Nirogacestat: What is the new treatment for desmoid tumors?

Last updated: 23 April 2024

Desmoid tumors are rare, accounting for only 0.03% of all tumors ⁷. Luckily, they are also rarely fatal, with an 87% survival rate at 15 years after diagnosis ⁸. However, a desmoid tumor can be associated with pain and functional impairment, which can decrease the quality of life for a patient population that is often between the ages of 10 and 40.

If you've been diagnosed with a desmoid tumor, you may be aware that there are limited treatment options outside of surgery. Nirogacestat, the newest treatment for desmoid tumor, is set to change this and provide more choice for doctors and patients like you.

Here's all you need to know about this latest desmoid tumor treatment.

Desmoid tumors: current treatment options and limitations

Desmoid tumors, also known as aggressive fibromatosis, are noncancerous formations usually found in the connective tissue of the stomach, arms and legs.

Unlike cancers, desmoid tumors don't spread to other areas of the body, but they can be very aggressive and grow locally.

Many patients don't require treatment unless the tumor grows or they experience additional symptoms. If treatment is required, it usually involves surgery, radiation therapy, or chemotherapy.

What is the first line treatment for desmoid tumors

The first-line treatment for desmoid tumors is usually surgical removal. Since desmoid tumors don't spread across the body, a surgery can be an efficient treatment.

However, surgery is not always an option. For example, the location of the tumor can make surgery risky. If the tumor has incorporated nearby structures, this can also make it difficult to remove completely.

If surgery cannot be performed, radiation therapy can be used as a first-line therapy for desmoid tumors. In cases when the tumor is growing quickly, your doctor may recommend to start with chemotherapy. This typically involves methotrexate and vinblastine, or vinorelbine combinations ¹.

Up until 2023, these were the only available first-line treatment options for desmoid tumors.

The FDA approval of Ogsiveo (nirogacestat) as a medicine specifically designed for this condition, has been a major step in the direction of a broader treatment landscape.

Nirogacestat: A new drug for desmoid tumor

In November 2023, the FDA gave its green light to the first targeted therapy for desmoid tumors. The medicine marketed as Ogsiveo (nirogacestat) is an oral gamma-secretase inhibitor.

Here's what you should know about it.

Is nirogacestat first-in-class?

Nirogacestat is indeed considered to be a first-in-class targeted therapy for desmoid tumors, according to Mrinal Gounder, MD, a sarcoma oncologist at Memorial Sloan Kettering Cancer Center ¹. With oncologists slowly trying to move away from surgery when treating desmoid tumors, it's of growing importance that they have more treatment alternatives available.

How does nirogacestat work?

Nirogacestat is a gamma-secretase inhibitor. It works by blocking specific enzymes that signal the tumors to grow. By blocking these enzymes, nirogacestat interferes with the pathways that regulate cell growth and division. Due to this mechanism of action, nirogacestat may help to slow down or stop the growth of desmoid tumor cells.

Nirogacestat is not a cure for desmoid tumors. However, it has shown potential in clinical trials to reduce the size of these tumors and improve symptoms in some patients.

What are the results of the nirogacestat clinical trial?

Ogsiveo (nirogacestat) received its FDA approval on the basis of a phase 3 clinical trial involving a total of 70 patients. In the trial, patients received either nirogacestat or placebo. Some of the key reported results were:

41% of patients treated with Ogsiveo had their tumors shrink or disappear. In the placebo group, only 8% reported this result;
7% of patients treated with Ogsiveo had a complete response (i.e. their tumors disappeared). No patients in the placebo group reported such a result;
The median time to response was 5.6 months;
The likelihood of having no disease progression 2 years after start of treatment was 76% with Ogsiveo and 44% with placebo ³.

It is important to note that while nirogacestat showed promising results in tumor shrinkage and growth prevention, it also caused certain side effects. Common side effects reported during the trial included nausea, diarrhea, fatigue, and increased blood creatinine levels. These side effects were generally manageable and did not significantly impact the patients' quality of life ³.

Among female trial participants, 75% of those of childbearing age reported adverse effects related to ovarian dysfunction. This is not an uncommon effect of gamma secretase inhibitors. The symptoms were generally resolved after stopping the treatment ⁴.

Is nirogacestat approved?

Yes, in the USA, where the FDA approved the medicine in November 2023 ⁵. Nirogacestat's EMA approval is still pending, as the medicine's application is under review.

As of April 2024, Ogsiveo (nirogacestat) is not approved anywhere else in the world.

Is nirogacestat available outside the USA?

Currently, not yet. Outside of the USA, nirogacestat's availability is limited to patients enrolled in clinical trials.

This being said, individual patients in non-US countries can also access the medicine outside a clinical trial, under specific conditions. According to compassionate use regulations, patients are allowed to import a medicine from abroad if it's not yet available in their country. This only applies to medicines for personal use, and which address life-threatening or debilitating conditions.

If your treating doctor is of the opinion that Ogsiveo could benefit you, you can get the medicine immediately. The upside is that you don't have to wait for local approval or availability. The downside is that you can't benefit from reimbursement and you will have to cover the medicine costs yourself.

How much does nirogacestat cost?

Nirogacestat is currently only approved in the USA. As a result, it's only available at its American price. As a general idea, it's estimated to cost around USD 30,000 for a 30-day supply ⁶.

It is important to note that these figures are subject to change, and the final cost will depend on a variety of factors such as your location, the supplier, and potential insurance coverage.

If you're interested in nirogacestat and have a prescription from your treating doctor, get in touch with our team of Medicine Access Experts. We can help you buy nirogacestat before it's approved or available in your country.