Pfizer said its drug tafamidis, an investigational treatment for transthyretin cardiomyopathy (TTR-CM), a rare, fata disease linked to progressive heart failure, has been granted U.S. Food and Drug Administration (FDA) fast track designation.
“The fast track designation for tafamidis is an important milestone, as there are no currently approved treatments for TTR-CM in the U.S.,” Brenda Cooperstone, senior vice president and chief development officer, Rare Disease, at Pfizer Global Product Development, said in a press release. “We look forward to working closely with the FDA to evaluate this medicine as a potential new treatment option for patients.”
Pfizer said in the release its Transthyretin Amyloid Cardiomyopathy Tafamidis Study, the first Phase 3 double-blind placebo-controlled clinical study initiated in TTR-CM, features patients with variant transthyretin familial amyloid cardiomyopathy (TTR-FAC), the hereditary form of the disease, as well as patients with wild-type TTR-CM, the one-hereditary form that can show up as people get older.
“The hereditary form of this disease not only impacts the lives of people with the disease, but also may affect multiple generations; patients often have been caregivers for a parent and (are) concerned for their children who also may inherit the disease,” Isabelle Lousada, CEO and president of Amyloidosis Research Consortium, said in the release. “Programs like the fast track designation offer real hope that the development of critically needed treatment options for people living with rare diseases will be expedited.”