The European Commission has approved Pfizer's Vyndaqel for the treatment of a rare genetic disease.
Vyndaqel (tafamidis) has been given the green light for transthyretin familial amyloid polyneuropathy (TTR-FAP), a progressive and fatal neurodegenerative disease that affects some 8,000 patients worldwide. The approval is based on studies where the novel specific transthyretin stabiliser showed efficacy in delaying peripheral neurologic impairment, plus 51%-81% less deterioration in neurologic function, plus large and small fibre function compared with patients treated with placebo.
Vyndaqel resulted in "improved nutritional status", while the adverse drug reactions were diarrhoea, upper abdominal pain, urinary tract infection and vaginal infection. Pfizer said it is working closely with the relevant national health authorities across the EU to launch the new treatment by early 2012.
The company quoted Teresa Coelho of the Hospital Santo Antonio in Porto, Portugal, who noted that 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”. Until now, there were no approved drugs, liver transplant being the only treatment option, so Vyndaqel "offers new hope", she added.
The news came hours after the US Food and Drug Administration’s Vaccines and Related Biological Products Advisory Committee voted 14 to one that Pfizer's Prevnar 13 vaccine is safe for the prevention of pneumococcal disease caused by the serotypes contained in the vaccine in adults 50 years and older.
The recommendation for expanded approval (Prevnar 13 is currently available for children aged six weeks to five years) came after FDA staffers said that the vaccine was at least as effective as Merck & Co's Pneumovax at helping prevent pneumococcal disease in adults.