Amgen and UCB announce U.S. FDA acceptance of biologics license application for romosozumab.
Amgen and UCB announced that the U.S. FDA has accepted for review the Biologics License Application (BLA) for romosozumab, an investigational monoclonal antibody for the treatment of osteoporosis in postmenopausal women at increased risk of fracture. Romosozumab works by binding and inhibiting the activity of the protein sclerostin, a protein naturally occurring in the bone, thereby increasing bone formation and decreasing bone resorption.
“We believe romosozumab could serve as an important therapeutic option for osteoporosis patients with an increased risk of fracture,” said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. “We look forward to potentially providing a new therapy to address a critical unmet medical need and further supporting patients worldwide.”
The BLA, submitted on July 19, 2016, is based on data from the pivotal Phase 3 placebo-controlled FRActure study in postmenopausal woMen with ostEoporosis (FRAME) in approximately 7,200 patients.
“Once a patient suffers a fragility fracture, the burden of osteoporosis can have a tremendous impact on a patient’s life,”1,2 said Dr. Pascale Richetta, head of bone and executive vice president, UCB. “We are pleased with the FDA’s acceptance of the BLA filing for review and hope to continue the regulatory process towards U.S. approval to introduce a potential therapy to reduce the risk of fractures for this patient population.”
The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of July 19, 2017, for romosozumab.
Romosozumab is an investigational bone-forming monoclonal agent and is not approved by any regulatory authority for the treatment of osteoporosis. It is designed to work by inhibiting the activity of the protein sclerostin, and has a dual effect on bone, both increasing bone formation and decreasing bone breakdown. Romosozumab is being studied for its potential to reduce the risk of fractures in an extensive global Phase 3 program. This program includes two large fracture trials comparing romosozumab to either placebo or active comparator in more than 10,000 postmenopausal women with osteoporosis. Amgen and UCB are co-developing romosozumab.
About the FRAME Study
FRAME is a multicenter, international, randomized, double-blind, placebo-controlled, parallel-group study in postmenopausal women with osteoporosis, defined as low bone mineral density at the total hip or femoral neck. The study evaluated the effectiveness of romosozumab treatment, compared with placebo, in reducing the risk of new vertebral fractures through 12 months. The study also further evaluated if romosozumab treatment for 12 months followed by denosumab treatment for 12 months, compared with placebo followed by denosumab treatment, was effective in reducing the risk of new vertebral fractures through 24 months. In addition, clinical fracture (a composite endpoint which encompasses all symptomatic fractures, both non-vertebral and painful vertebral fractures) risk reduction, non-vertebral fracture (fractures outside of the spine, excluding sites that are not considered osteoporotic, fractures due to high trauma or pathologic fractures) risk reduction and other endpoints were assessed at 12 and 24 months.
7,180 patients were randomized 1:1 to receive either 210 mg romosozumab subcutaneous (SC) monthly (QM) or placebo SC QM for the 12-month double-blind study period. After the placebo-controlled study period, patients entered the open-label phase where all patients received 60 mg denosumab SC every six months (Q6M) for 12 months, while remaining blinded to initial treatment. An additional 12 month extension period of open-label 60 mg denosumab SC Q6M is currently ongoing.
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