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Ultra-rare, potentially life-threatening genetic disease.
The FDA has approved givosiran (Givlaari, Alnylam Pharmaceuticals) for the treatment of adults with acute hepatic porphyria (AHP), an ultra-rare genetic disease.
Givosiran is an aminoelvulinate synthase 1-directed small interfering RNA that targets aminoelvulinic acid synthase I (ALASI).
Approval for givosiran was granted based upon results of a phase 3 ENVISION study that included 94 patients across 36 study sites in 18 countries.
In the givosiran arm, patients experienced 70% fewer porphyria attacks compared to those receiving the placebo treatment. Givosiran also resulted in similar reductions in IV hemmin use, urinary aminolevulinic acid, and urinary porphobilinogen, according to Alnylams’ official release.
“Adults with AHP now have a new treatment option that has demonstrated the ability to reduce the frequency of porphyria attacks by specifically addressing factors associated with attacks and other disease manifestations of AHP,” said Manisha Balwanii, MD, principle investigator and associate professor of the department of genetics and genomic sciences and department of medicine at the Icahn School of Medicine at Mount Sinai.
Givosiran is contraindicated in patients with severe hypersensitivity to givosiran.
Warnings and precautions issued with givosiran’s prescribing information include anaphylactic reactions, hepatic toxicity, renal toxicity, and injection site reactions.
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Concomitant use with CYP1A2 or CYP2D6 substrates for which minimal concentration changes may lead to serious or life-threatening toxicities should be avoided as givosiran has been known to increase the concentration of the substrates thus potentially increasing adverse reactions.
The most common adverse reactions reported with the use of givosiran include nausea and injection site reactions.