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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.


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.


New Results for Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy

New Results for Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy

Earlier this week at the European Society of Cardiology Congress in Munich, Germany, Pfizer announced that in patients with transthyretin amyloid cardiomyopathy, Tafamidis was associated with reductions in all-cause mortality and cardiovascular-related hospitalizations and reduced the decline in functional capacity and quality of life.

Read the entire press release here.


The Assistance Fund Announces New Hereditary Amyloidosis Financial Assistance Program

The Assistance Fund Announces New Hereditary Amyloidosis Financial Assistance Program

The Assistance Fund, an independent charitable patient assistance foundation, recently launched a new fund to support hereditary amyloidosis patients. This type of amyloidosis is one of the systemic amyloidosis diseases that are caused by inheriting a gene mutation.

“Finally, patients have hope for treating this disease,” said Mary O’Donnell, President and CEO of the Amyloidosis Foundation. “The Assistance Fund’s financial assistance program for hereditary (hATTR) amyloidosis will provide extremely important support for patients who may otherwise be unable to seek treatment due to high out-of-pocket costs.”

They provide financial assistance for medication copays, health insurance premiums, and basic healthcare needs to patients with certain disease types.

For more information, call (855) 512-2801.

Read the entire press release here.

To apply for assistance, 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.


Alnylam Receives Orphan Drug Destination from FDA to Treat ATTR Amyloidosis

Alnylam Receives Orphan Drug Destination from FDA to Treat ATTR Amyloidosis

Alnylam Pharmaceuticals, Inc. announced today that the FDA has granted Orphan Drug Designation to ALN-TTRsc02. This drug has the potential to be a once-quarterly, low volume, subcutaneously administered RNAi medication in the management of ATTR amyloidosis.

The FDA 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.

The European Commission also recently issued the decision to adopt the opinion of the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) and designate ALN-TTRsc02 as an orphan medicinal product in the European Union (EU) for the treatment of transthyretin-mediated amyloidosis.

Read the press release here.


Inotersen for hATTR Amyloidosis Received Positive Opinion by CHMP in Europe

Inotersen for hATTR Amyloidosis Received Positive Opinion by CHMP in Europe

The Akcea Team announced that the Committee for Medicinal Products for Human Use (CHMP) of the Eurpoean Medicines Agency (EMA) adopted a postive opinion recommending approval of TEGSEDI (inotersen) for the treatment of state 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).

From here, the positive opinion will be referred to the European Commission (EC), which grants marketing authorization for medicines in the European Union, as well as to European Economic Area members Iceland, Liechtenstein and Norway.

Click here for the press release.

 


FDA Grants Breakthrough Therapy Designation for Tafamidis

FDA Grants Breakthrough Therapy Designation for Tafamidis

Pfizer recently announced that tafamidis, its investigational therapy for the treatment of patients with, transthyretin (TTR) cardiomyopathy, received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA).

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.

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. If a drug is designated as a breakthrough therapy, the FDA may expedite the development and review of such drug.

In patients with this disease, TTR breaks up and may form fibrils called amyloid. Amyloid can build up around your nerves and in other places in your body, preventing them from working normally. Eventually, the amyloid causes the symptoms of this disease.

Click here for the press release.