Rare Daily Staff
Alnylam Pharmaceuticals said that Sanofi Genzyme has opted out of its rights to develop and commercialize Lumasiran, Alnylam’s experimental RNAi therapeutic for the treatment of Primary Hyperoxaluria Type 1, an ultra-rare condition that causes painful and recurrent kidney stones, which often leads to kidney failure before adulthood.
In January 2014, Alnylam and Sanofi Genzyme formed an alliance to accelerate the advancement of RNAi therapeutics for rare genetic diseases. The alliance enables Sanofi Genzyme to expand its rare disease pipeline with Alnylam’s novel RNAi technology. Sanofi Genzyme retains the right to opt into other Alnylam rare genetic disease programs for development and commercialization in territories outside of the United States, Canada, and Western Europe, as well as one right to a global license for a rare disease program, through the end of 2019 and, potentially for up to two years thereafter.
Alnylam said it intends to advance Lumasiran into a late-stage pivotal study in late 2018, and to commercialize the drug globally if approved.
“We are extremely pleased to retain worldwide development and commercialization rights for Lumasiran, expanding the number of global rare disease opportunities for Alnylam,” said Yvonne Greenstreet, chief operating officer of Alnylam. “Based on phase 1/2 clinical data presented to date, we believe Lumasiran could be a transformative treatment for patients with PH1.”
The company also announced that the U.S. Food and Drug Administration granted Lumasiran Breakthrough Therapy designation. The designation is the third the company has received to date for programs in its pipeline. Breakthrough Therapy designation is intended to expedite the development and review of experimental drugs for the treatment of serious or life-threatening conditions based on preliminary clinical evidence indicating that the drug may demonstrate substantial improvement on other available therapies.
March 12, 2018
Photo: Yvonne Greenstreet, Alnylam COO