FDA Approves First-Ever Oral Anemia Drug, From GSK

The United States Food and Drug Administration issued a landmark approval for a new anemia drug this week. Made by GlaxoSmithKline LLC (GSK), the new, first-of-its-kind oral treatment aims to specifically treat the kind of anemia resulting from chronic kidney disease (CKD) in adults on dialysis. The drug is only intended for these patients who are presently on dialysis, as this is the only test group for which the drug’s safety has been verified.

Under the brand name Jesduvroq, the drug is known clinically as daprodustat and it is the first treatment to be issued orally. Other FDA-approved CKD treatments (for adults on dialysis) must be injected, either under the skin or directly into the bloodstream.

Jesduvroq, to put it plainly, increases erythropoietin hormone levels in the body. CKD patients on dialysis have low erythropoietin levels because the condition—coupled with the treatment—reduces the kidney’s efficiency, resulting in lower hormone levels. This can lead to anemia, resulting in a need for this type of medication.

As with many medications, Jesduvroq has a warning label that includes potential side effects and other risks. For example, taking the drug can increase the risk of stroke, heart attack, death, and blood clots in the legs, lungs, or dialysis access site. Other potential warnings include heart failure risk resulting in hospitalization, stomach erosions, gastrointestinal bleeding, and the exacerbation of pre-existing blood pressure issues. Less-severe side effects include dizziness and abdominal pain.

The treatment could greatly benefit any of the 500,000 adults in the US who suffer from CKD that requires dialysis. However, CKD affects roughly 39 million people in the US; of that population, approximately 6 million have anemia. From that population, about 810,000 have CDK with end-stage renal disease, and within that cohort, it is estimated about 558,000 patients are on treatment with dialysis (hence the number mentioned above).

While the group that most benefit from this drug represents only about 1.3% of those with CKD, the treatment has much bigger implications. For one, the FDA previously rejected two separate oral anemia drugs, so this one’s approval is more meaningful and gains momentum for more innovation. Secondly, an estimated 1 in 7 patients with CKD could, at some point, develop anemia and eventually need more options like this. And finally, of course, it could be a profit windfall for the company (and shareholders), though these figures are not yet available.