NEW YORK–(BUSINESS WIRE)–Pfizer Inc. (NYSE:PFE) announced today that the United States Food and
Drug Administration’s (FDA) Peripheral and Central Nervous System Drugs
Advisory Committee voted on Pfizer’s clinical data package for tafamidis
meglumine. Tafamidis is a novel, investigational, oral therapy for the
treatment of Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) in
adult patients with symptomatic polyneuropathy to delay neurologic
impairment. The Advisory Committee did not find substantial evidence of
efficacy on a clinical endpoint. The Committee then voted 13-4 that the
data provide substantial evidence of efficacy for a surrogate endpoint
that is reasonably likely to predict a clinical benefit. This
recommendation will be taken into consideration by the FDA when making
its decision on Pfizer’s New Drug Application (NDA) for tafamidis as a
treatment for TTR-FAP, a rare and fatal neurodegenerative disease.
“TTR-FAP is an irreversible and devastating disease with no FDA-approved
treatment in the U.S.”
“TTR-FAP is an irreversible and devastating disease with no FDA-approved
treatment in the U.S.,” said Dr. Yvonne Greenstreet, senior vice
president and head of Medicines Development Group for Pfizer’s Specialty
Care Business Unit. “The panel’s assessment represents a positive step
forward in our goal to provide this much-needed medicine to patients
suffering from this rare and fatal disease. Pfizer will continue to work
with the FDA as the Agency finalizes its review of our NDA for
tafamidis.”
TTR-FAP is a rare, progressive and fatal neurodegenerative disease that
affects approximately 8,000 patients worldwide.1,2,3 Because
it is a hereditarydisease, family members may also be at
risk for developing the disease.4 In the U.S., a non-endemic
region, the incidence is estimated to be about 1 in 100,000, impacting
approximately 3,000 people.5,6 There is currently no
FDA-approved treatment in the U.S. designed specifically to treat
TTR-FAP. If approved by the FDA, tafamidis would be the first and only
medication in the U.S. indicated to treat patients with this
debilitating genetic disease.
The FDA has granted the tafamidis NDA both an orphan drug and a priority
review designation. The Orphan Drug Designation program provides orphan
status to drugs and biologics that are defined as those intended for the
safe and effective treatment, diagnosis or prevention of rare diseases
and disorders that affect fewer than 200,000 people in the U.S. 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.7,8 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.4,7,8 As the disease progresses,
patients often lose the ability to walk, needing wheelchair assistance,
and eventually become bedridden and unable to care for themselves.9
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.8,10
About Tafamidis
Tafamidis is a novel, selective stabilizer of the TTR protein approved
in November 2011 by the European Commission (the trade name in the
European Union is VYNDAQEL®). 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.
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 18 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.
Pfizer Inc: Working together for a healthier world™
At Pfizer, we apply science and our global resources to improve health
and well-being at every stage of life. We strive to set the standard for
quality, safety and value in the discovery, development and
manufacturing of medicines for people and animals. Our diversified
global health care portfolio includes human and animal biologic and
small molecule medicines and vaccines, as well as nutritional products
and many of the world’s best-known consumer products. Every day, Pfizer
colleagues work across developed and emerging markets to advance
wellness, prevention, treatments and cures that challenge the most
feared diseases of our time. Consistent with our responsibility as the
world’s leading biopharmaceutical company, we also collaborate with
health care providers, governments and local communities to support and
expand access to reliable, affordable health care around the world. For
more than 150 years, Pfizer has worked to make a difference for all who
rely on us. To learn more about our commitments, please visit us at www.pfizer.com.
DISCLOSURE NOTICE: The information contained in this release
is as of May 24, 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, 2011 and in its reports on Form 10-Q and Form 8-K.
1. Dattilo PB. Familial (ATTR) amyloidosis misdiagnosed as primary (AL)
variant: a case report. Cases J. 2009;2:9295-9298.
2. Ando Y, Nakamura M, Araki S. Transthyretin-related familial
amyloidotic polyneuropathy. Arch Neurol. 2005;62:1057-1062.
3. Data on file. Pfizer Inc, New York, NY.
4. 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.
5. Benson MD. Amyloidosis. Encyclopedia of Life Sciences. Available at: http://onlinelibrary.wiley.com/doi/10.1038/npg.els.0002146/pdf.
Published April 2001.
6. US Census Bureau. Annual Estimates of the Resident Population by Sex
and Five-Year Age Groups for the United States: April 1, 2000 to July 1,
2009. Suitland, MD: US Census Bureau.
7. Benson MD, Kincaid JC. The molecular biology and clinical features of
amyloid neuropathy. Muscle Nerve. 2007;36:411-423.
8. 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.
9. 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.
10. Plante´-Bordeneuve V, Ferreira A, Lalu T, et al. Diagnostic pitfalls
in sporadic transthyretin familial amyloid polyneuropathy (TTR-FAP).
Neurology. 2007;69:693–698.
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