SpringWorks Therapeutics' Nirogacestat Granted Priority Review by FDA for Desmoid Tumor Treatment

SpringWorks Therapeutics, a clinical-stage biopharmaceutical company, has received acceptance from the U.S. Food and Drug Administration (FDA) for its New Drug Application (NDA) for nirogacestat, an investigational gamma-secretase inhibitor. The drug is intended to treat adults with desmoid tumors and has been granted Priority Review with a Prescription Drug User Fee Act (PDUFA) action date of August 27, 2023.

Publish Date: 27-02-2023 Source: SpringWorks Therapeutics

Desmoid tumor (DT) is a rare type of monoclonal, fibroblastic proliferation that can have an unpredictable clinical course. The World Health Organization (WHO) classifies DT as a “clonal fibroblastic proliferation that arises in the deep soft tissues and is characterized by infiltrative growth and a tendency toward local recurrence but an inability to metastasize.” While DT can occur in multiple areas of the same limb or body part, it typically appears in abdominal, intra-abdominal, and extra-abdominal locations. About 5-10% of DT cases occur in individuals with familial adenomatous polyposis (FAP). CTNNB1 mutations and APC mutations are mutually exclusive in DT. Diagnosis depends on a combination of morphologic as well as immunohistochemical findings. DT is most often poorly circumscribed on a surgical specimen, featuring a scar-like, whitish-cut surface and ranging in size. Microscopically, a DT is composed of a cytologically bland, at most moderately cellular, infiltrative spindle cell proliferation set in a variably collagenous stroma. Peripheral infiltration of skeletal muscle is often seen.

Desmoid tumor (DT) incidence ranges from 5 to 7.5 cases per million population in the USA.

However, the current Desmoid Tumor treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (AL102, Nirogacestat, etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the DT treatment. The key companies in the advanced development stage are Ayala Pharmaceuticals, Inc,, SpringWorks Therapeutics, Inc., etc..

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Desmoid Tumor to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2021 to 2032.

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