http://www.raredr.com/news/emdac-panel-8-5-favor-natpara-approval
EMDAC Panel Votes 8-5 in Favor of Natpara Approval

James Radke


On Friday Sept 12, 2014, the Endocrinologic and Metabolic Drugs Advisory Committee voted 8 – 5 in favor of the U.S. Food and Drug Administration (FDA) approving Natpara (rhPTH [1-84]) for the treatment of hypoparathyroidism. A PDUFA data is scheduled for October 24, 2014.

In a press release, the developers of Natpara, NPS Pharmaceuticals, stated, “We are very pleased with the Committee’s vote. Their recommendation reinforces our belief in the favorable benefit-risk profile of Natpara and its potential as a long-term treatment option for Hypoparathyroidism”

Francois Nader, MD, president and chief executive officer of NPS Pharmaceuticals said, “We are very pleased with the Committee’s vote. Their recommendation reinforces our belief in the favorable benefit-risk profile of Natpara and its potential as a long-term treatment option for Hypoparathyroidism,” adding,  “As a bioengineered replacement therapy targeting the underlying cause of the disorder, Natpara has been shown in clinical studies to maintain serum calcium while demonstrating similar physiologic effects to the native parathyroid hormone. We look forward to working with the FDA to complete the final stages of review of our BLA for Natpara.”

The Committee reviewed data submitted by NPS for FDA approval, including its pivotal phase 3 randomized, double-blind, placebo controlled study and the largest clinical trial conducted to date in Hypoparathyroidism.

During a conference call late Friday, Dr Nader noted that in anticipation of gaining approval, they have 38 contracted sales professionals to reach approximately 3500 endocrinologists who they think will be the primary prescribers.  Educational initiatives are also underway to raise awareness of hypoparathyroidism to both patients and clinicians. NPS also plan to have a patient support program in place once the drug is approved and distributed via specialty pharmacies.
 
Dr Nader also noted that once they gain approval, they plan to have the drug available a few weeks following approval but the official launch will not be until early 2015.

Hypoparathyroidism

Hypoparthyroidism is a rare endocrine disorder that can arise after removal or damage to the parathyroid glands during neck surgery. The subsequent reduced parathyroid hormone levels that in turn causes hypocalcemia. An estimated 60K-80K people have hypoparathyroidism and symptoms may include paresthesias, muscle spasmbs, tetany, back pain, muscle twitching, throat tightness, musculoskeletal pain, anxiety, and depression. The current treatment approach is calcium and vitamin D supplements but they do not treat the underlying cause of the problem whereas Natpara does. As we wrote recently, patients with hypoparathyroidism tend to be displeased with current treatment options.
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