in the 36 females of reproductive potential), rash (68%), nausea (54%), fatigue (54%), stomatitis (39%), headache (30%), abdominal pain (22%), cough (20%), alopecia (19%), upper respiratory tract infection (17%), and dyspnea (16%).
  • Serious adverse reactions occurred in 20% of patients who received OGSIVEO. Serious adverse reactions occurring in ≥2% of patients were ovarian toxicity (4%).
  • The most common laboratory abnormalities (≥15%) were decreased phosphate, increased urine glucose, increased urine protein, increased AST, increased ALT, and decreased potassium.


  • CYP3A Inhibitors and Inducers: Avoid concomitant use with strong or moderate CYP3A inhibitors (including grapefruit products, Seville oranges, and starfruit) and strong or moderate CYP3A inducers.
  • Gastric Acid Reducing Agents: Avoid concomitant use with proton pump inhibitors and H2 blockers. If concomitant use cannot be avoided, OGSIVEO can be staggered with antacids (e.g., administer OGSIVEO 2 hours before or 2 hours after antacid use).
  • Consult the full Prescribing Information prior to and during treatment for important drug interactions.


  • Because of the potential for serious adverse reactions in breastfed children, advise women not to breastfeed during treatment with OGSIVEO and for 1 week after the last dose.

Please see accompanying full Prescribing Information.