FDA Postpones Its Final Decision On NPS Pharmaceuticals, Inc.'s Natpara
Published: Oct 24, 2014
October 24, 2014
By Jessica Wilson, BioSpace.com Breaking News Staff
NPS Pharmaceuticals, Inc. today announced that the U.S. Food and Drug Administration (FDA) has extended the company’s Prescription Drug User Fee Act (PDUFA) action date for the company’s Natpara Biologics License Application (BLA) until Jan. 24, 2015.
As a result, Goldman Sachs analysts downgraded the company’s stock from “buy” to “neutral” this morning. NPS Pharma’s stock price was down 7.39 percent in early morning trading.
The Bedminster, N.J.-based NPS Pharma said in a statement that “the FDA extended the action date to provide time for a full review of a major amendment.” The company did not elaborate on the amendment. The FDA also requested the company submit a Risk Evaluation and Mitigation Strategy (REMS) for Natpara.
Natpara is a bioengineered replacement therapy for the endogenous parathyroid hormone (PTH) intended to treat a rare endocrine disorder called hypoparathyroidism. The body needs PTH for a variety of key physiological functions. When the level of PTH is low, the body has low levels of calcium, high levels of phosphate and cannot convert vitamin D into its active state, which prevents the absorption of oral calcium. The disease affects approximately 50,000 people in the U.S.
The existing treatments of hypoparathyroidism do not address the underlying cause of the disease, which Natpara is intended to do. Prolonged use of existing treatments could lead to irreversible calcium-phosphate deposits in the kidneys, heart or brain.
The extension of a PDUFA action date is a rare occurrence, according to Mark Senak of the blog “Eye on FDA.” He explains that, the [PDUFA] date is a target date for the FDA to make a decision about a new drug application (NDA) and whether or not the product is approved or there is need for some further work.”
The FDA would move a PDUFA date when there is a “major amendment” made to the initial submission because the FDA requests the information (a solicited amendment) or because the company or “sponsor of the compound” provides new data from a study (an unsolicited amendment). NPS Pharma did not say which type of amendment would be under consideration.
“We continue to work closely with the FDA to finalize the review of our BLA for Natpara and appreciate the effective collaboration with the agency to date,” said Francois Nader, president and chief executive officer of NPS Pharma, in the statement. “We continue to advance our commercial readiness activities and are planning for the launch of Natpara in the second quarter of 2015.”