Protocols

Alnylam’s updated lumasiran data points to PhIII short track; Genentech snatches fourth approval for Rituxan

Pritesh Gandhi

→ Alnylam $ALNY came out with some updated data from a Phase I/II study Friday, showing the company’s rare disease drug lumasiran is on the short track for a Phase III trial. The trial — testing the drug in 20 patients with the ultra-rare primary hyperoxaluria type 1 (PH1) — has a primary endpoint of reducing the urinary oxalate at six months. Preliminary results, announced last year, suggested that lumasiran led to a mean maximal reduction in urinary oxalate of 66% in an unblinded group of four patients. In this Part B study, lumasiran demonstrated a mean maximal reduction in urinary oxalate of 64% in patients. “Based upon our recent discussions with the FDA, we are on track to advance this program into Phase III development at mid-year, with the goal of bringing lumasiran to patients around the world as rapidly as possible,” said Alnylam’s VP and GM of the lumasiran program Pritesh Gandhi in a statement.

→ Roche’s Genentech arm has scored its fourth FDA approval for Rituxan, announcing Friday that its got the green light to treat pemphigus vulgaris, a rare, serious, and potentially life-threatening condition characterized by progressive painful blistering of the skin and mucous membranes. Genetech says Rituxan is the first biologic therapy approved by the FDA for PV and the first major advancement in the treatment of the disease in more than 60 years.


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