Pfizer announced today that the European Commission has approved Vyndaqel® (tafamidis) for the treatment of Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) in adult patients with stage 1 symptomatic polyneuropathy. TTR-FAP is a rare, progressive and fatal neurodegenerative disease that affects approximately 8,000 patients worldwide.
"A diagnosis of TTR-FAP, usually made in patients during their mid-life years, impacts both the physical and emotional well-being of patients and caregivers and significantly limits daily activities," said Dr. Teresa Coelho, Hospital Santo Antonio in Porto, Portugal, who participated in the clinical trials of Vyndaqel. "Until now, there were no approved medications to treat this degenerative and fatal disease. Vyndaqel offers new hope to patients who are diagnosed with TTR-FAP."
Mutations of the transthyretin (TTR) gene can result in the production of unstable TTR proteins which can accumulate as amyloid fibrils. Amyloid fibrils can deposit in a variety of organs including the nerves, heart and kidneys, interfering with normal function. Vyndaqel is a novel specific transthyretin stabilizer designed to prevent the formation of these misfolded proteins and the subsequent amyloid deposits that induce neurodegeneration and decline of neurologic function. In the pivotal trial (Fx-005), transthyretin stabilization (as demonstrated by an in vitro assay) was observed in 98 percent of patients on Vyndaqel, and in no patients on placebo, at 18 months.
"Today marks a real breakthrough for patients in the EU living with TTR-FAP," said Yvonne Greenstreet, senior vice president and head of Medicines Development Group for Pfizer's Specialty Care Business Unit. "This community urgently needs an effective therapy, and we are proud to be able to provide the first and only approved medication for patients with this rare and debilitating genetic disease. Pfizer is focused on meeting the needs of patients suffering from rare diseases and this approval is an important step forward in our commitment to providing treatment options for patients."
The approval is based on results from a pivotal clinical trial (Fx-005) and an open-label, 12-month extension study (Fx-006), which evaluated the long-term safety and efficacy of Vyndaqel in patients with TTR-FAP. Across these clinical studies, Vyndaqel showed efficacy in delaying peripheral neurologic impairment. Additional data from these studies showed 51 to 81 percent less deterioration in neurologic function, large fiber function (measure of motor strength) and small fiber function (measure of sensation) compared with patients treated with placebo. Vyndaqel resulted in improved nutritional status (modified body mass index or mBMI); decline in mBMI was shown to correlate with disease progression in the pivotal 18-month study.
The adverse drug reactions reported in the pivotal study of Vyndaqel were diarrhea, upper abdominal pain, urinary tract infection and vaginal infection.
Pfizer is working closely with the relevant national health authorities across the EU to launch the new treatment and anticipates that health care professionals will be able to prescribe the treatment in European markets by early 2012.