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FDA Postpones Final Decision on Thyroid Drug Natpara
The FDA has extended its Prescription Drug User Fee Act (PDUFA) action date for the thyroid treatment Natpara by 3 months –– from October 24, 2014 to January 24, 2015.
The FDA extended the action date to provide time for a full review of a major amendment. In addition, the agency requested that the product’s developer (NPS Pharma) submit a risk evaluation and mitigation strategy (REMS) for the treatment. To date, the FDA has not requested that additional clinical studies be completed before the approval of Natpara; however, the company expects a post-approval study commitment.
If approved, Natpara is expected to be launched in the U.S. in the second quarter of 2015.
Natpara is a bioengineered replacement therapy for endogenous parathyroid hormone (PTH) that has been developed for the treatment of hypoparathyroidism, a rare endocrine disorder characterized by insufficient levels of PTH. Insufficient PTH levels can lead to numerous physiologic abnormalities, including low levels of calcium and high levels of phosphate in the blood, and an inability to convert native vitamin D into its active state, which helps the body properly absorb oral calcium. Hypoparathyroidism affects approximately 50,000 people in the U.S.
With no approved replacement therapy available for hypoparathyroidism, the only clinical option is to use large doses of oral calcium and active vitamin D to try and manage symptoms of hypocalcemia by increasing serum calcium, but this does not address the underlying cause of the disorder. In addition, the administration of this regimen does not allow the activation of native vitamin D and is often difficult because of calcium fluctuations and the challenges of balancing symptom control while minimizing the amount of excess calcium being filtered through the kidneys. The long-term use of calcium and vitamin D may can lead to serious complications because increasing serum calcium without decreasing serum phosphate may lead to irreversible calcium–phosphate deposits in the kidneys, heart, or brain.
Source: NPS Pharma; October 23, 2014.