NPS Pharmaceuticals ($NPSP) got a big boost this morning when the FDA issued an upbeat preview of its short bowel syndrome drug Gattex, endorsing the developer's efficacy claims and key aspects of the risk evaluation and mitigation strategy it proposed to manage a potential cancer risk, among other safety issues.
NPS shares swiftly jumped 14% on the FDA review, which precedes an FDA panel scheduled on Tuesday.
Gattex (teduglutide) is an orphan drug designed to reduce dependence among SBS patients on extended intravenous feeding following the removal of a portion of their intestines, a condition which can be triggered by Crohn's disease. The company has projected about $350 million in sales for the drug.
There are about 10,000 patients in the U.S., says the CEO, with about as many in Europe, where Takeda has licensing rights. If NPS gets an approval at the FDA, it plans to market the drug itself.
The FDA review notes that there are three key safety concerns--gastrointestinal obstructions, the growth of polyps and pancreatic disorders--but concluded that the drug's use would not need to be restricted due to safety concerns, a big plus for NPS. An expert panel will have to reach its own conclusions on efficacy next week, but the review highlighted a recent thumbs-up in Europe in support of the biotech's data.
"During the European Medicines Agency expert group meeting of the CHMP (May 8, 2012) experts concluded that reduction in parenteral fluid requirements was clinically meaningful in this population," the FDA noted. "The CHMP issued a positive opinion for granting marketing authorization on June 21, 2012, and the commission adopted this decision on Aug. 30, 2012, granting marketing authorization for 'Revestive-teduglutide' and an orphan medicinal product for human use."
"The briefing book from the FDA had no surprises," NPS CEO Francois Nader told FierceBiotech ahead of today's release. "We had quite an alignment between safety and efficacy." Of course, the panel could go in a different direction, he added, but "we've been preparing for this for the past 10 months" with mock advisory committee sessions.
Gattex is designed to boost the flow of nutrients and blood across the gut, reducing dependence on IV feeding. But there were three cancer cases which occurred in trials, two of which the CEO said were not linked to the drug while the third instance could have been prevented if the patient had been properly screened for risks and excluded from the study. The company believes the right REMS program can manage the cancer risk.
Under NPS' proposed REMS, doctors would be advised against prescribing the drug to any patient with active cancer, patients would need to be cancer-free for 5 years, all patients would have to undergo a colonoscopy within 6 months of beginning treatment and any polyps found should be removed during the procedure. A patient registry could gather long-term responses to the drug and, like most REMS programs, physicians would need to complete an education program on risks. And the company proposes to help manage risk with a direct patient support program.
"In general, the agency supports the applicant's proposed REMS elements," noted the briefing document, which went on to suggest that the education effort be beefed up--not a major roadblock at this stage of the game.
- here's the FDA review (PDF)