Authored by James Sheppard
US rare disease specialist NPS Pharmaceuticals announced that it has submitted a Biologic License Application (BLA) to the US FDA for Natpara. The product is a bioengineered replacement for endogenous parathyroid hormone (PTH) which is indicated for the treatment of Hypoparathyroidism, a rare endrocrine disorder where the body produces insufficient levels of PTH, a principal regulatory of mineral homoeostasis in the body.
Natpara was granted orphan drug status by the FDA in 2011 and recently completed phase III clinical trials, which will form the basis of the BLA application to the FDA. The results of the phase III trial, which enrolled 134 patients, were extremely positive. The results showed that 53% of patients hit their primary endpoint, in contrast only 2% of the placebo group hit the endpoint.
Francois Nader, president and chief executive officer of NPS Pharmaceuticals said, ‘Submitting our BLA for Natpara is a significant step forward for NPS in our effort to bring the first replacement therapy to patients with hypoparathyroidism. Patients face a significant burden of disease given the multitude of physical, cognitive, and emotional symptoms associated with this disorder.’
Currently there are no FDA approved replacement therapies for Hypoparathyroidism, which is currently managed by large dose calcium supplements. The submission follows the news that NPS had enrolled its first patient in their new global registry for hypoparathyroidism, where patients will be tracked for a minimum of 10 years.
NPS Pharma has received many plaudits from the rare disease world for their advancing treatments. Investors have also been reaping the rewards of NPS’s efforts with the share price increasing greatly over the past months as investors have poured in to the biotech sector and orphan drugs in particular. Along with Natpara, NPS also has been busy commercialising Gattex for the treatment of short bowl syndrome which received marketing approval in the USA and in the EU under the name Revestive