|NPS CEO Francois Nader|
NPS Pharmaceuticals ($NPSP) cleared a hurdle in pursuit of FDA approval of Gattex. On Tuesday afternoon a panel of FDA advisers voted that the drug's benefits outweigh the potential harms and backed a plan to reduce potential cancer risks and other safety issues facing people on the therapy. If all goes as planned for NPS, the FDA will heed the advice of its panel of non-agency experts and stamp an approval on the drug before the end of the year.
As TheStreet's Adam Feuerstein reported on his live blog of the meeting, the FDA's Gastrointestinal Drugs Advisory Committee endorsed approval of NPS' treatment with a 12-0 vote that the benefits of NPS' compound, known as teduglutide, outweigh the risks to patients for the treatment of a relatively rare condition known as short bowel syndrome. And the panel voted 10 in favor with one against and one abstention on the question of whether the company's proposed risk evaluation and mitigation strategy (REMS) was sufficient.
NPS CEO Francois Nader has seen his wish come true in 2012 for the Bedminster, NJ-based company to transition from a development-stage biotech to one with an approved product. In August the European Commission took a recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) and green-lighted Gattex.
The GLP-2 analog is approved in the European Union, where Takeda has marketing rights, for treating adults with short bowel syndrome, a condition that results from the removal of a portion of their intestines because of Crohn's disease or other medical problems. Gattex, which mimics the activity of a hormone involved in restoration of the intestinal lining, is supposed to reduce those patients' dependence on IV feeding.
"It is a huge, transformative year for us, from a development company to a commercial company," Nader told FierceBiotech in January.
NPS could reap the most financial upside from the marketing of Gattex in the U.S., where the company controls full marketing rights to the injected therapy as it does in the rest of North America. The company estimates annual sales of Gattex could reach $350 million.
First, of course, the company needs FDA approval of Gattex. There are no guarantees that the agency will follow the advice of its advisory committee, but the regulator often does. And last week the agency published a staff review that seemed to raise no new red flags about NPS' application for approval. The review appeared to support the company's REMS proposal, which included restricting prescriptions of the therapy to patients with active cancer, after three cases of cancer were observed in patients on the treatment in clinical trials.
NPS plans to market Gattex on its own in the U.S., and the FDA has set an action date of Dec. 30 for its decision on whether to grant approval.
- here's the company's release