8:05AM NPS Pharm: Pivotal study published in the Lancet Diabetes & Endocrinology; Findings confirm the potential of Natpara as a parathyroid hormone replacement therapy for adult hypoparathyroidism (NPSP) 34.79 : Co announced that its pivotal Phase 3 study of Natpara, known as REPLACE, was published online in The Lancet Diabetes & Endocrinology. Study findings underscore Natpara's potential as a replacement therapy for endogenous parathyroid hormone (PTH) in hypoparathyroidism, a rare endocrine disorder characterized by insufficient production of PTH, a principal regulator of the body's mineral homeostasis.
"Managing hypoparathyroidism can be challenging because it is limited to controlling symptoms, which does not address the underlying cause of the disorder and may have deleterious effects on major organs,"
REPLACE, a 24-week international phase 3 study of efficacy and safety of daily subcutaneous Natpara in 134 patients with hypoparathyroidism, is the largest randomized, placebo-controlled clinical trial conducted to date in patients with this rare and complex endocrine disorder. Patients were randomized 2:1 to 50 g subcutaneous once daily Natpara or placebo. In the study active vitamin D and oral calcium were progressively reduced, while Natpara could be titrated up from 50 to 75 and then 100 g.
Patients self-administered treatment for 24 weeks and were then followed for four additional weeks after the completion of the treatment phase. 53 percent of Natpara-treated patients achieved the primary endpoint by decreasing doses of oral calcium and active vitamin D by 50 percent or more, while maintaining serum calcium levels by the end of the treatment phase. In contrast, only 2 percent of the placebo group (P<0.001) met the primary endpoint.
Among secondary endpoints, by Week 24, 43 percent (36/84) of patients treated with Natpara were able to achieve independence from active vitamin D therapy and required only 500 mg/day or less of oral calcium, as compared to five percent (2/37) of patients treated with placebo (p<0.0001).
