On Friday, the US Food and Drug Administration (FDA) approved NPS Pharmaceuticals’ rare endocrine disorder drug Natpara (parathyroid hormone), justifying Shire’s $5.2 billion acquisition of the company.
The agency approved NPS’ Natpara to control hypocalcemia (low blood calcium levels) in patients with hypoparathyroidism, a rare disease that affects approximately 60,000 people in the US. Hypoparathyroidism occurs when the body secretes abnormally low levels of parathyroid hormone, which helps regulate calcium and phosphorus levels in the body. It is caused by loss of function of the parathyroid glands and most commonly occurs following surgical removal of the parathyroid glands, and in some cases as a result of autoimmune or congenital diseases.
Patients with hypoparathyroidism can experience numbness, tingling, muscle twitching, spasms or cramps, abnormal heart rhythm and seizures as a consequence of low blood calcium levels. Additionally, hypoparathyroidism is associated with long-term complications such as kidney damage, kidney stones, development of cataracts and calcification of soft tissues.
Natpara is a once-daily, injectable drug, which helps regulate the body’s calcium levels. The drug received orphan drug designation from the FDA, since it is intended to treat a rare disease. Natpara is the first drug approved to treat hypoparathyroidism. Its approval comes with a boxed warning, alerting patients of the risk of bone cancer, which was seen during studies on rats with the drug.
The agency’s approval followed positive results from a late-stage trial, which showed that 53 percent of patients administered Natpara successfully cut down their intake of calcium and Vitamin D supplements, compared to only two percent of patients who were given placebo.
Earlier this month, Shire agreed to acquire NPS to expand its rare-disease portfolio. The $5.2 billion deal is Shire’s biggest acquisition and its second deal since its proposed $52 billion purchase by AbbVie fell through.
“The FDA’s approval of Natpara provides a new treatment option for patients with hypoparathyroidism – a devastating rare disease with significant unmet need. The Natpara label is in line with our expectations, and we believe this approval further validates Shire’s decision to acquire NPS Pharma, which is an excellent strategic fit allowing us to leverage our market expertise, core capabilities in rare disease patient management, and global footprint. We look forward to combining our strengths with NPS Pharma to launch Natpara in the US after the expected close of the transaction in Q1 of this year,” said Shire’s Chief Executive Officer, Flemming Ornskov, MD, MPH.
Sources: Food and Drug Administration; Shire plc