The Food and Drug Administration (FDA) has granted approval for nirogacestat (OGSIVEO, SpringWorks Therapeutics, Inc.) as a treatment for adults diagnosed with progressing desmoid tumors necessitating systemic therapy.
This marks a significant milestone, being the first-ever sanctioned therapy specifically tailored for desmoid tumors.
The phase 3 DeFi trial’s results were the basis for the approval. The results showed a 71% decreased risk of disease progression or death with nirogacestat compared to placebo in desmoid tumor patients (HR, 0.29; 95% CI, 0.15-0.55; P < .001).
The suggested nirogacestat dosage is 150 mg taken orally twice daily, with or without food, until disease progression or unacceptable side effects. Each dose comprises three 50 mg tablets.
SpringWorks anticipates submitting a Marketing Authorisation Application for nirogacestat to the European Medicines Agency (EMA) by the first half of 2024.
Source: FDA approves nirogacestat for desmoid tumors
https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-nirogacestat-desmoid-tumors
