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.

For more information, click here


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.

For more information, click here


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.

 

For more information, click here



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

Watch Here

 

 

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

Watch Here

 

 

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

Watch Here

 

 

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

Watch Here

more


Amyloidosis Foundation Announces 2019 Research Grant Recipients

Amyloidosis Foundation Announces 2019 Research Grant Recipients

The Amyloidosis Foundation recently awarded two research grants for 2019. The Donald C. Brockman Memorial Research Grant went to Siyang Leng, MD from Columbia University Medical Center in New York City and the David Seldin, MD, PhD Memorial Research Grant recipent is Amandeep Godara, MD from Tufts Medical Center in Boston, MA.

Since 2005, the AF has funded over $1.8 million to promising clinical and scientific amyloidosis investigators from around the world. We applaud their efforts and look forward to the success this work will bring to patients.

Click here for photos and to read more about their proposals.

Congratulations!


FDA Approves Two Treatments for hATTR Amyloidosis in 2018

FDA Approves Two Treatments for hATTR Amyloidosis in 2018

 by Frederick L. Ruberg, MD – Boston University, BUMC, Amyloidosis Center

What a momentous Summer of 2018 it has been for transthyretin (ATTR) amyloidosis. First, in July 2018, in the same issue of the New England Journal of Medicine, two different phase 3 clinical trials were reported demonstrating the efficacy of two different pharmaceuticals as treatments for ATTR amyloidosis polyneuropathy. In randomized, double-blinded, placebo-controlled trials, each of these agents showed efficacy in slowing of progression or even improvement of peripheral neuropathy associated with hereditary ATTR (hATTR) amyloidosis.

These trials were accepted as sufficient demonstrations of efficacy to permit the US FDA to approve both patisaran or OnpattroTM (Alnylam Pharmaceuticals) and inotersen or TegsediTM (Ionis/Akcea Therapeutics) as the first treatments for ATTR amyloidosis. Both drugs were approved for hereditary ATTR amyloidosis with evidence of neuropathy. Furthermore, in August, a third phase 3 clinical trial was reported in the New England Journal of Medicine – this one describing the efficacy of yet another medication (tafamidis or VyndaqelTM, Pfizer) but tested in patients with ATTR cardiomyopathy. This trial (ATTR-ACT, Maurer MS et. al, NEJM 2018), in distinction from those above, showed that tafamidis was superior to placebo for survival among patients with congestive heart failure meaning that patients randomized to the drug lived longer than those who were not. FDA has yet to act on approval of this agent, but the community of patients and caregivers with ATTR amyloidosis with cardiomyopathy is eagerly awaiting its decision.

Each of these 3 new drugs acts through different mechanisms to achieve the same clinical objective – that is to arrest or reverse the progression of neuropathy or heart failure associated with ATTR amyloidosis. Thus, we have gone from a disease without pharmaceutical treatment (previously only treated with organ transplantation) to one where we now have 3 effective agents, albeit with different target populations, in a few short months.

Each of these new agents already approved by FDA was done so through accelerated pathways including Fast Track, Priority Review, and Breakthrough Therapy designations. In addition, the drugs have received Orphan Drug designation, which FDA defines as: “The Orphan Drug Designation Program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.” Cost remains a key sticking point, as now that we have approved drugs, discussions/negotiations are ongoing between sponsors (ie, pharmaceutical companies) and 3rd party payors (insurers) over pricing. As a care provider for patients with ATTR amyloidosis, I am grateful that so many of our partners in industry looked to ATTR amyloidosis as a space in which to develop desperately needed treatments. Further, I think we all can agree that the sponsors which invested great resources in dollars and time should be permitted the opportunity to recover their investment. However, the cost of these new drugs, which would in theory be given for years to decades (i.e., the duration of a patient’s life), must be balanced with the cost to the patient directly, and further the cost to our healthcare system. Such decisions are even more difficult when we are speaking about individuals, with real symptoms, and real disabilities.

Determination of a drug cost naturally considers the numbers of potential patients who can be treated. If more patients can be treated, than the cost per unit can be lower for a similar return. As I noted above, FDA considers hATTR amyloidosis an Orphan disease affecting < 200,000 people at any given time, and while this appears undoubtedly true for hATTR polyneuropathy, is it also true for ATTR cardiomyopathy? A disease is rare because it truly doesn’t affect a large number of people, or it is rare because it is unrecognized. In the case of ATTR amyloidosis cardiomyopathy, which includes both hereditary and wild-type amyloidosis (ATTRwt) the answer is probably a combination of the two.

This October, in the Journal of the American College of Cardiology, a team of cardiologists and orthopedic surgeons at the Cleveland Clinic collaborated on an innovative screening study for amyloidosis. Led by Mazen Hanna, MD (Sperry BW, J Am Coll Cardiol 2018), the investigators looked at sequential patients undergoing carpal tunnel release surgery and analyzed the resected tissue under the microscope for the presence of amyloidosis. Bilateral carpal tunnel syndrome has long been associated with systemic amyloidosis, but up to this point no one has actually examined whether one could use carpal tunnel release surgery as a means to screen for the disease. The authors placed relatively few exclusions on their study population casting the broadest possible net to include all men over age 50 years, all women over age 60 years, and excluded only those with known amyloidosis, prior trauma, or rheumatoid arthritis. Of the 319 surgeries performed over the course of a year, 98 patients were included in the final analysis, and previously unrecognized amyloidosis was found in 10 total (or 10%) patients. As predicted, 100% of patients with amyloidosis had a history of bilateral carpal tunnel syndrome, but so did 83% of those without amyloidosis. What we learned from this important study is that bilateral carpal tunnel syndrome is indeed common in systemic amyloidosis, but it is also common in patients without systemic amyloidosis, so as a clinical filter it is relatively non-specific. But we also learned that unsuspected amyloidosis can be diagnosed with carpal tunnel surgery, and furthermore, amyloidosis in this population may be seen in as many as 1 in 10. Uncommon yes, but not a rare disease. So, if this rate proves accurate in larger populations, there are likely more patients with amyloidosis than currently appreciated. How many is not yet clear. Such data feed directly into the determination of cost for these promising new agents.

Another important aspect of this study is that screening afforded a means to identify patients early in their amyloidosis disease course. In fact, 3 of the 10 identified (1 case of AL amyloidosis, 1 ATTRwt, and 1 hATTR) were given amyloidosis specific therapy. Early identification is critical in any medical disease, but particularly so in the case of systemic amyloidosis where the damage done by amyloid deposits is either slow to reverse (as in the case of neuropathy) or largely irreversible (as in the case of cardiopathy) with currently available treatments. In fact, in the ATTR-ACT trial of tafamidis, those who had less severe congestive heart failure (a marker of earlier disease) did better. Unlike in cancers such as breast or prostate, we have yet to settle upon the best screening approach for amyloidosis to apply to larger populations. In addition, we know that for AL amyloidosis patients often remain undiagnosed for months to years and see many providers before the final diagnosis of amyloidosis is made correctly (Lousada I, Advances in Therapy 2015). The same is likely also true for ATTR amyloidosis. Studies showing therapeutic success in prominent medical journals, FDA approvals, and high-profile screening reports all raise awareness of amyloidosis. These advances all work synergistically to bring amyloidosis onto the clinician’s differential diagnosis when seeing potential new cases. Screening algorithms will definitively involve integration of different modalities of testing and collaboration between providers. It is now our challenge to translate these new discoveries into clinical practice.

Disclosures

Dr. Ruberg acknowledges consulting income from Pfizer and research support from Eidos Therapeutics.