FDA Accepts Pfizer's New Drug Application for Tafamidis Meglumine, a Novel, Investigational Treatment for Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP)
NEW YORK, Feb 15, 2012 (BUSINESS WIRE) -- Pfizer Inc. today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for tafamidis meglumine, the company's investigational, novel, oral therapy for the treatment of Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP). TTR-FAP is a rare, progressive and fatal neurodegenerative disease that affects approximately 8,000 patients worldwide.(1,2,3) The European Commission approved tafamidis (the trade name in the European Union is VYNDAQEL(R)) in November 2011.
The FDA has granted the tafamidis NDA a priority-review designation and has provided an anticipated Prescription Drug User Fee Act (PDUFA) action date in June 2012. Priority-review designation is granted to drugs that have the potential to offer significant improvement in treatment or provide a treatment where no adequate therapy exists.
About Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP)
Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) is a rare and fatal neurodegenerative disease primarily caused by a genetic mutation of the transthyretin (TTR) gene.(1,2) In TTR-FAP, TTR destabilization leads to misfolded proteins that form amyloid fibrils in the peripheral and autonomic nerves, as well as other organs including the GI tract, kidneys and heart.(3,4) Patients with TTR-FAP experience significantly diminished quality of life due to symptoms including polyneuropathy characterized by sensory loss, pain and weakness in the lower limbs; as well as severe impairment of the autonomic nervous system commonly manifesting as erectile dysfunction, alternating diarrhea and constipation, unintentional weight loss, orthostatic hypotension, urinary incontinence, urinary retention and delayed gastric emptying.(3,4,5) As the disease progresses, patients often lose the ability to walk, needing wheelchair assistance, and eventually become bedridden and unable to care for themselves.(6,7,8) TTR-FAP typically occurs during active adult years with onset as early as the 30s, followed by disease progression that may reach the terminal stage in approximately 10 years on average.(4,9)
Tafamidis is a novel, selective stabilizer of the TTR protein approved in November 2011 by the European Commission. It is indicated in the European Union for the treatment of TTR amyloidosis in adult patients with stage 1 symptomatic polyneuropathy to delay peripheral neurologic impairment.(10) The drug is currently under review by the FDA in the U.S.
About Pfizer's Specialty Care Business
Pfizer's Specialty Care Business Unit is the world's largest specialty pharmaceuticals business, with a commitment to the eradication, remission, and relief of serious diseases. Pfizer's Specialty Care Business Unit brings together the best scientific minds to challenge the most feared diseases of our time, and we seek solutions to prevent and relieve suffering of patients with serious diseases, regardless of prevalence. Pfizer is an established global leader in rare diseases, offering marketed products treating 17 orphan indications in the U.S. to address the unique needs of small patient populations affected by uncommon and often life- threatening conditions. We are on the front lines of discovering innovative medicines and delivering hope through continued focus on research, development and commercialization of orphan medicines.
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DISCLOSURE NOTICE: The information contained in this release is as of February 15, 2012. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.
This release contains forward-looking information that involves substantial risks and uncertainties about a product in development, tafamidis, including its potential benefits, that is under review by the FDA. Such risks and uncertainties include, among other things, the uncertainties inherent in research and development; whether and when the FDA and regulatory authorities in other jurisdictions in which applications may be filed will approve applications for tafamidis as well as their decisions regarding labeling and other matters that could affect its availability or commercial potential; and competitive developments.
A further description of risks and uncertainties can be found in Pfizer's Annual Report on Form 10-K for the fiscal year ended December 31, 2010 and in its reports on Form 10-Q and Form 8-K.
1. Ando Y, Nakamura M, Araki S. Transthyretin-related familial amyloidotic polyneuropathy. Arch Neurol. 2005;62:1057-1062.
2. Dattilo PB. Familial (ATTR) amyloidosis misdiagnosed as primary (AL) variant: a case report. Cases J. 2009;2:9295-9298.
3. Benson MD, Kincaid JC. The molecular biology and clinical features of amyloid neuropathy. Muscle Nerve. 2007;36:411-423.
4. Hou X, Aguilar M-I, Small DH. Transthyretin and familial amyloidotic polyneuropathy: recent progress in understanding the molecular mechanism of neurodegeneration. FEBS J. 2007;274:1637-1650.
5. Sekijima Y, Yoshida K, Tokuda T, Ikeda S. Familial transthyretin amyloidosis. In: Pagon RA, Bird TD, Dolan CR, Stephens K, eds. GeneReviews [Internet]. Seattle WA: University of Washington, Seattle; 1993-2009. http://www.ncbi.nlm.nih.gov/books/NBK1194/ . Accessed January 31, 2011.
6. Jonsen E, Athlin E, Suhr O. Familial amyloidotic patients' experience of the disease and of liver transplantation. J Adv Nurs. 1998;27:52-58.
7. Coutinho P, da Silva AM, Lima JL, Barbosa AR. Forty years of experience with type 1 amyloid neuropathy: review of 483 cases. In: Glenner GG, e Costa PP, de Freitas AF, eds. Amyloid and Amyloidosis. Amsterdam: Excerpta Medica; 1980:88-98.
8. Suhr OB, Svendsen IH, Andersson R, et al. Hereditary transthyretin amyloidosis from a Scandinavian perspective. J Intern Med. 2003;254:225-235.
9. Plante'-Bordeneuve V, Ferreira A, Lalu T, et al. Diagnostic pitfalls in sporadic transthyretin familial amyloid polyneuropathy (TTR-FAP). Neurology. 2007;69:693--698.
10. Data on file Pfizer Inc, New York, NY.
SOURCE: Pfizer Inc.