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.


Alnylam Receives Positive CHMP Opinion for ONPATTRO™ (patisiran) for the Treatment of Hereditary Transthyretin-Mediated Amyloidosis

Alnylam Receives Positive CHMP Opinion for ONPATTRO™ (patisiran) for the Treatment of Hereditary Transthyretin-Mediated Amyloidosis

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a Positive Opinion recommending marketing authorization of patisiran for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adults with stage 1 or stage 2 polyneuropathy. If approved by the European Commission (EC), the medicine will be commercialized under the brand name ONPATTRO.

“Today’s recommendation by the CHMP takes us one step closer to bringing RNAi therapeutics, an entirely new class of innovative medicines, to patients around the world,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam Pharmaceuticals.

Read more details here in the press release.


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.