Alnylam suspends dosing in all ongoing fitusiran studies after patient death

Alnylam Pharmaceuticals (ALNY) announced today an update on the company’s fitusiran and givosiran investigational RNAi therapeutic programs.

With fitusiran, an RNAi therapeutic in development for the treatment of hemophilia A and B with or without inhibitors, Alnylam is reporting a fatal thrombotic event in a patient with hemophilia A without inhibitors in the Phase 2 open-label extension study of fitusiran.

As a result, the company has suspended dosing in all ongoing #fitusiran studies pending further review of the safety event and development of a risk mitigation strategy. “Based on overall consideration of fitusiran’s benefit-risk profile, Alnylam is guiding that it aims to resume dosing as soon as possible upon agreement with global regulatory authorities and with appropriate protocol amendments in place for enhanced patient safety monitoring.”

With givosiran, an RNAi therapeutic in development for the treatment of acute hepatic porphyrias, Alnylam said it has reached alignment with the FDA on a Phase 3 study design which includes an interim analysis based on reduction of a urinary biomarker, aminolevulinic acid, as a surrogate endpoint reasonably likely to predict clinical benefit.

Based on the new givosiran Phase 3 design, the company is now guiding that pending FDA review of the program at the time of interim analysis and assuming positive results, it expects to submit a new drug application at or around year-end 2018.

Based on today’s update, Alnylam will postpone its fitusiran RNAi Roundtable webinar previously scheduled for September 12 until a later date.

“We are deeply saddened to learn of this patient’s death, and we extend our sympathies to his family,” said Akshay Vaishnaw, M.D., Ph.D, Executive Vice President of R&D at Alnylam.

“We believe that fitusiran holds great promise as a potential treatment option for patients with hemophilia, and we remain fully committed to its ongoing development.

Following further investigation of this safety finding, implementation of a risk mitigation strategy, and alignment with global regulatory authorities, we expect to resume fitusiran dosing in our clinical studies as soon as possible, potentially as early as late 2017, with a goal of advancing this innovative investigational medicine to hemophilia patients in need.”

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ALNY has a 52-weeks trading range of $31.38 – $89.45. It closed at $86.02, last traded at $77.00.

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