The U.S. Food and Drug Administration (FDA) has approved Natpara (parathyroid hormone) to control hypocalcemia (low blood calcium levels) in patients with hypoparathyroidism, a rare disease that affects approximately 60,000 people in the United States.
This is great news for NPS Pharma, Shire, and patients with hypoparathyroidism. Natpara was developed by NPS Pharma, a company expected to be purchased by Shire for $5.2 Billion in the first quarter of 2015.
Francois Nader, MD, president and chief executive officer of NPS Pharma said
“Natpara is a significant advance in the care of hypoparathyroidism and we are very pleased to offer the first and only parathyroid hormone approved for people living with this rare disorder. In clinical studies, Natpara has been shown to increase serum calcium levels and reduce the need for large doses of calcium and active vitamin D.”
Shire's Chief Executive Officer, Flemming Ornskov, MD, MPH, commented
"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 U.S. after the expected close of the transaction in Q1 of this year."
Hypoparathyroidism occurs when the body secretes abnormally low levels of parathyroid hormone, which helps regulate calcium and phosphorus levels in the body. In most cases, hypoparathyroidism occurs as a result of surgical removal of the parathyroid glands. 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. Hypoparathyroidism is also associated with long-term complications such as kidney damage, kidney stones, development of cataracts and calcification of soft tissues.
Jean-Marc Guettier, M.D., director of the Division of Metabolism and Endocrinology Products in the FDA’s Center for Drug Evaluation and Research said
“People with hypoparathyroidism have limited treatment options and face challenging symptoms that can severely impact their quality of life.”
“This product offers an alternative to patients whose calcium levels cannot be controlled on calcium supplementation and active forms of vitamin D.”
Natpara, a hormonal injection administered once daily, helps to regulate the body’s calcium levels. The safety and effectiveness of Natpara were evaluated in a clinical trial of 124 participants randomized to receive Natpara or placebo. The study found that 42% of Natpara-treated participants achieved normal blood calcium levels on reduced doses of calcium supplements and active forms of vitamin D, compared to three percent of placebo-treated participants.
Natpara carries a boxed warning that bone cancer (osteosarcoma) has been observed in rat studies with Natpara. It is unknown whether Natpara causes osteosarcoma in humans, but because of a potential risk of osteosarcoma, Natpara is only recommended for use in patients whose hypocalcemia cannot be controlled on calcium supplementation and active forms of vitamin D, and for whom the potential benefits are considered to outweigh this potential risk. Natpara is only available through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS).
The most common side effects observed in Natpara-treated participants were sensations of tingling, tickling, pricking, or burning of the skin (paraesthesia); low blood calcium; headache; high blood calcium; and nausea.
At press time, it was unclear when the drug will be available but NPS Pharma has noted that its NPS Advantage program is in place to assist patients. For more information, visit www.npsadvantage.com
How availability will be affected by the merger with Shire is also unknown but it is assumed the merger with the bigger company (with its bigger sales force) will improve access.