Does Pasireotide work?
Yes, in large controlled trials. The main trial had 162 people.
Approval for Cushing's disease rests on a randomized, double-blind phase 3 trial in 162 patients that compared two doses, with normalization of urinary cortisol as the primary endpoint. The trial lacked a placebo arm, which is common in Cushing's because untreated disease is dangerous, and response rates were modest. It is the largest prospective drug trial in the disease. 12
The phase 3 trial randomized 162 adults with persistent, recurrent or newly diagnosed Cushing's disease. None could have surgery. They got one of two doses of pasireotide twice a day, double-blind, for six months. Open-label treatment followed up to twelve months. The main measure was normal 24-hour urinary free cortisol at month 6 without a dose increase. About 15 percent of the lower-dose group met it. In the higher-dose group it was 26 percent. Cortisol fell in most patients even when it did not normalize. Blood pressure, weight, cholesterol and quality of life improved in step. Patients whose cortisol was going to normalize generally showed a drop within the first two months. The authors suggested this could identify non-responders early. 1
In acromegaly, researchers compared the long-acting form with octreotide LAR. It produced biochemical control in a higher share of patients. That is the basis of the approved use as described in the label. 2
Evidence checklist
- Randomised controlled trial Randomised double-blind, two doses, no placebo
- Human study over 100 people Phase 3, 162 patients
- More than one research group Article does not say who ran the trial
- Clinical outcomes, not surrogate markers Surrogate: normal urinary cortisol
- Human safety data beyond case reports Trial data: 73% had high blood sugar events
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More on Pasireotide
Citations
- Colao 2012, N Engl J Med: a 12-month phase 3 study of pasireotide in Cushing's disease doi.org
- Recordati Rare Diseases: Signifor (pasireotide) prescribing information recordatirarediseases.com
Reference information only, not medical advice. Drawn from the Pasireotide article, fact-checked against the cited sources. No dosing anywhere on this site.