NPS Pharmaceuticals provided an update on their various orphan drugs – both approved and in development – during their report
of third quarter 2014 results.
The company’s two largest money streams are Gattex for treating patients with short bowel syndrome (third quarter net sales of $28.1 million) and royalty reviews totalling $29.1 million this quarter from (i) Amgen’s sales of Sensipar®
(cinacalcet HCl), (ii) Kyowa Hakko Kirin’s sales of Regpara®
(cinacalcet HCl), and (iii) Janssen Pharmaceuticals’ sales of Nucynta®
(tapentadol) and Nucynta®
ER (tapentadol extended-release tablets).
The company hopes to add a third revenue source early next year following the anticipated approval by the FDA of Natpara [rhPTH[1-84]] to treat patients with hypoparathyroidism (PDUFA data set for January 24, 2015).
Net sales for Gattex continue to grow and the drug becomes available in more morkets workwide.
Francois Nader, MD, president and chief executive officer of NPS Pharma said:
“We are pleased with the continued success of Gattex/Revestive, which has achieved $68 million of net sales so far this year leaving us on track to deliver more than 200% year-over-year growth.”
“We continue to identify new Short Bowel Syndrome patients and grow our prescriber base leaving us confident in the long-term outlook for Gattex. Internationally, we are also making important progress. Revestive is now officially launched in Germany and we filed for orphan drug designation in Japan.”
Gattex is approved to treat patients with short bowel syndrome, which is a rare and life altering problem that afflicts persons who have had a large part of their small intestine removed. These patients cannot absorb nutrients and fluids properly and many receive their nutrition parenterally in addition to a plethora of medications to maximize absorption with the limited intestinal tissue available. Gattex increases intestinal absorption so that these patients can be relieved of some of the parenteral infusions needed.
In October, the FDA announced
a scheduled decision on Natpara would be delayed 3 months and moved its PDUFA date from October 24, 2014 to January 24, 2015. Neither the FDA nor NPS Pharmaceuticals has stated the reason for the delay but the 8-5 vote
earlier by the FDA Advisory Committee in September plus the 3 month delay in October is of concern.
However, NPS Pharmaceuticals remains optimistic. Dr. Nader said:
“We were very gratified to receive a positive Advisory Committee vote recommending the approval of Natpara for the long-term treatment of hypoparathyroidism. We are working with the FDA to finalize our label and Risk Evaluation and Mitigation Strategy. In parallel, we are advancing a number of pre-commercial activities to prepare for the successful launch of Natpara in the second quarter of 2015.”
During the Q&A session of their webcast
, Dr Nader said they are unable to comment on the reason for the delay during this review process.
Hypoparathyroidism is a rare endocrine disorder that can arise after removal or damage to the parathyroid glands during neck surgery. The subsequent reduced parathyroid hormone levels that in turn causes hypocalcemia. An estimated 60K-80K people have hypoparathyroidism and symptoms may include paresthesias, muscle spasms, tetany, back pain, muscle twitching, throat tightness, musculoskeletal pain, anxiety, and depression. The current treatment approach is calcium and vitamin D supplements but they do not treat the underlying cause of the problem whereas Natpara does. As we recently wrote
, patients with hypoparathyroidism are displeased with current treatment options.