- Advisers to the Food and Drug Administration on Wednesday delivered a split verdict on GSK’s experimental anemia pill, backing its use only certain patients with chronic kidney disease.
- The panel voted 13-3 that the benefits of the drug, called daprodustat, outweigh its risks for kidney disease patients who require dialysis. For the larger population of patients who aren’t on dialysis, the committee voted against the medicine by an 11-5 vote, citing safety concerns.
- Though not bound by the committee’s decision, the FDA generally follows the advice of its expert panels. The agency’s target date to complete its review of the GSK drug is Feb. 1.
The partial recommendation may be enough for GSK to sell the first anemia pill in the U.S. for chronic kidney disease patients, even if it’s for a smaller market than the British drugmaker had sought. In a little more than a year, the FDA has rejected two other anemia pills, developed by FibroGen and Akebia Therapeutics, because of concerns about heart-related side effects.
Given the earlier rejections, the mixed result from the FDA committee “is probably better than widely anticipated,” Jefferies analyst Peter Welford wrote in a note to investors. He estimates the drug could have peak sales of $1 billion, largely based on its use in dialysis patients alone.
Two large studies found daprodustat had similar benefits as approved injectable drugs for anemia, a condition in which patients don’t have enough red blood cells to carry oxygen to tissues.
In patients on dialysis, the experimental medicine did not “unacceptably increase the risk” of heart complications when compared with injectable anemia drugs, the FDA said in briefing documents ahead of the committee meeting. But in patients who don’t depend on dialysis, “several other important risks” emerged in the research, the FDA said.
While the FDA acknowledged the added convenience of a pill, the agency noted that most patients on dialysis receive injectable medications for anemia while they are getting dialysis, making the advantage of an oral treatment unclear. And for patients not on dialysis, there’s an added risk of less monitoring for side effects, which might lead to more problems, the FDA said.
Committee members voiced similar hesitations and indicated they’d like to see more data.
“I think there’s real potential for the future of this oral medication in this population, but not with the data presented today,” said Christopher O’Connor, a cardiologist and executive director of the Inova Heart and Vascular Institute in Virginia, after voting “no” on the drug’s use in non-dialysis patients. He supported its use in dialysis patients.
Jacqueline Alikhaani, a consumer representative and advocate with the American Heart Association, said she was “really enthusiastic” about the possibility of more convenient treatments for kidney patients, but didn’t believe there was enough evidence to recommend approval of the drug in either group yet.
“Safety is just paramount,” she said. “We need more long-term outcome data.”
Daprodustat is part of a new class of drugs called hypoxia-inducible factor prolyl hydroxylase inhibitors. The drugs are designed to raise levels of red blood cells by tricking the body into reacting as if it’s in a high-altitude environment and needs more oxygen.
The medicines are approved in other countries around the world, but the FDA has been hesitant to clear one for the U.S. market. Daprodustat is already sold in Japan as Duvroq and is also under review in Europe. GSK in the past has estimated the drug could bring in peak sales of as much as 1 billion pounds, or about $1.2 billion.