- Updated data from an ongoing Phase 1/2 open-label extension study evaluating Alnylam Pharmaceuticals ' (ALNY -0.8%) RNAi therapeutic givosiran in patients with acute hepatic porphyria (AHP) showed a sustained treatment benefit and favorable safety profile. The results were presented at The Liver Meeting in San Francisco.
- At data cutoff, the mean time on treatment was 13.6 months. Monthly dosing produced annualized attack rate (AAR) by 93% and annualized hemin use by 94% (hemin for injection, branded as Panhematin by Recordati Rare Diseases, works by lowering the production of certain enzymes in the body that play key roles in hepatic porphyria attacks). 44% (n=7/16) of patients achieved AARs of zero.
- Serious adverse events were reported in four patients, one (severe allergic reaction) definitely related to givosiran (resolved with medical management).
- A Phase 3 study, ENVISION, is ongoing. The company plans to seek full U.S. approval for the indication. A rolling NDA filing will be initiated this quarter.
- Previously: Alnylam to seek full approval for givosiran for AHP (Oct. 15)
- Now read: Geron: Of Abstracts And Abstractions
Original article