AstraZeneca PLC (LSE:AZN) will file a new drug application later this year after a successful phase III clinical trial of a treatment for a rare hereditary disease.
Eplontersen hit all its goals when researchers used it on people with transthyretin-mediated amyloid polyneuropathy.
This is a slowly progressive condition that results in the build-up of a protein called amyloid in the body's organs and tissues.
"Amyloid transthyretin polyneuropathy is a rare and fatal disease that can affect up to 40,000 people worldwide,” said Mene Pangalos, AZ’s head of biopharmaceutical research.
“These promising results show eplontersen has the potential to be a new and much-needed treatment where limited options exist and significant unmet medical need remains."
Eplontersen will be jointly commercialised with Ionis in the US and sold in the rest of the world by AstraZeneca.