Abbisko Therapeutic announces that its next-generation FGFR4 mutant inhibitor ABSK012 has been granted orphan drug designation in the US for the treatment of Soft Tissue Sarcoma

3 April 2023, Shanghai – Abbisko Therapeutics Co., Ltd. (“Abbisko Therapeutics” hereafter) today announced that its next-generation FGFR4 mutant inhibitor ABSK012 has been granted the orphan drug designation (ODD) by the US Food and Drug Administration (FDA) for the treatment of Soft Tissue Sarcoma (STS).

The FDA grants ODD to investigational drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. orphan drug designation provides drug developers with various benefits designed to support the development of novel drugs and biologics, including market exclusivity for seven years upon FDA approval, tax credits for qualified clinical trials, and exemption from FDA application fees.

ABSK012 is an orally bioavailable, highly selective, next-generation FGFR4 inhibitor with strong potency against both wild-type and mutant FGFR4. In pre-clinical studies, ABSK012 has demonstrated strong activities in vitro and in cells against both wild-type FGFR4 and various FGFR4 mutants that are resistant to current FGFR4 inhibitors in clinical development, and excellent in vivo efficacy in FGF19-driven and FGFR4-mutant models.

Soft Tissue Sarcoma

Soft tissue sarcoma (STS) accounts for ~7% of cancers in children and 1% of cancers in adults. The rate of new cases was 2.91 and 3.4 per 100,000 people per year in China and the US, respectively. There are more than 50 subtypes of STS and the management is increasingly subtype-dependent. The average overall 5-year survival rate for STS is approximately 65%. Rhabdomyosarcoma (RMS) (one type of STS) impacts even fewer patients. All studies highlight a poorer outcome in adults than in children, with 5-year overall survival in the range of 20–50%. Therefore, there is an urgent need for new therapies to improve overall survival rates. FGFR4 overexpression and activating mutation have been described in RMS.