According to a press release from FirstWord Pharma, Gen and Alnylam Pharmaceuticals have signed an exclusive distribution deal in Turkey for RNAi therapeutic ONPATTRO. This therapy drug will treat patients with hereditary ATTR amyloidosis who also have polyneuropathy.

Alnylam Pharmaceuticals, who currently offers RNAi therapy options throughout North America, Europe, Africa, and the Middle East, has stated that they have yet to performed extensive studies – or offered their therapies – in Turkey. As such, they look forward to their partnership with Gen and newfound opportunity to bring these therapies to patients in need.

Read the full release here.

What is Amyloidosis?

Broadly, amyloidosis is a condition in which amyloids, an insoluble protein, build up throughout the body. These build-ups often occur in important organs, causing poor organ function. Symptoms of amyloidosis can include nausea, fatigue, renal failure, weight loss, and neuropathy.

Hereditary ATTR amyloidosis (hATTR amyloidosis) is an inherited form of this disease that more specifically affects the heart and nervous system. The proteins in hATTR amyloidosis are misshapen. Learn more about hereditary ATTR amyloidosis here. 

Hereditary ATTR Amyloidosis and Polyneuropathy

Polyneuropathy, also known as peripheral neuropathy, common displays in patients with hATTR amyloidosis, particularly if the amyloids are building up throughout the nervous system.

According to the Mayo Clinic, polyneuropathy occurs when peripheral nerves on the brain and spinal cord are damaged. The condition usually occurs as a result of another condition or injury. Symptoms of polyneuropathy include weakness, sensitivity to touch, and pain.

If you have hATTR amyloidosis and are experiencing pain or tingling in your feet or hands, please consider visiting a doctor to ask about polyneuropathy. Catching it early can increase chances of both stopping the nerve damage and reducing your pain.

About ONPATTRO

The Creators

Gen

Gen is a Turkish pharmaceutical company with offices across the globe. The GMP-certified company has been operating for over 20 years. They aim to provide patients with rare diseases with ethical, effective, and reliable treatments. Learn more on their website. 

Alnylam Pharmaceuticals

Alnylam Pharmaceuticals, headquartered in Cambridge, MA, is a biopharmaceutical company focused on treating patients with rare diseases and disorders through RNA interference (RNAi) therapies. Founded in 2002, Alnylam aims to create RNAi therapies for patients with central nervous system disorders, genetic diseases, ocular diseases, and more. They are specifically drawn to assisting patients who have limited treatment options. Learn more about Alnylam Pharmaceuticals and their 2020 strategy on their website.

ONPATTRO for hATTR amyloidosis and polyneuropathy

ONPATTRO is a RNAi drug that was approved in North America, Japan, Brazil, Switzerland, and the European Union for the treatment of hATTR amyloidosis with polyneuropathy. It is delivered intravenously. ONPATTRO targets a protein called transthyretin (TTR). Mutations in TTR are a leading cause of amyloidosis.

To prevent TTR from accumulating in the liver, and thus reducing the negative affects of polyneuropathy, ONPATTRO silences TTR messenger RNA to keep the protein from being created. Learn more about ONPATTRO here.

RNAi Therapies

RNA interference (RNAi) is a leading type of contemporary gene therapy. Using RNAi helps researchers silence specific genes, keeping those with mutations or that are harmful from causing problems throughout the body. As a result, researchers are able to create more targeted treatments to address genetic diseases and disorders.

Safety Information

Although ONPATTRO has shown to provide benefits to patients with hereditary ATTR amyloidosis, some adverse reactions have occurred. For instance, 19% of study patients experienced infusion-related reactions as compared to 9% of placebo-treated patients. These reactions include shortness of breath, nausea, and back and abdominal pain. 30% of patients also experienced peripheral edema, or swelling in the legs or other extremities. In addition, some patients may experience vitamin A deficiency. This can cause ocular issues including inflammation, dry eyes, or corneal ulceration.

However, researchers offer ways to combat negative side effects. Patients taking ONPATTRO can take 2500 IU vitamin A daily to address ocular impacts. Additionally, receiving a mix of corticosteroids, paracetamol, and antihistamines an hour before intravenous medication delivery can help resolve negative symptoms.