chemistry

Pexidartinib

Pexidartinib is a chemistry 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 Pexidartinib rather than just read about it. In short: Pexidartinib, sold under the brand name Turalio, is a kinase inhibitor drug for the treatment of adults with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery. Pexidartinib blocks the activity of the colony-stimulating factor-1 receptor (CSF-1R).

Key takeaways

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

Reference excerpt

Pexidartinib, sold under the brand name Turalio, is a kinase inhibitor drug for the treatment of adults with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery. Pexidartinib blocks the activity of the colony-stimulating factor-1 receptor (CSF-1R). Common side effects are increased lactate dehydrogenase (proteins that helps produce energy in the body), increased aspartate aminotransferase (enzymes that are mostly in the liver but also in muscles), loss of hair color, increased alanine aminotransferase (enzymes that are primarily in the liver and kidney) and increased cholesterol. Additional side effects include neutropenia (low level of white blood cells that help the immune system defend against disease and infection), increased alkaline phosphatase (enzymes that are mostly in the cells of bone and the liver), decreased lymphocytes (white blood cells that help the immune system defend against disease and infection), eye edema (swelling around the eyes), decreased hemoglobin (protein in red blood cells that carry oxygen), rash, dysgeusia (altered sense of taste) and decreased phosphate (electrolytes that help with energy). The US prescribing information for pexidartinib includes a boxed warning about the risk of serious and potentially fatal liver injury. In August 2019, it was approved by U.S. FDA for treatment of giant-cell tumor of the tendon sheath (GC-TS). Pexidartinib is available in the US only through the Turalio Risk Evaluation and Mitigation Strategy (REMS) Program. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.

History The approval of pexidartinib was based on the results of a multi-center international clinical trial of 120 subjects, 59 of whom received placebo. The primary efficacy endpoint was the overall response rate (ORR) analyzed after 25 weeks of treatment. The clinical trial demonstrated a statistically significant improvement in ORR in subjects who received pexidartinib, with an ORR of 38%, compared to no responses in subjects who received placebo. The complete response rate was 15% and the partial response rate was 23%. A total of 22 out of 23 responders who had been followed for a minimum of six months following the initial response maintained their response for six or more months, and a total of 13 out of 13 responders who had been followed for a minimum of 12 months following the initial response maintained their response for 12 or more months. The U.S. Food and Drug Administration (FDA) granted the application for pexidartinib breakthrough therapy designation, orphan drug designation, and priority review designation. The FDA granted the approval of Turalio to Daiichi Sankyo.

References

Further reading "Pexidartinib". LiverTox: Clinical and Research Information on Drug-Induced Liver Injury. Bethesda, MD: National Institute of Diabetes and Digestive and Kidney Diseases. October 2019. PMID 31869194. NBK551730. Lamb YN (November 2019). "Pexidartinib: First Approval". Drugs. 79 (16): 1805–1812. doi:10.1007/s40265-019-01210-0. PMC 7044138. PMID 31602563. Roskoski R (February 2020). "Properties of FDA-approved small molecule protein kinase inhibitors: A 2020 update". Pharmacol. Res. 152 104609. doi:10.1016/j.phrs.2019.104609. PMID 31862477. S2CID 209435035.

External links "Pexidartinib". Drug Information Portal. U.S. National Library of Medicine. Archived from the original on March 18, 2020.

Illustrations

Worked examples

Example 1 — a first encounter with Pexidartinib

Start with the simplest possible case. Write down what Pexidartinib claims or describes in one sentence, then invent the smallest concrete situation in which that sentence is true. In chemistry, 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 Pexidartinib 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 Pexidartinib 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 Pexidartinib

In research: Pexidartinib appears in chemistry 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 Pexidartinib 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: Pexidartinib is common in secondary-school and first-year university syllabi. It links to neighbouring topics CYP3A4 inducers, Chloroarenes, Disubstituted pyridines, so understanding it makes those chapters shorter.
In everyday life: Look for Pexidartinib 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 Pexidartinib in 20 minutes

Read the reference excerpt below once, without taking notes.
Close the page and write down what Pexidartinib 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 Pexidartinib out loud to somebody else — or to Teacher Smith in the lgStudy chat.

Frequently asked questions

What is Pexidartinib in simple terms?

Why does Pexidartinib matter?

Because it connects several chemistry 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 Pexidartinib?

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 Pexidartinib.