Givlaari approved in EU following recent CHMP nudge

Alnylam has received approval from the European Commission for its second RNAi medicine - this time an acute hepatic porphyria (AHP) drug, Givlaari (givosiran).

The drug has been approved for the ultra-rare genetic condition in adults and adolescents aged 12 years and older, to treat the debilitating attacks of severe abdominal pain, vomiting and seizures that patients experience.

The drug, which is a first-of-its kind injectable RNAi therapeutic, has been shown to reduce the frequency of porphyria attacks.

The commission says that the marketing authorisation is based on data from the ENVISION Phase III trial, which showed a 74% reduction in the rate of porphyria attacks. The results mean that the RNAi therapy is the first and only proven drug to prevent attacks, reduce chronic pain and improve quality of life.

Just last month the European Medicines Agency (EMA)’s human medicines committee (CHMP) recommended the drug be approved, supported by the organisation’s PRIority Medicines (PRIME) scheme, which assists developers of promising new medicines that address unmet medical needs.

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