My Life and Limb Saving Drug: Nirogacestat

Nirogacestat is a targeted tumor treatment for desmoid type fibrosarcoma. SpringWorks Therapeutics reportedly plans to submit a New Drug Application (NDA) to the FDA for review in the upcoming near future.

In the below video link, Bernd Kasper, MD, PhD, Mannheim Cancer Center, Germany, discusses findings from the phase 3 DeFi trial, in which nirogacestat demonstrated a 71% reduction in the risk of disease progression or death vs placebo in patients with progressing desmoid tumors. This trial marks the first positive phase 3 results for a gamma secretase inhibitor in any indication.

https://www.hmpgloballearningnetwork.com/site/onc/videos/nirogacestat-demonstrates-potential-standard-care-progressing-desmoid-tumors

After failure of multiple oral tumor treatments and ten months of weekly IV chemotherapy infusions to stop the growth and morbidity from a large locally aggressive infiltrative desmoid tumor, extended compassionate care was approved. It is a true life and limb saver for me.

Compassionate use or expanded access of an experimental drug outside of clinical trials is regulated through the FDA but to qualify you must

• have an illness that is life-threatening
• have no standard treatments available
• not be eligible for a clinical trial

Last year I was totally cane dependent and hopeless with an enlarging rare tumor at risk of limb loss. Now closing out 2022 as a patient on Nirogacestat, the desmoid tumor has greatly decreased pain and bulk as well as my tumor has stabilized. Stay tuned on the blog for more of my lower extremity desmoid tumor and nirogacestat news this upcoming year.