FDA Delays Decision On NPS' Natpara BLA For Hypoparathyroidism
By Cyndi Root
NPS Pharmaceuticals made a major amendment to its Biologics License Application (BLA) for Natpara, causing the Food and Drug Administration (FDA) to delay its decision for another three months. The company announced the delay in a press release, stating that the new Prescription Drug User Fee Act (PDUFA) action date is January 24, 2015.
Francois Nader, MD, president and CEO of NPS Pharma, said, "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. We continue to advance our commercial readiness activities and are planning for the launch of Natpara in the second quarter of 2015."
FDA Action
The FDA’s original action date was October 24, 2014. However, due to NPS’ amendment, under regulations, the federal agency is entitled to extend the date in order to provide a full review of the amendment. The FDA also requested that NPS submit a Risk Evaluation and Mitigation Strategy (REMS) for Natpara. NPS states that it is finalizing the REMS and hopes to have it ready by the revised PDUFA action date. The FDA has not requested any further clinical studies. NPS does expect the FDA to require a post-approval study.
Natpara
Natpara is an endogenous parathyroid hormone (PTH). NPS bioengineered the agent for the treatment of hypoparathyroidism, a rare endocrine disorder. Currently, patients manage symptoms with calcium and vitamin D, but the treatment does not alter the course of the disease. The FDA granted Natpara Orphan Drug status, as did the European Medicines Agency (EMA) under the trade name Natpar. In September 2014, the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted 8 to 5 to recommend the drug for approval. The committee published briefing documents to support its position.
About NPS Pharma
In the U.S., NPS currently markets Gattex (teduglutide [rDNA origin]) for injection for adults with Short Bowel Syndrome (SBS) who are dependent on parenteral support. In development are teduglutide and NPSP795. Teduglutide, a recombinant analog of human glucagon-like peptide 2 (GLP-2), is in development as a treatment for pediatric Short Bowel Syndrome (SBS). NPSP795, a small molecule antagonist of the calcium-sensing receptor, is in development as a treatment for Autosomal Dominant Hypocalcemia (ADH).