science

Osilodrostat

Osilodrostat is a science topic covered in the lgStudy science library. This page brings together a partial reference excerpt, illustrations, worked examples, real-world applications and a short study plan, so you can understand Osilodrostat rather than just read about it. In short: Osilodrostat, sold under the brand name Isturisa, is a medication used for the treatment of adults with Cushing's disease who either cannot undergo pituitary gland surgery or have undergone the surgery but still have the disease. Osilodrostat is an orally active (taken by mouth), nonsteroidal corticosteroid biosynthesis inhibitor which was developed by Novartis for the treatment of Cushing's syndrome and pituitary A…

Key takeaways

Osilodrostat belongs to science; place it in that map before memorising details.
Learn the definition first, then one example that makes the definition concrete.
Connect Osilodrostat to a quantity you can measure, compute or draw — that is where exam questions come from.
Reproduce the core statement of Osilodrostat from memory before moving on to harder problems.

Reference excerpt

Osilodrostat, sold under the brand name Isturisa, is a medication used for the treatment of adults with Cushing's disease who either cannot undergo pituitary gland surgery or have undergone the surgery but still have the disease. Osilodrostat is an orally active (taken by mouth), nonsteroidal corticosteroid biosynthesis inhibitor which was developed by Novartis for the treatment of Cushing's syndrome and pituitary ACTH hypersecretion (a specific subtype of Cushing's syndrome). It specifically acts as a potent and selective inhibitor of 11β-hydroxylase (CYP11B1). The most common side effects are adrenal insufficiency, headache, vomiting, nausea, fatigue, and edema (swelling caused by fluid retention). Hypocortisolism (low cortisol levels), QTc prolongation (a heart rhythm condition) and elevations in adrenal hormone precursors (inactive substance converted into a hormone) and androgens (hormone that regulates male characteristics) may also occur in people taking osilodrostat. Osilodrostat was approved for medical use in the European Union in January 2020, and for medical use in the United States in March 2020. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.

History In October 2014, an orphan designation was granted by the European Commission for osilodrostat for the treatment of Cushing's syndrome. Osilodrostat's safety and effectiveness for treating Cushing's disease among adults was evaluated in a study of 137 adult subjects (about three-quarters women) with a mean age of 41 years. The majority of subjects either had undergone pituitary surgery that did not cure Cushing's disease or were not surgical candidates. In the 24-week, single-arm, open-label period, all subjects received a starting dose of 2 milligrams (mg) of osilodrostat twice a day that could be increased every two weeks up to 30 mg twice a day. At the end of this 24-week period, about half of subjects had cortisol levels within normal limits. After this point, 71 subjects who did not need further dose increases and tolerated the drug for the last 12 weeks entered an eight-week, double-blind, randomized withdrawal study where they either received osilodrostat or a placebo (inactive treatment). At the end of this withdrawal period, 86% of subjects receiving osilodrostat maintained cortisol levels within normal limits compared to 30% of subjects taking the placebo. The US Food and Drug Administration (FDA) approved osilodrostat based on the evidence from one clinical trial (NCT02180217) of 137 subjects with Cushing's disease. The trial was conducted at 66 sites across 19 countries (United States, Argentina, Austria, Bulgaria, Canada, China, Columbia, Germany, Spain, France, Great Britain, India, Italy, Japan, Korea, Netherlands, Russia, Thailand, and Turkey). There was one trial of 48 weeks duration that assessed the benefits and side effects of osilodrostat. The trial enrolled subjects with Cushing's disease for whom pituitary gland surgery was not an option or did not work. The trial was divided in four periods. Subjects received osilodrostat two times a day in all four periods. After the first two periods (24 weeks), the benefit of osilodrostat was assessed by the percentage of subjects who had 24-hour urinary free cortisol levels within normal limits. In the third period (which lasted eight weeks), half of the subjects who had normal urinary free cortisol levels after 24 weeks of treatment continued taking osilodrostat and the other half was switched to placebo. Neither the subjects nor the healthcare providers know which treatment was given during this period. The benefit of osilodrostat was assessed on the percentage of subjects who had normal cortisol levels at the end of this period versus the subjects who received placebo. The FDA granted osilodrostat an orphan drug designation and granted the approval of Isturisa to Novartis.

Society and culture

Legal status Osilodrostat was authorized for medical use in the European Union in January 2020, and approved for medical use in the United States in March 2020.

Economics At the recommended starting dose of 2 mg, a year's supply would cost US$172,800 at 2021 prices in the United States.

Research A systematic review and meta-analysis of osilodrostat, published in 2024, found it to have efficacy and safety in normalizing serum cortisol levels in people with Cushing's Syndrome.

References

Further reading Turcu A, Smith JM, Auchus R, Rainey WE (October 2014). "Adrenal androgens and androgen precursors-definition, synthesis, regulation and physiologic actions". Compr Physiol. 4 (4): 1369–81. doi:10.1002/cphy.c140006. ISBN 9780470650714. PMC 4437668. PMID 25428847. NIHMSID: NIHMS689229.{{cite journal}}: CS1 maint: overridden setting (link) CS1 maint: periodical has ISBN (link)

External links Clinical trial number NCT02697734 for "Efficacy and Safety Evaluation of Osilodrostat in Cushing's Disease (LINC-4)" at ClinicalTrials.gov

Illustrations

Worked examples

Example 1 — a first encounter with Osilodrostat

Start with the simplest possible case. Write down what Osilodrostat claims or describes in one sentence, then invent the smallest concrete situation in which that sentence is true. In science, the smallest case is usually a single object, a single equation or a single measurement. Check that every symbol or term in your sentence has a meaning in that case.

Example 2 — changing one variable

Take the situation from Example 1 and change exactly one quantity: double it, halve it, or set it to zero. Predict what should happen to Osilodrostat before you calculate. Comparing your prediction with the result is the fastest way to find out whether you understand the idea or only the words.

Example 3 — an exam-style question

Typical questions about Osilodrostat ask you to (a) state it precisely, (b) apply it to given data, and (c) explain a limitation. Practise writing all three answers in under five minutes; the third part is what separates a full-mark answer from an average one.

Applications of Osilodrostat

In research: Osilodrostat appears in science research whenever the underlying quantities have to be modelled precisely. Papers usually cite it as a starting assumption and then explore where it breaks down.
In technology and industry: Engineering practice reuses Osilodrostat in design rules, simulations and safety margins. Knowing the idea lets you read a specification sheet and understand why the numbers look the way they do.
In the classroom: Osilodrostat is common in secondary-school and first-year university syllabi. It links to neighbouring topics 11β-Hydroxylase inhibitors, Aldosterone synthase inhibitors, Antiglucocorticoids, so understanding it makes those chapters shorter.
In everyday life: Look for Osilodrostat outside the textbook — in sport, cooking, traffic, electronics or the sky above you. An example you found yourself is remembered far longer than one you were given.

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How to study Osilodrostat in 20 minutes

Read the reference excerpt below once, without taking notes.
Close the page and write down what Osilodrostat means in your own words.
Compare your version with the excerpt and mark what you missed.
Work through the three examples above with pen and paper.
Explain Osilodrostat out loud to somebody else — or to Teacher Smith in the lgStudy chat.

Frequently asked questions

What is Osilodrostat in simple terms?

Why does Osilodrostat matter?

Because it connects several science ideas at once: it gives you a definition you can apply, a quantity you can calculate, and a way to check whether a result is plausible.

How should I study Osilodrostat?

Read the excerpt, restate it from memory, then work through the examples and applications listed on this page. The five-step study plan above takes about twenty minutes.

What does this page cover?

It gives you a compact reference excerpt plus original lgStudy explanations, examples, applications and study material on Osilodrostat.