- The FDA's Endocrinologic and Metabolic Drugs Advisory Committee has voted 8-5 to endorse the approval of Naptara (rhPTH[1-84]) for the treatment of hypoparathyroidism.
- Hypoparathyroidism is a rare endocrine disease in which a diminished concentration of parathyroid hormone in the blood cause calcium and phosphorous deficiencies.
- Naptara, which was granted orphan drug status by the FDA, has a PDUFA action date of October 24.
Naptara is a bioengineered replacement therapy that targets the underlying cause of hypoparathyroidism. Thus far, the clinical development program supporting NPS Pharma’s application consists of four efficacy and safety studies and one pharmacology study.
One of the phase III studies -- the REPLACE trial -- is a randomized, controlled, double-blind, placebo-controlled study that is also the largest clinical trial to date in patients with hypoparathyroidism. The drug is also being reviewed by the European Medicines Association in the EU and has been granted orphan drug status there as well. Based on available safety and efficacy data, the chances of approval in both markets is good.
Naptara is expected to be a major blockbuster in the orphan drug space.