chemistry

Givinostat

Givinostat 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 Givinostat rather than just read about it. In short: Givinostat, sold under the brand name Duvyzat is a medication used for the treatment of Duchenne muscular dystrophy. It is a histone deacetylase inhibitor with potential anti-inflammatory, anti-angiogenic, and antineoplastic activities.

Key takeaways

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

Reference excerpt

Givinostat, sold under the brand name Duvyzat is a medication used for the treatment of Duchenne muscular dystrophy. It is a histone deacetylase inhibitor with potential anti-inflammatory, anti-angiogenic, and antineoplastic activities. It works by targeting pathogenic processes to reduce inflammation and loss of muscle. The most common side effects include diarrhea, abdominal pain, low platelets (thrombocytopenia), nausea and vomiting, an increase in triglycerides (a type of fat in the body) (hypertriglyceridemia), and fever. Givinostat was approved for medical use in the United States in March 2024. Givinostat is the first nonsteroidal medication approved by the US Food and Drug Administration (FDA) to treat people with all genetic variants of Duchenne muscular dystrophy. The FDA considers it to be a first-in-class medication.

Medical uses Givinostat is indicated for the treatment of Duchenne muscular dystrophy (DMD) in people six years of age and older.

Available forms Givinostat is available in the form of an oral suspension.

Adverse effects In clinical trials of givinostat as a salvage therapy for advanced Hodgkin's lymphoma, the most common adverse reactions were fatigue (seen in 50% of participants), mild diarrhea or abdominal pain (40% of participants), moderate thrombocytopenia (decreased platelet counts, seen in one third of patients), and mild leukopenia (a decrease in white blood cell levels, seen in 30% of patients). One-fifth of patients experienced prolongation of the QT interval, a measure of electrical conduction in the heart, severe enough to warrant temporary suspension of treatment.

Pharmacology

Mechanism of action

Givinostat is a potent pan-histone deacetylase (HDAC) inhibitor. It non-selectively inhibits all 11 of the HDAC enzymes, with several-fold variation in inhibitory potency across them. The drug inhibits several pro-inflammatory cytokines, including reducing expression of tumour necrosis factor (TNF), interleukin-1α (IL-1α) and 1β (IL-1β), and interleukin-6 (IL-6) in preclinical research and in humans. It also has activity against cells expressing JAK2(V617F), a mutated form of the janus kinase 2 (JAK2) enzyme that is implicated in the pathophysiology of many myeloproliferative diseases, including polycythaemia vera. In patients with polycythemia, the reduction of mutant JAK2 concentrations by givinostat is believed to slow down the abnormal growth of erythrocytes and ameliorate the symptoms of the disease.

Pharmacokinetics The bioavailability of givinostat is unknown. The time to peak levels of givinostat is 2 to 3 hours taken orally. Givinostat efficiently crosses into the brain in rodents. It is described as unique in this regard, as many other histone deacetylase inhibitors poorly penetrate into the brain. However, in another study, givinostat was reported to have poor brain penetrance and only subtle brain HDAC inhibition in rodents. The plasma protein binding of givinostat is 96%. It is extensively metabolism, but cytochrome P450 (CYP450) and glucuronosyltransferase (UGTs) enzymes are not majorly involved. The elimination half-life of givinostat is approximately 6 hours. It is mainly eliminated via metabolism and is excreted in urine less than 3% unchanged.

History Givinostat was discovered at Italfarmaco of Milan, Italy. It was patented in 1997 and first described in the scientific literature in 2005. The efficacy of givinostat for the treatment of Duchenne muscular dystrophy was evaluated in a randomized, double-blind, placebo-controlled 18-month phase III study. The primary endpoint was the change from baseline to month 18 using a four stair climb to measure muscle function. All participants continued to receive a standard of care steroid regimen throughout the study and, after 18 months of treatment, participants treated with givinostat showed statistically significant less decline in the time it took to climb four stairs compared to placebo. The mean change from baseline to month 18 in time to climb four stairs was 1.25 seconds for participants receiving givinostat compared to 3.03 seconds for participants receiving placebo. A secondary efficacy endpoint was the change from baseline to month 18 in physical function as assessed by the North Star Ambulatory Assessment (NSAA)—a scale commonly used to rate the motor function in boys with Duchenne muscular dystrophy who are capable of walking. Compared to placebo, participants treated with givinostat saw less worsening in their NSAA score after 18 months. The US Food and Drug Administration (FDA) granted the application for givinostat priority review, fast track, orphan drug, and rare pediatric disease designations. The FDA granted the approval of Duvyzat to Italfarmaco S.p.A. The FDA approved givinostat based on evidence from a single clinical trial (NCT02851797) of 179 males with Duchenne muscular dystrophy who were six years of age and older who could walk and were on stable background therapy with steroids. The trial was conducted at 45 sites in 11 countries in North America and Europe. Twenty-eight of the participants were from the United States.

Society and culture

Legal status In April 2025, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a conditional marketing authorization for the medicinal product Duvyzat, intended for the treatment of Duchenne muscular dystrophy (DMD). The applicant for this medicinal product is Italfarmaco S.p.A. The CHMP's opinion is based on data from a subgroup of 120 participants (79 treated with givinostat and 41 with placebo) in a randomized, placebo-controlled study in ambulant participants with Duchenne muscular dystrophy aged six years of age or older on concomitant steroid treatment. The safety profile of Duvyzat is based on data from 179 participants. The most common events in participants treated with givinostat were diarrhoea, abdominal pain, thrombocytopenia (low levels of blood platelets), vomiting, hypertriglyceridaemia (high blood levels of triglycerides, a type of fat) and fever. Givinostat was authorized for medical use in the European Union in June 2025.

Names Givinostat is the generic name of the drug and its International Nonproprietary Name (INN).

… excerpt ends here. Continue reading the full article.

Worked examples

Example 1 — a first encounter with Givinostat

Start with the simplest possible case. Write down what Givinostat 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 Givinostat 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 Givinostat 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 Givinostat

In research: Givinostat 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 Givinostat 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: Givinostat is common in secondary-school and first-year university syllabi. It links to neighbouring topics Anti-inflammatory agents, Diethylamino compounds, Histone deacetylase inhibitors, so understanding it makes those chapters shorter.
In everyday life: Look for Givinostat 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.

Ask Teacher Smith questions about this articleOpens your AI tutor with a question about “Givinostat” →

Affiliate

Preply — study more efficiently by working with a personal tutor. 50% off.

How to study Givinostat in 20 minutes

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

Frequently asked questions

What is Givinostat in simple terms?

Why does Givinostat 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 Givinostat?

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 Givinostat.