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| NPS CEO Francois Nader |
NPS Pharma ($NPSP) is moving toward FDA approval with its treatment for a rare disease, as agency staff applauded the drug's efficacy ahead of a make-or-break panel vote.
The treatment, dubbed Natpara, replaces human parathyroid hormone 1-84, which is underproduced in patients with the rare endocrine disorder hypoparathyroidism. Low levels of the hormone lead to calcium deficiency and difficulty converting Vitamin D, spurring complications including paresthesia, muscle pain, bone-density issues and, in acute cases, cardiac arrhythmias, the company said.
In briefing documents posted ahead of a Friday panel discussion of Natpara, FDA reviewers noted that the injectable therapy met its primary endpoint in Phase III trials, significantly beating out placebo in weaning patients with hypoparathyroidism off of calcium and Vitamin D treatments while maintaining healthy calcium levels. On the safety side, Natpara's adverse event profile remained in line with that of the placebo group, the agency noted.
However, while the treatment led to some positive changes along several bone biomarkers, it's unclear whether those are clinically relevant, reviewers wrote, and the drug charted no meaningful improvements in bone mineral density. Furthermore, Natpara failed to beat placebo in reducing patients' urinary calcium, a secondary goal of the study.
But despite those shortcomings--and the fact that the FDA tossed out pivotal data from one trial site over "multiple and significant" violations--investors were encouraged by the staff review, sending NPS' shares up as much as 17% on Wednesday morning.
Before the agency posted the documents Wednesday, NPS CEO Francois Nader told FierceBiotech that there wasn't anything in the documents that took him by surprise. "Really, none," he noted.
Provided the company goes on to an approval, as Nader expects, NPS plans to roll out the drug itself, as it did with Gattex, an FDA-approved treatment for the rare short bowel syndrome.
"We don't believe we'll need a partner," he said, given that the company can add a small group to its 35-member Gattex sales team to roll out the treatment.
Next, Natpara will go before the FDA's Endocrinologic and Metabolic Drugs Advisory Committee, which will issue a nonbinding recommendation for or against approval. The agency is expected to hand down a final decision on the orphan drug by Oct. 24.
Hypoparathyroidism affects nearly 60,000 U.S. patients, according to the National Center for Biotechnology Information, and there are no approved treatments for the root cause of the disease.
- read the briefing (PDF)