FDA Grants Fast Track Designation to Quince for Experimental Therapy for Ataxia-Telangiectasia

Rare Daily Staff

The U.S. Food and Drug Administration granted Quince Therapeutics Fast Track designation for its EryDex System for the treatment of patients with the rare condition ataxia-telangiectasia.

Ataxia-telangiectasia (A-T) is an inherited autosomal recessive neurodegenerative and immunodeficiency disorder caused by mutations in the ATM gene, which is responsible for cell homeostatic and cell division functions including but not limited to double-stranded DNA repair. Typically, A-T is first diagnosed before the age of five as children begin to develop an altered gait and fall with greater frequency. Neurological symptoms worsen and patients with A-T frequently become wheelchair-bound by adolescence. Teenage years for patients with A-T are typically marked by repeated infections, pulmonary impairment, and malignancies. The median lifespan is approximately 25 to 30 years old with mortality due to infections and malignancy. There are currently no approved therapeutic treatments in any global market for A-T.

EryDex is comprised of dexamethasone sodium phosphate (DSP) encapsulated in a patient’s own red blood cells utilizing Quince’s proprietary Autologous Intracellular Drug Encapsulation technology platform. DSP is a corticosteroid well known for its anti-inflammatory properties as well as its dose-limiting toxicity due to adrenal suppression. EryDex is designed to provide the efficacy of corticosteroids and to reduce or eliminate the significant adverse effects that accompany chronic corticosteroid treatment.

“The granting of Fast Track status for EryDex System marks another important milestone in our endeavor to identify a beneficial therapeutic solution for patients with A-T,” said Dirk Thye, CEO of Quince.

The company has initiated a pivotal phase 3 study in the United States, United Kingdom, and European Union.

Photo: Dirk Thye, CEO of Quince