U.S. FDA APPROVES VYNDAQEL® AND VYNDAMAX™ FOR USE IN PATIENTS WITH TRANSTHYRETIN AMYLOID CARDIOMYOPATHY, A RARE AND FATAL DISEASE

U.S. FDA APPROVES VYNDAQEL® AND VYNDAMAX™ FOR USE IN PATIENTS WITH TRANSTHYRETIN AMYLOID CARDIOMYOPATHY, A RARE AND FATAL DISEASE

— FIRST AND ONLY MEDICINES APPROVED FOR PATIENTS WITH EITHER WILD-TYPE OR HEREDITARY TRANSTHYRETIN AMYLOID CARDIOMYOPATHY —

Monday, May 6, 2019 – 6:45am EDT

Pfizer Inc. (NYSE:PFE) announced today that the U.S. Food and Drug Administration (FDA) has approved both VYNDAQEL® (tafamidis meglumine) and VYNDAMAX (tafamidis) for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. VYNDAQEL and VYNDAMAX are two oral formulations of the first-in-class transthyretin stabilizer tafamidis, and the first and only medicines approved by the FDA to treat ATTR-CM.

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HealthWell Foundation Opens New Amyloidosis Fund to Assist Patients with Treatment Costs

HealthWell Foundation Opens New Amyloidosis Fund to Assist Patients with Treatment Costs

GERMANTOWN, Md. – April 23, 2019 – The HealthWell Foundation, an independent non-profit that provides a financial lifeline for inadequately insured Americans, has launched a new fund to provide copayment and premium assistance to patients living with amyloidosis.  Through the fund, HealthWell will provide up to $8,000 in financial assistance for a 12-month grant to eligible patients who have annual household incomes up to 500 percent of the federal poverty level.

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Eidos Therapeutics Initiates ATTRibute-CM, a Phase 3 Study of AG10 in ATTR-CM with Registrational 12-month Endpoint

Eidos Therapeutics Initiates ATTRibute-CM, a Phase 3 Study of AG10 in ATTR-CM with Registrational 12-month Endpoint

February 27, 2019 07:00 ET – Source: Eidos Therapeutics, Inc.

San Francisco, Feb. 27, 2019 (GLOBE NEWSWIRE) — Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), today announced the initiation and design of its pivotal global Phase 3 trial (ATTRibute-CM) of AG10 in patients with transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM). The design of the ATTRibute-CM study, which incorporates feedback from FDA, includes two potentially registrational endpoints. In Part A, benefit in change from baseline in 6-minute walk distance (6MWD) will be evaluated at 12 months, potentially accelerating the time to registration. In Part B, reduction in all-cause mortality and frequency of cardiovascular-related hospitalizations will be evaluated at 30 months.

 

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Missouri

Missouri

Tuesdays 1:00 pm – 3:00 pm

  • March 5, 2019
  • June 4, 2019
  • September 3, 2019
  • December 3, 2019

Center for Advanced Medicine

4921 Parkview Place, 3rd Floor

Farrell Conference Room #1

St. Louis, MO  63110

Contact:  Kimberly Gal  314-273-2255

Parking Information:  https://www.barnesjewish.org/Patients-Visitors/Locations-Directions/Center-for-Advanced-Medicine



The Amyloidosis Foundation Spreads Awareness Through ‘Amyloidosis Awareness Month’ in March 2019

The Amyloidosis Foundation Spreads Awareness Through ‘Amyloidosis Awareness Month’ in March 2019

Most people are unfamiliar with amyloidosis, often diagnosed late since they are unaware of what this rare disease is or what the symptoms are. The Amyloidosis Foundation is working to change that, declaring March as Amyloidosis Awareness Month.

Charlotte Haffner, a member of the AF Board of Directors and an amyloidosis patient herself, says “Raising awareness for amyloidosis is one of the most important things you can do as a patient or if your life has been touched by this disease. You can help more people understand amyloidosis, and help raise funds for research, education, and one day a cure. Today there is more hope than ever. Please use your power as a patient or care-giver and spread the word far and wide. You never know whose life you might help save.  It could even be your own.”

We began in 2018, by passing state resolutions to recognize Amyloidosis Awareness Month in Tennessee, Michigan and Louisiana. Indiana passed a resolution in January 2019. We have advocates working on similar legislation in the following states for 2019: Arkansas, California, Florida, Georgia, Kentucky, Nebraska, New York, Pennsylvania, Utah, Virginia and Wisconsin.

The AF is encouraging patients, caregivers, physicians and donors to participate this March by using the hashtag #amyloidosiswarrior on all social media platforms. By spreading amyloidosis awareness online, this will increase the chances that others will be diagnosed and treated earlier, which is an important part to creating positive outcomes for all patients.

We are thankful that in the Summer of 2018, two therapies were approved by the FDA for ATTR amyloidosis, which is the hereditary type of this disease. For more information about Amyloidosis Awareness Month, or if you’d like to become an advocate, email kathi@amyloidosis.org.

 


Untangling Amyloidosis Symposium

Untangling Amyloidosis Symposium

Here are the individual speakers from the “Untangling Amyloidosis” Symposium at the 60th ASH (American Society of Hematology) Conference in November, 2018. The Amyloidosis Foundation was proud to support Junior Researcher Travel Grants for this event. Click below to listen to each topic.

Welcome Message

 

Initial Therapy of AL Amyloidosis in Transplant-Eligible and -Ineligible Patients
Giovanni Palladini, MD, PhD
Acting Director, Amyloidosis Research & Treatment Center
University of Pavia Pavia, Italy

Watch Here

 

 

Incorporating Monoclonal Antibodies into Anti-Plasma Cell Regimens in AL Amyloidosis
Suzanne Lentzsch, MD, PhD
Director, Multiple Myeloma and Amyloidosis Service
Columbia University Medical CenterNew York City, USA

Watch Here

 

 

The Evidence for Use of Doxycycline as Adjunctive Therapy for Cardiac Amyloidosis
Prof. Ashutosh Wechalekar
Professor of Medicine and Haematology
University
College London

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Protein- and Fibril-Directed Therapies in AL Amyloidosis
Jeffrey Zonder, MD
Leader, Myeloma and Amyloidosis Program
Karmanos Cancer Institute/Wayne State University
Detroit, USA

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Gene Silencing Therapies in ATTR Amyloidosis
Sascha Tuchman, MD MHS
Director, Multiple Myeloma and Amyloidosis Program
University of North Carolina Lineberger Comprehensive Cancer Center
Chapel Hill, NC

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Developing New Therapies for Amyloidosis in a Complex Research Landscape
John Berk, MD
Assistant Director, Amyloidosis Center
Boston University School of Medicine
Boston, USA

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