Clinical trial news : Alnylam Pharmaceuticals said that it has wrapped up enrollment of patients for a phase 3 trial called ENVISION for evaluating givosiran for the treatment of acute hepatic porphyrias (AHPs), a family of rare, genetic diseases.
According to the US biopharma company, givosiran is an RNAi therapeutic that has been designed to target aminolevulinic acid synthase 1 (ALAS1).
A total of 94 patients with acute hepatic porphyrias were enrolled in the late-stage trial who were randomized in 36 sites across 18 countries, said Alnylam Pharmaceuticals.
Patients were randomized on an equal basis to be subjected to 2.5 mg/kg of givosiran or placebo subcutaneously on a monthly basis, for a treatment period of six months.
The primary endpoint of the ENVISION trial is the annualized rate of porphyria attacks requiring hospitalization, urgent healthcare visit or hemin administration at home during the six-month treatment period.
The US biopharma company estimates to report topline results of the interim analysis of the givosiran phase 3 trial by the end of September and topline results on the primary endpoint of annualized attack rate in early 2019, after six months of treatment.
Alnylam Pharmaceuticals revealed that the interim analysis of the ENVISION clinical trial is based on reduction of urinary aminolevulinic acid (ALA) levels from about 30 patients at three months of treatment as a surrogate biomarker that is reasonably expected to predict clinical benefit.
The US biopharma company is aiming to submit an NDA for givosiran at or around year-end 2018, subject to its review and also that of the US FDA’s at the time of interim analysis and also positive results and acceptable safety of the RNAi therapeutic. Alnylam Pharmaceuticals revealed that it is pursuing an accelerated FDA approval for givosiran.
Akin Akinc – Vice President and General Manager, Givosiran Program at Alnylam Pharmaceuticals, commenting on the ENVISION phase 3 trial of givosiran, said: “We’re pleased to have completed enrollment in the ENVISION Phase 3 study, and we’re thankful to the porphyria community for their support in helping to raise awareness about the importance of clinical trials in rare diseases.
“The fact that we achieved this important milestone significantly ahead of schedule – in addition to exceeding the initial enrollment target – highlights the urgent demand and high unmet need for novel therapies in this disease setting. We remain committed to advancing givosiran, which we believe has the potential to transform the treatment landscape for patients with AHPs.”
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