FDA approves Tegsedi ( inotersen )

On October 8th, Akcea and Ionis' Tegsedi were approved by the US FDA for hereditary parathyroid hormone amyloidosis (HATTR), a rare disease that forms starch in the peripheral nervous system and multiple organs. Protein accumulation. Tegsedi (inotersen) is designed to block the production of TTR proteins, and the slow accumulation of TTR proteins causes sensory, motor and autonomic dysfunction in patients with the disease.

Within 3-15 years of the onset of symptoms, hATTR may cause death, and no drug that has improved the disease has been approved for the treatment of the disease, affecting approximately 50,000 patients worldwide. In the clinical trial (NEURO-TTR), according to the company, Tegsedi significantly reduced protein levels regardless of the type of mutation or stage of the disease.

"Tegsedi is the first and only RNA-targeted therapy, administered subcutaneously once a week, which effectively reduces TTR protein production and provides effective treatment for patients with multiple neuropathy caused by hATTR amyloidosis. We believe this effect will Make TEGSEDI a good choice for many patients,” said Acteropoulos, CEO of Akcea.

"hATTR amyloidosis is a deadly disease that affects multiple organs and body systems, taking away people's ability to work and the ability to perform everyday tasks, all of which makes patients feel that their lives are under control," Blood Gertz, an academic expert and honorary chairman of the Mayo Clinic, pointed out. "Tegsedi has been shown to reduce TTR by nearly 80%, a proven method for treating this disease. Compared to placebo, rapid and sustained improvement in a significant proportion of patients, as well as disease indicators The reversal, in addition, the independence of self-injection not only gives patients, but also hope for their caregivers and families.” The therapy was approved in Europe in July.

DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

TEGSEDI is a drug for the treatment of nerve damage in adult patients with hereditary transthyretin-mediated amyloidosis. Transthyretin-mediated amyloidosis is the buildup of abnormal deposits of a substance called amyloid in the body's organs and tissues. Amyloid disrupts the function of organs and tissues.

How is this drug used?

TEGSEDI is injected under the skin (a subcutaneous injection) once weekly.

What are the benefits of this drug?

Compared to patients receiving placebo injections, patients who received TEGSEDI had fewer symptoms of nerve damage, as well as better muscle strength and reflexes.

Were there any differences in how well the drug worked in clinical trials among sex, race and age?

• Sex: TEGSEDI worked similarly in males and females.
• Race: The majority of patients in the trial was White. Differences in how well the drug worked among races could not be determined because of the small number of patients in other races.
• Age: TEGSEDI worked similarly in patients younger and older than 65 years of age.