Leading pharmaceutical conglomerate, Akcea Therapeutics and parent Ionis Pharmaceuticals Inc. have recently received approval for their hereditary transthyretin amyloidosis (hATTR) treatment drug, Tegsedi™ by the U.S. Food and Drug Administration (FDA).
hATTR being a rare disease causes buildup of amyloid in the peripheral nervous system and multiple organs, and can result in death within 3 to 15 years of symptom onset. On the contrary, Tegsedi is designed to block production of the TTR protein, substantially reducing the levels of the protein irrespective of mutation type or stage of disease.
Despite Tegsedi’s capabilities the drug can yet be associated with serious bleeding issues caused due to low platelet counts, as well as potential kidney causes. Keeping these issues in mind, Tegsedi will only be made available via a restraining program called Tegsedi REMS (Risk Evaluation and Mitigation Strategy), cite sources.
Paula Soteropoulos, CEO at Akcea Therapeutics was quoted saying that Tegsedi is the first and only RNA-targeting therapeutic that could effectively reduce the production of TTR protein through a once-per-week intravenous injection, offering patients a treatment for polyneuropathy caused by hATTR amyloidosis.
The company believes in the drug’s efficiency, combined with its ease of self-administration and expects Tegsedi to be an excellent choice for many patients, Soteropoulos said.
Sources cite that Akcea, in addition to the regulatory approval, has also announced its new struck deal with Accredo specialty pharmacy, a subsidiary of Express Scripts, as the distributor of Tegsedi.
As per Steve Miller, Express Scripts’ Chief Medical Officer, Tegsedi being a self-administered medication should reduce administration costs and drug wastage. By introducing competition into the marketplace, the total cost of care of Tegsedi can be reduced.
In a bid to enable hATTR amyloidosis patients access to Tegsedi, Akcea has also created a patient support program called, Akcea Connect. Sarah Boyce, Akcea Therapeutics’ President, said the company is ready to help patients through every stage of treatment with Tegsedi.