Authored by Karl Simpson
In recent weeks, Liftstream has featured a range of articles in the rare disease and orphan drug area. One of the companies particularly catching our eye right now is NPS Pharmaceuticals, led by CEO Dr. Francois Nader.
Biotechnology companies remain the heartland of orphan drug development, each aiming to bring particular therapies to address the estimated 6000+ rare diseases. NPS Pharmaceuticals is such a company, researching and developing therapeutics for orphan diseases in gastroenterology and endocrinology indications. The company is approaching the launch of its first products, Gattex and Natpara, although presently generates its revenues from royalties associated with Sensipar which continues on its double-digit growth path. The company presently has about $119m cash which it foresees as adequate to fund the commercial launch of Gattex.
Gattex (teduglutide) is a product that is indicated for short bowel syndrome (SBS). The drug was recently reviewed by the FDA and they voted in favour of FDA approval. Teduglutide is a recombinant glucagon-like peptide- (GLP-2) analog which acts as a growth hormone specific to intestines. Short bowel syndrome is a very rare disease and as with many rare diseases, it is difficult to forecast the exact population of patients. For SBS, a high end calculation of 10,000 to 15,000 patients in the US market has been projected. Gattex is actually approved in the EU (Sept. 2012) too under the name Revestive and NPS Pharmaceuticals licensed the product to Takeda, who will lead the commercialisation in the European region. The commercial launch of Gattex/Revestive is tabled for early 2013 and the company is highly confident in its commercial, pricing & reimbursement and market access strategy. It will earn royalties from Takeda on the sale of the product as they ramp it up in the market in Europe. A price has so far not been set.
Natpara is another product NPS has in late stage clinical development. The product is a recombinant human parathyroid hormone for the treatment of hypoparathyroidism. Essentially, the product would offer a hormone replacement therapy for patients. The company says it is on track for a BLA to be filed in the first half of next year, which would bring it quickly on the heels of Gattex. NPS have already engaged in pre-launch activities and market access research the company has conducted suggests ‘encouraging’ pricing for Natpara.
With Gattex, the company seem to be pursuing an ultra-orphan pricing and market access strategy and payers seemed to perceive the product’s price value proposition in line with other ultra-orphan analogs. The company conducted a robust market access and pricing study.
It is often the case though with ultra-orphan drugs that the market size is not as big as the projected number, however, NPS seem confident of materialising upwards of $350m sales from the product in the year. Equally though, for any marketing professional experienced in rare diseases, you will know that most orphan indicated products take a while to get out in the market to their full potential.
In summary though, with a strong European partner in Takeda and a clear commercialisation strategy in the US, NPS looks very much like a company about to reap the rewards of its endeavours and so at Liftstream, this is one we’ll be watching.