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FDA committee approves stem cell-based romosozumab for treating osteoporosis

THOUSAND OAKS, CA (US), BRUSSELS (BE), January 2019 — The US Food and Drug Administration (FDA) Bone, Reproductive, and Urologic Drugs Advisory Committee (BRUDAC) recently voted in favor of approving Amgen and UCB’s stem cell-based therapy for spinal cord injury, romosozumab (EVENITY), for the treatment of postmenopausal women with osteoporosis at high risk for fracture.  

In the vote, the committee also stressed the necessity of post-marketing follow-up. This was due mainly to some concern over whether the cardiovascular (CV) safety of romosozumab has been adequately characterized, and whether the indication for the drug should be narrowed to adults at high risk for fracture with low CV risk.. The advisory panel’s recommendations are nonbinding, although the FDA often follows its suggestions.

Amgen reported that 19 clinical trials were included in the romosozumab development program, which enrolled approximately 14,000 patients. The primary phase 3 clinical trials included in the review were FRAME, a placebo-controlled study involving 7,180 postmenopausal women with osteoporosis at risk for fracture; ARCH, an active comparator-controlled study involving 4,093 postmenopausal women with osteoporosis and with prior history of fracture; and STRUCTURE, an active comparator-controlled study involving 436 postmenopausal women with osteoporosis.

In the phase 3 FRAME trial, 16 of 3321 patients (.5 percent) in the romosozumab-treated group experienced new vertebral fractures compared to 59 of 3322 (1.8 percent) in the placebo-treated patients. This difference demonstrated a 73 percent lower risk with romosozumab; P<.001. Clinical fractures occurred in 58 of 3589 romosozumab-treated patients (1.6 percent) compared with 90 of 3591 (2.5 percent) in the placebo-treated patients, showing a 36 percent lower risk with romosozumab; P=.008). Additionally, 56 of 3589 of the romosozumab-treated patients (1.6 percent) experienced nonvertebral fractures compared to 75 of 3591 (2.1 percent) in the placebo-treated patients (P=.10).

After each group made the transition to denosumab, the rates of vertebral fractures were significantly lower at 24 months in the romosozumab-treated patients than in the placebo-treated patients (.6 percent [21 of 3325 patients] in the romosozumab group vs. 2.5 percent [84 of 3327] in the placebo group, a 75% lower risk with romosozumab; P<.001).

Hyperostosis, cardiovascular events, osteoarthritis, and cancer included adverse events, which were balanced between the groups. Two cases of osteonecrosis of the jaw and 1 atypical femoral fracture were noted in the romosozumab group.

In 2017, the FDA rejected Amgen’s application for approval of romosozumab after phase 3 data linked it to an increased risk of CV adverse events. Amgen resubmitted its application last July (2018).

Earlier this month, Japan announced approval of the drug to treat osteoporosis in that country.

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