Palopegteriparatide is a potential long-term therapy for adults with hypoparathyroidism, new findings suggest.
"The company has said that they're hopeful the issues will be addressable and that the FDA did not have any concerns about safety," Rubin toldRubin presented long-term efficacy and safety data from the Phase 2, which involved 57 of the initial 59 participants who completed week 110 of an open-label extension of the trial.
At 110 weeks, all 57 patients were able to stop taking active vitamin D, and 53 of the 57 patients achieved independence from conventional therapy, defined as taking 0 µg/day of active vitamin D and no more than 600 mg/day of calcium . A total of 44 patients were not taking any calcium or active vitamin D.
"This is a really important outcome because we know that high urine calcium in these patients sets them at risk for going on to develop nephrocalcinosis, nephrolithiasis, and ultimately, chronic kidney disease," Rubin said. Bone mineral density assessed by dual-energy X-ray absorptiometry normalized, primarily in the first 26 weeks. For lumbar spine L1-L4, mean Z-scores dropped from 1.6 to 1.0 at 26 weeks and down to 0.7 by week 100. For total hip, those values were 1.0, 0.6, and 0.4, respectively. The values approached age- and sex-matched norms, Rubin noted, to"perhaps where their skeleton would be if they hadn't had hypoparathyroidism.
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