A recent article on Yahoo Finance, reports that Alnylam Pharmaceutical Inc.’s RNAi therapeutic, Amvuttra, has been approved by the FDA to treat polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis.

Amvuttra (Vutrisiran)

Amvuttra is an RNAi therapeutic that is used to treat adults with polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis. Yahoo Finance reports the FDA has approved it to be used as a subcutaneous injection which will be administered to patients once every three months at their given indication.

FDA Approval

The FDA approved the treatment following a phase III HELIOS-A study for nine months. The study was focused on discovering the safety and effectiveness of Amvuttra in hATTR amyloidosis patients with polyneuropathy. The results of the study concluded that Amvuttra greatly improved the signs and symptoms associated with polyneuropathy. The article notes that more than half of the patients in the study either had their symptoms stopped or even reversed.

The approval of Amvuttra means that it is the first and only FDA-approved therapy that can reverse neuropathy impairment with subcutaneous administration.

Read more about Amvuttra’s FDA approval over at Yahoo Finance.

Hereditary ATTR Amyloidosis

Hereditary ATTR amyloidosis is a familial subtype of amyloidosis. Hereditary ATTR amyloidosis is an inherited genetic disease affecting the nervous system and the heart.

The genetic mutation that causes hereditary ATTR amyloidosis produces a protein that forms into an abnormal shape. These abnormal “misfolded” proteins can be deposited and cluster in the body’s nerves and organs, causing complications.

There are three categories of ATTR amyloidosis:

1. Familial amyloid cardiomyopathy (FAC)
2. Familial amyloid polyneuropathy (FAP)
3. Senile systemic amyloidosis also knows as wild-type ATTR amyloidosis

 Symptoms

For each patient, the symptoms will depend on which organs are affected by the protein deposits:

Heart

• Nausea
• Weight loss
• Insomnia
• Progressive fatigue
• Dizziness
• Shortness of breath
• Leg swelling
• Palpitations and abnormal heart rhythms (atrial fibrillation)
• Chest pains

Nervous System

• Peripheral neuropathy
• Sensorimotor impairment
• Autonomic neuropathy

Digestive System

• Nausea
• Diarrhea or constipation
• Bladder control issues
• Weight loss
• Loss of appetite or a feeling of fullness after eating small amounts

Kidneys

• Nephrotic syndrome (excess protein in the urine)
• Swelling of legs, belly, arms, and lungs
• Renal