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osilodrostat(Isturisa)治疗库欣综合征的疗程及治疗费用?

Author: Medicalhalo
Release time: 2025-10-19 11:44:20

Since each patient's condition, personal constitution, and response to drug treatment are different, the specific treatment course and cost of Osilodrostat (Isturisa) also vary.

osilodrostat (Isturisa) drug introduction

It is an oral small molecule 11β-hydroxylase inhibitor developed by Novartis for the treatment of Cushing's disease. Osilodrostat (Isturisa) is approved in the European Union for the treatment of endogenous Cushing's syndrome in adults based on results from a pivotal Phase III trial

osilodrostat (Isturisa) treatment regimen

osilodrostat (Isturisa) is a treatment for Cushing's syndrome. The length and number of sessions will vary based on the patient's specific condition and will usually require continued use over a period of time.

When using osilodrostat (Isturisa) to treat Cushing's syndrome, it is recommended to follow your doctor's guidance and recommendations. Doctors will develop an appropriate treatment plan based on the patient's clinical symptoms, laboratory test results, and individualized treatment needs.

Typically, treatment with osilodrostat (Isturisa) takes several months to several years. In the early stages of treatment, the dose needs to be gradually increased based on the patient's medication response until an effective therapeutic dose is reached. The patient will then continue this for a period of time to control symptoms of Cushing's syndrome and monitor relevant laboratory indicators.

osilodrostat (Isturisa) listing information

Currently, osilodrostat (Isturisa) was launched in the United States, France, Japan, and Germany on March 6, 2020, January 9, 2020, June 2021, and January 13, 2020. However, as of December 5, 2023, it has not yet been launched in China.

The treatment cost of osilodrostat (Isturisa)

Recordati Company announced that osilodrostat (Isturisa) can treat patients with Cushing's disease who cannot undergo pituitary surgery or cannot be cured after surgery. The price of 10mgx60 tablets is about $67,500 per box.

A bottle of osilodrostat (Isturisa) contains 60 tablets, which is 600 mg. Patients need to take 2 mg orally twice a day, which is 4 mg a day. Therefore, one bottle of the drug can be taken for about 150 days, which is 5 months.

Therefore, patients can calculate the specific treatment cost based on the number of days of their medication course.

Therapeutic efficacy of osilodrostat (Isturisa)

Background: Prospective studies have demonstrated the efficacy of osilodrostat (Isturisa) in Cushing's disease. There are no studies evaluating osilodrostat (Isturisa) in a range of patients with paraneoplastic Cushing's syndrome/ectopic adrenocorticotropin syndrome (PNCS/EAS).

Purpose: This study aims to evaluate the actual efficacy and safety of osilodrostat (Isturisa) in patients with PNCS/EAS in France.

Methods: In this retrospective, multicenter, real-world study, a total of 33 patients with PNCS/EAS and intense/severe hypercortisolism participated. Patients received osilodrostat (Isturisa) between May 2019 and March 2022 at a median initial dose (range) of 4 mg/day (1-60) and a maximum dose of 20 mg/day (4-100), first on a patient-by-patient basis, then on a cohort-by-cohort basis for interim authorization, and after marketing authorization. The protocols were titration (n=6), block and replace (n=16), or titration followed by block and replace (n=11).

Results: Among 11 patients who received osilodrostat (Isturisa) as first-line monotherapy, median 24-hour urinary free cortisol (24h-UFC) decreased significantly (from 26 times the upper limit of normal [ULN; 2.9-659] to 0.11 × ULN [0.08-14.9]). Nine of these cases achieved 24-hour UFC normalization within 2 weeks (median). An additional 13 patients had previously been treated with classical steroidogenesis inhibitors, but 10 of these 13 patients were not controlled.

In these patients, second-line osilodrostat (Isturisa) monotherapy significantly reduced 24-hour UFC (from 2.6 × ULN [1.1-144] to 0.22 × ULN [0.12-0.66]). An additional nine patients received osilodrostat (Isturisa) in combination with another anticorticoid, which reduced 24-hour UFC from 11.8 × ULN (0.3-247) to 0.43 × ULN (0.33-2.4).

At the same time, as blood pressure, hyperglycemia, and hypokalemia improved, major clinical symptoms/comorbidities improved significantly, allowing patients to discontinue treatment or reduce dosage. Adrenal insufficiency (grade 3-4) was reported in 8 of 33 patients.

Conclusion: osilodrostat (Isturisa) is a rapidly effective drug for the treatment of PNCS/EAS with severe hypercortisolism. osilodrostat (Isturisa) is generally well tolerated, with adrenal insufficiency being the main side effect.

Summary

During the period of using osilodrostat (Isturisa), it is recommended that patients follow the doctor's advice for regular check-ups. Under the guidance of a doctor, the patient can evaluate whether the dose needs to be adjusted or continued treatment based on the treatment effects and adverse reactions. The specific course of treatment should be determined according to the patient's condition to ensure the effectiveness of drug treatment.

References

Dormoy A, Haissaguerre M, Vitellius G, Do Cao

C, Geslot A, Drui D, Lasolle H, Vieira-Pinto O, Salenave S, François M,

Puerto M, Boullay HD, Mayer A, Rod A, Laurent C, Chanson P, Reznik Y,

Castinetti F, Chabre O, Baudin E, Raverot G, Tabarin A, Young J.

Efficacy and Safety of Osilodrostat in Paraneoplastic Cushing Syndrome: A

Real-World Multicenter Study in France. J Clin Endocrinol Metab. 2023

May 17;108(6):1475-1487. doi: 10.1210/clinem/dgac691. PMID: 36470583;

PMCID: PMC10188310.

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