Wainzua approved by MHRA for rare hereditary disease

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has approved Wainzua (eplontersen) to treat adults with polyneuropathy associated with hereditary transthyretin amyloidosis (ATTRv), which is a rare progressive condition that runs in families caused by a protein called transthyretin (TTR).

Developed by the UK’s AstraZeneca (LSE: AZN) and Ionis Pharmaceuticals (Nasdaq: IONS), Wainzua was also approved in the European Union (EU) last month for the same indication, and by the US Food and Drug Administration in December 2023.

According to analytics firm GlobalData, the  ATTR therapy sector is forecast to reach a value of $14.1 billion across the seven major markets by 2029.