NPS Pharmaceuticals, Inc., a biopharmaceutical company developing innovative therapeutics for rare gastrointestinal and endocrine disorders, is panning to submit its Biologic License Application (BLA) for Natpara (recombinant human parathyroid hormone (rhPTH [1-84])) in mid-2013.
The change in the company's forecasted timeline is due to a request from the Medical Device Division of the US Food and Drug Administration (FDA) to modify the company's instructions for using the injection pen device to deliver Natpara. The FDA has not requested any new clinical data or clinical studies of Natpara. The requested changes will require the company to repeat its Usability Testing of the injection pen device using the modified instructions before submitting its BLA.
"I have every confidence that we will complete the requested testing of the injection pen instructions and deliver our BLA submission in line with our mid-2013 guidance," said Francois Nader, MD, president and chief executive officer of NPS Pharmaceuticals. "We are continuing to prepare the remaining components of the Natpara BLA to ensure that we assemble a high-quality submission. In addition, we are making good progress toward resolving the previously disclosed manufacturing issue and we fully believe this matter will be resolved well in advance of our BLA submission."
Natpara has been classified by the FDA as a biologic-device combination. Usability Testing is used to show that a medical device, such as the injection pen used to deliver Natpara, is reasonably safe and effective when used by the intended user population. It involves conducting a simulated use test, in which prospective users operate the device in a meaningful way, in a realistic, but simulated environment.
Hypoparathyroidism is a rare endocrine disorder in which the body produces insufficient levels of parathyroid hormone, the principal regulator of calcium and phosphorus. When the body has too little parathyroid hormone, blood calcium levels drop and phosphorus levels increase, which can cause a number of physical and mental symptoms, including uncontrollable muscle spasms and cramps, tetany, seizures, fatigue, anxiety, and depression. It is the only classic endocrine disorder for which there are no FDA-approved replacement therapies. Current treatment approaches focus on symptom management through high doses of calcium and active vitamin D supplementation, which can lead to serious side effects and long-term consequences. Over time, calcium may build up in the body and result in serious health risks, including calcifications in the kidneys, heart or brain.