Newsroom

Information on amyloidosis is provided for informational purposes only and is not intended to be medical advice. Information concerning medical care or the suitability or use of any medication should be discussed with a medical doctor. Any information on specific treatment protocols in not an endorsement of said product.


National study seeks earlier diagnosis of ATTR cardiac amyloidosis in minorities

National study seeks earlier diagnosis of ATTR cardiac amyloidosis in minorities

NEWS RELEASE 3-SEP-2019

Boston Medical Center and Columbia University Irving Medical Center awarded grant for new trial

BOSTON – Researchers at Boston Medical Center (BMC) and Boston University School of Medicine (BUSM), in collaboration with Columbia University Irving Medical Center (CUIMC), are leading a national, multi-site study aimed to achieve earlier diagnosis of transthyretin cardiac amyloidosis (ATTR-CM). The National Institutes of Health has awarded a five-year, $7.2M grant to fund the Screening for Cardiac Amyloidosis with Nuclear Imaging in Minority Populations (SCAN-MP) trial.

Led by co-principal investigators Frederick L. Ruberg, MD at BMC/BUSM’s Amyloidosis Center of Excellence and Mathew S. Maurer, MD from CUIMC’s Cardiac Amyloidosis Program, the grant will enable the establishment of an important, community-based cohort of older minority patients with congestive heart failure. A total of 800 participants will be recruited from BMC/BUSM in Boston and the CUIMC, NYP-Allen and Harlem Hospitals in New York City.

Read full article here.


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


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.

 


FDA Approves Second Treatment for Hereditary Amyloidosis (hATTR)

FDA Approves Second Treatment for Hereditary Amyloidosis (hATTR)

Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc., announced today that the U.S. Food and Drug Administration (FDA) has approved TEGSEDI (inotersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. TEGSEDI is now approved in the U.S., European Union and Canada.

TEGSEDI powerfully reduces the production of TTR protein through a once-weekly subcutaneous injection offering patients an effective treatment for people living with polyneuropathy caused by hATTR amyloidosis.

In hATTR amyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body. TEGSEDI targets the disease at its source by reducing the production of TTR protein.

Read the entire press release here.


Alnylam Receives Approval of ONPATTRO™ (patisiran) in Europe

Alnylam Receives Approval of ONPATTRO™ (patisiran) in Europe

Alnylam Pharmaceuticals, Inc, announced recently that the European Commission (EC) has granted marketing authorization for ONPATTRO (patisiran) for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage 1 or stage 2 polyneuropathy. ONPATTRO is the first-ever RNA interference (RNAi) therapeutic to be approved in the European Union.

Read the press release here.


Alnylam Announces First-Ever FDA Approval of an RNAi Therapeutic, ONPATTRO™ (patisiran) for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults

Alnylam Announces First-Ever FDA Approval of an RNAi Therapeutic, ONPATTRO™ (patisiran) for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Aug. 10, 2018– Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the United States Food and Drug Administration (FDA) approved ONPATTRO™ (patisiran) lipid complex injection, a first-of-its-kind RNA interference (RNAi) therapeutic, for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

For more information, click here


Akcea and Ionis Announce Approval of TEGSEDI™ (inotersen) in the European Union

Akcea and Ionis Announce Approval of TEGSEDI™ (inotersen) in the European Union

TEGSEDI™ (inotersen) has received marketing authorization approval from the European Commission (EC) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).

TEGSEDI™ is now the world’s first and only RNA-targeted therapeutic approved for patients with hATTR amyloidosis. This drug brings significant benefits in both measures of neuropathy and quality of life for people living with this rare disease.

Read the complete press release here.


Combination Therapy Holds Promise for Newly Diagnosed AL Amyloidosis Patients

Combination Therapy Holds Promise for Newly Diagnosed AL Amyloidosis Patients

Updated research regarding the ANDROMEDA study indicated the efficacy of subcutaneous daratumumab and cyclophosphamide, bortezomib, and dexamethasone (CyBorD) as a combination therapy in newly diagnosed light chain AL amyloidosis patients.

“The ANDROMEDA trial, [daratumumab is] being used in a novel way because the preparation of daratumumab is being given subcutaneously and not intravenously,” said Ray Comenzo, MD, professor at Tufts University School of Medicine. “This is a big deal for patients with systemic AL amyloidosis because these patients often have some degree of cardiac compromise, therefore, getting IV infusions can be challenging for them.”

“If daratumumab and CyBorD work better together than alone, we have a new standard of therapy for patients with light chain amyloidosis,” Dr Comenzo stressed. “It’s a very exciting prospect for the newly diagnosed amyloid patients of the future to potentially have a combination of 4 drugs that are safe, and that are almost 100% effective. That is what we expect; we expect many patients to be treated and to recover.”

Watch the video interview here.