Shire's $5.2 Billion Bet Pays Off as NPS Pharmaceuticals, Inc.'s Natpara Wins Approval
January 26, 2015
By Riley McDermid, BioSpace.com Breaking News Sr. Editor
Irish drugmaker Shire has finally seen some pay off from its $5.2 billion investment in hormone treatment Natpara, after the U.S. Food and Drug Administration said Friday that it has approved the drug (parathyroid hormone) to control low blood calcium levels in patients with hypoparathyroidism.
Shire said the drug will be available commercially in the second half of 2015 and added that it will be teaming up with the American produced of Natpara, NPS Pharmaceuticals, Inc. to launch the drug. The Bedminster, NJ-based NPS Pharma did not comment but Pfizer Inc. said the deal is slated to close in the first quarter of 2015.
This most commonly occurs in patients who have had their parathyroid glands removed as part of the treatment for autoimmune or congenital diseases, and can lead to numbness, tingling, muscle twitching, spasms or cramps, abnormal heart rhythm and seizures.
The seriousness of the disease led the FDA to grant Natpara Orphan Disease status in FDA in 2007 and from the EMA in 2013, a lucrative designation that includes patent protections for multiple years and provides drugmakers with financial incentives to develop drugs that will be used by a tiny fraction of the overall population.
For its part, Shire said it was gratified that the drug had finally received approval, despite carrying a boxed warning that bone cancer (osteosarcoma) has been observed in rat studies with Natpara.
"The FDA's approval of Natpara provides a new treatment option for patients with hypoparathyroidism a devastating rare disease with significant unmet need,” said Shire's Chief Executive Officer Flemming Ornskov in a statement. “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."
The FDA based its decision on data from a clinical trial of 124 participants who were randomly assigned to receive Natpara or a placebo.
“Results showed 42 percent 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,” said the FDA.
Hypoparathyroidism can do grave damage to internal organs, including kidney damage, kidney stones, development of cataracts and the calcification of soft tissues.
“People with hypoparathyroidism have limited treatment options and face challenging symptoms that can severely impact their quality of life,” said Jean-Marc Guettier, director of the Division of Metabolism and Endocrinology Products in the FDA’s Center for Drug Evaluation and Research, in a statement. “This product offers an alternative to patients whose calcium levels cannot be controlled on calcium supplementation and active forms of vitamin D.”
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