Potentially good news arrived for patients with hereditary TTR amyloidosis, as the treatment drug inotersen received it’s New Drug Application approval and will go into review by the U.S. Food and Drug Administration (FDA), originally reported by Pharmavoice. If approved by the FDA, it could lead to significant improvement to the lives of those diagnosed with the fatal disease.
Hereditary TTR amyloidosis, (hATTR) is a rare disease that causes a abnormal buildup of proteins in the peripheral nerves, which then can lead to disfunction with heart rate, blood pressure, digestion and more. To read more about this rare disease, click here.
Unfortunately for those with the disease, there are a limited amount of treatment options available. Many companies are starting to realize the urgent need for treatment. Receiving the next steps for approval is huge for Ionis Pharmaceuticals Incorporation, the company who developed inotersen. Sarah Boyce the chief business officer at IPI has high hopes in the drug and is laying down next step preparation so, hoping they get the approval, they will have everything in place to move forward as quickly as possible.
After Phase 3 of their study, results showed a significant improvement to the quality of life for patients with the disease. This drug is used to limit the production of transthyretin, TTR, as those protein buildups are lead to dysfunction throughout the body. This could lessen the buildups which have led to early onset of death for many patients. Thankfully, the FDA approved a fast track order for the drug, giving the researchers and patients alike hope in the product’s potential.
Ionis continues to be the leading company for RNA-focused drug development, and they continually look to find treatments for diseases with minimal treatment options. Ionis has seen passed success with other drugs such as volanesorsen, as well as much success through their affiliate, Akcea Therapeutics, that develops and commercializes similar drugs. Time will tell the future of inotersen and patients with hATTR, but right now all is looking positive.
