FDA Grants Breakthrough Therapy Designation for Tafamidis for the Treatment of Patients with Transthyretin Cardiomyopathy

Wednesday, May 23, 2018 8:00 am EDT

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PFE
US7170811035
"We look forward to working with the FDA through this expedited process to fulfill an unmet patient need."

NEW YORK--(BUSINESS WIRE)--Pfizer Inc. (NYSE:PFE) announced today that tafamidis received Breakthrough Therapy designation from the US Food and Drug Administration (FDA) for the treatment of patients with transthyretin cardiomyopathy, a rare, fatal, and underdiagnosed condition associated with progressive heart failure.1,2

This decision is supported by topline results from the tafamidis Phase 3 Transthyretin Cardiomyopathy (ATTR-ACT) study, in which tafamidis demonstrated a statistically significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalizations.1 Currently, there are no approved pharmacological treatments specifically indicated for this disease, and the average life expectancy for people with transthyretin cardiomyopathy is 3 to 5 years from diagnosis.3.4

“This designation is an important step forward in the path to bringing a potential new treatment option to those with transthyretin cardiomyopathy, a rare, fatal disease,” said Brenda Cooperstone MD, Senior Vice President and Chief Development Officer, Rare Disease, Pfizer Global Product Development. “We look forward to working with the FDA through this expedited process to fulfill an unmet patient need.”

Breakthrough Therapy designation was initiated as part of the Food and Drug Administration Safety and Innovation Act (FDASIA) signed in 2012. As defined by the FDA, a breakthrough therapy is a drug intended to be used alone or in combination with one or more other drugs to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. If a drug is designated as a breakthrough therapy, the FDA may expedite the development and review of such drug.5

About Tafamidis

Tafamidis is an investigational treatment for transthyretin cardiomyopathy and is not approved for this indication. In 2012, tafamidis was granted orphan drug designation for transthyretin cardiomyopathy in both the EU and US. In May 2017, the US FDA granted Fast Track designation to tafamidis for transthyretin cardiomyopathy; additionally, in March 2018, the Ministry of Labor Health and Welfare in Japan granted SAKIGAKE designation to tafamidis for this indication.

About Transthyretin Cardiomyopathy

Transthyretin cardiomyopathy is a rare, fatal, and underdiagnosed condition associated with progressive heart failure.1,2 The prevalence of transthyretin cardiomyopathy is presently unknown; however, it is estimated that less than 1% of people with the disease are diagnosed. Currently, there are no approved pharmacological treatments specifically indicated for this disease, and the average life expectancy is 3 to 5 years from diagnosis.3,4

Pfizer Rare Disease

Rare disease includes some of the most serious of all illnesses and impacts millions of patients worldwide,6 representing an opportunity to apply our knowledge and expertise to help make a significant impact on addressing unmet medical needs. The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including hematology, neuromuscular, and inherited metabolic disorders.1

Pfizer Rare Disease combines pioneering science and deep understanding of how diseases work with insights from innovative strategic collaborations with academic researchers, patients, and other companies to deliver transformative treatments and solutions. We innovate every day leveraging our global footprint to accelerate the development and delivery of groundbreaking medicines and the hope of cures.

Click here to learn more about our Rare Disease portfolio and how we empower patients, engage communities in our clinical development programs, and support programs that heighten disease awareness.

Working together for a healthier world ®

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products. Our global portfolio includes medicines and vaccines as well as many of the world's best-known consumer health care 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 one of the world's premier innovative biopharmaceutical companies, we 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, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.pfizer.com. In addition, to learn more, please visit us on www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

DISCLOSURE NOTICE: The information contained in this release is as of May 23, 2018. 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 about a potential indication for tafamidis for the treatment of transthyretin cardiomyopathy (the “Potential Indication”) and Pfizer’s rare disease portfolio, including their potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical trial commencement and completion dates and regulatory submission dates, as well as the possibility of unfavorable clinical trial results, including unfavorable new clinical data and additional analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations, and, even when we view data as sufficient to support the safety and/or effectiveness of a product candidate, regulatory authorities may not share our views and may require additional data or may deny approval altogether; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when any new or supplemental drug applications may be filed in any jurisdictions for tafamidis for the Potential Indication; whether and when regulatory authorities in any such jurisdictions where applications for tafamidis may be pending (including the application pending with the FDA for the potential treatment of transthyretin familial amyloid polyneuropathy, for which the company received a complete response letter in 2012) or filed may approve any such applications, which will depend on the assessment by such regulatory authority of the benefit-risk profile suggested by the totality of the efficacy and safety information submitted, and, if approved, whether tafamidis will be commercially successful; decisions by regulatory authorities regarding labeling and other matters that could affect the availability or commercial potential of tafamidis, including for the Potential Indication; 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, 2017 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at  www.sec.gov  and  www.pfizer.com .

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1   Data on file. Pfizer Inc. New York, NY.
2

THAOS - Transthyretin Amyloidosis Outcomes Survey. Disease Background - transthyretin amyloidosis. https://www.thaos.net/Physicians/DiseaseBackground.cfm. Accessed May 14, 2018.

3

Connors LH, Doros G, Sam F, Badiee A, Seldin DC, Skinner M. Clinical features and survival in senile systemic amyloidosis: comparison to familial transthyretin cardiomyopathy. Amyloid. 2011;18(sup1):157-159. doi:10.3109/13506129.2011.574354059

4 Ando Y, Coelho T, Berk JL, et al. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet Journal of Rare Diseases. 2013;8(1):31. doi:10.1186/1750-1172-8-31.
5

Center for Drug Evaluation and Research. Food and Drug Administration Safety and Innovation Act (FDASIA) - Fact Sheet: Breakthrough Therapies. U S Food and Drug Administration Home Page. https://www.fda.gov/RegulatoryInformation/LawsEnforcedbyFDA/SignificantAmendmentstotheFDCAct/FDASIA/ucm329491.htm. Published March 28, 2018. Accessed May 14, 2018.

6

Pfizer Inc. Rare disease. http://www.pfizer.com/health-and-wellness/health-topics/rare-diseases/areas-of-focus. Accessed May 14, 2018.

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