Duvyzat (givinostat)
An approved treatment for Duchenne Muscular Dystrophy.
The same compound appears under different names depending on the context. Here is how to identify Givinostat wherever you encounter it, plus the key facts at a glance.
- Generic name
- Givinostat
- Brand name
- Duvyzat
- Development code
- ITF2357
- Drug class
- HDAC inhibitor
- Manufacturer
- Italfarmaco
- How it's taken
- Taken orally as a flavored liquid suspension twice daily with food.
The first non-steroid systemic treatment for DMD that works regardless of the patient's specific dystrophin mutation. Duvyzat targets the downstream muscle damage (inflammation and fibrosis) rather than the genetic defect itself.
We'll email you when Duvyzat's FDA label changes, when the FDA acts on it, and when new trials for Duchenne Muscular Dystrophy open. Unsubscribe anytime.
Where Givinostat fits
First non-exon-skipping, non-corticosteroid disease-modifying therapy for DMD. Works by reducing inflammation and fibrosis in muscle tissue, applicable to all DMD patients regardless of mutation.
How Givinostat works
In DMD, the lack of dystrophin causes ongoing muscle damage, which triggers chronic inflammation and replacement of muscle with scar tissue (fibrosis). Givinostat inhibits histone deacetylase enzymes, which reduces the inflammatory signals and slows down the fibrotic process. By preserving muscle quality, it helps maintain strength and function even though the underlying genetic defect remains.
Mechanism: Histone deacetylase (HDAC) inhibitor that reduces inflammation and fibrosis in dystrophic muscle
Side effects and safety
The most common side effects are diarrhea, stomach pain, low platelet counts (thrombocytopenia), nausea or vomiting, high triglycerides and fever. Low platelets happened in 33% of treated boys in the main trial and can cause bleeding such as nosebleeds or bruising. It can also lower red and white blood cell counts and can prolong the QTc interval on an ECG, so it is avoided in people at higher risk of abnormal heart rhythms. Blood counts are checked every 2 weeks for the first 2 months, at month 3 and then every 3 months, and triglycerides at months 1, 3 and 6 and then every 6 months.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Givinostat
Taken orally as a flavored liquid suspension twice daily with food. Dose is weight-based. Approved for people with DMD aged 6 and older. It should not be started if the platelet count is below 150 x 10^9/L (150,000 per microliter). The label does not limit use to people who can still walk, although the main trial enrolled boys who could walk. Can be used alongside corticosteroids and exon-skipping therapies.
Availability and cost
Only available as the brand-name product.
First-in-class histone deacetylase inhibitor for DMD, targeting muscle inflammation and fibrosis. Orphan drug for a rare progressive muscular dystrophy.
Help paying for Duvyzat
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Insurance and case manager help
Case managers help with benefits verification, prior authorizations and appeals, home delivery, pharmacist guidance, and in-home lab monitoring.
The official page does not say who qualifies. Ask the program. · source - Copay help
Commercially insured patients whose plan covers DUVYZAT may pay as little as $0 per month; annual maximum applies. Government-insured patients are not eligible.
For: private insurance · source - Free medicine program
Free DUVYZAT for eligible patients who have no insurance or whose insurance does not cover it.
For: no insurance, underinsured · source - Bridge or quick-start supply
Temporary supply programs provide medicine while waiting for insurance approval or other processes.
The official page does not say who qualifies. Ask the program. · source
Good to know: Eligibility is reviewed case by case. Case managers available Mon-Fri 8 AM-8 PM ET.
- From a charity · NORD RareCareDuchenne Muscular Dystrophy Medical Assistance fundOpen
Pays for: Medical and medication costs.
The foundation says: “Accepting new applications and re-enrollments for current year” - From a charity · NORD RareCareDuchenne Muscular Dystrophy Premium Copay Assistance fundOpen
Pays for: Insurance premiums and copays.
The foundation says: “Accepting new applications and re-enrollments for current year” - From a charity · The Assistance FundDuchenne Muscular Dystrophy fundOpen
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
The foundation says: “OPEN — Accepting New Patients. TAF is currently accepting new patient enrollments for this program.” - From a charity · Muscular Dystrophy AssociationMDA Durable Medical Equipment (DME) Grant Program fundApply directly
Pays for: Medical equipment (wheelchairs, lifts, canes and other DME), up to $1,000 per year.
The foundation says: “Status not shown on page”
Clinical trial results
The EPIDYS Phase 3 trial showed givinostat significantly slowed decline in functional assessments compared to placebo over 18 months. Treated boys maintained better climbing, walking, and rising ability than those on placebo.
Development history
Developed by Italfarmaco, an Italian pharmaceutical company. Approved by FDA in March 2024. Notable as the first DMD treatment that works across all mutation types (mutation-agnostic), complementing the exon-skipping drugs that only work for specific mutations.
Explore Duchenne Muscular Dystrophy trials
Other Duchenne Muscular Dystrophy treatments
Common questions about Givinostat
▸What is Givinostat (Duvyzat)?
The first non-steroid systemic treatment for DMD that works regardless of the patient's specific dystrophin mutation. Duvyzat targets the downstream muscle damage (inflammation and fibrosis) rather than the genetic defect itself.
▸How does Givinostat work?
In DMD, the lack of dystrophin causes ongoing muscle damage, which triggers chronic inflammation and replacement of muscle with scar tissue (fibrosis). Givinostat inhibits histone deacetylase enzymes, which reduces the inflammatory signals and slows down the fibrotic process. By preserving muscle quality, it helps maintain strength and function even though the underlying genetic defect remains.
▸What are the side effects of Givinostat?
The most common side effects are diarrhea, stomach pain, low platelet counts (thrombocytopenia), nausea or vomiting, high triglycerides and fever. Low platelets happened in 33% of treated boys in the main trial and can cause bleeding such as nosebleeds or bruising. It can also lower red and white blood cell counts and can prolong the QTc interval on an ECG, so it is avoided in people at higher risk of abnormal heart rhythms. Blood counts are checked every 2 weeks for the first 2 months, at month 3 and then every 3 months, and triglycerides at months 1, 3 and 6 and then every 6 months.
▸How is Givinostat taken?
Taken orally as a flavored liquid suspension twice daily with food. Dose is weight-based. Approved for people with DMD aged 6 and older. It should not be started if the platelet count is below 150 x 10^9/L (150,000 per microliter). The label does not limit use to people who can still walk, although the main trial enrolled boys who could walk. Can be used alongside corticosteroids and exon-skipping therapies.
▸Is Givinostat FDA approved?
Yes, Givinostat (Duvyzat) is FDA approved (2024) for the treatment of Duchenne Muscular Dystrophy.
▸Why is givinostat important for DMD patients regardless of mutation?
Most DMD drugs (exon-skipping therapies) only work for patients with specific dystrophin gene mutations, each covering about 8-13% of the DMD population. Givinostat works through a completely different mechanism: it reduces the inflammation and fibrosis (scarring) that damages muscle tissue in DMD, regardless of which specific mutation a patient has. This makes it the first non-steroid systemic therapy available to all DMD patients, not just those with specific mutations.
▸Can givinostat be used with other DMD treatments?
Yes. Givinostat can be used alongside corticosteroids (deflazacort, vamorolone, or prednisone) and exon-skipping therapies (eteplirsen, golodirsen, viltolarsen, casimersen). Because givinostat targets muscle inflammation and fibrosis rather than dystrophin production, it is complementary to treatments that address the genetic defect. Many patients in clinical trials were on background corticosteroids.
▸How does givinostat differ from corticosteroids?
Both reduce inflammation in muscle, but through different mechanisms. Corticosteroids broadly suppress the immune system, causing significant side effects like bone loss, weight gain, and growth suppression. Givinostat is an HDAC inhibitor that more selectively modulates inflammatory pathways and also directly reduces fibrosis (scar tissue formation). It does not cause the metabolic and skeletal side effects associated with corticosteroids. However, it is an add-on therapy, not a replacement for steroids.
▸What monitoring is needed while taking givinostat?
Platelet counts and triglycerides are checked before starting. Blood counts are then checked every 2 weeks for the first 2 months, at month 3 and every 3 months after that, and triglycerides at months 1, 3 and 6 and every 6 months after that. Children with heart disease or on other medicines that affect heart rhythm may also need ECGs. Your doctor will establish a monitoring schedule appropriate for your child.
▸Is givinostat only for boys who can still walk?
No. The FDA approval is for patients with DMD aged 6 and older, with no requirement that they can still walk. However, the main trial enrolled only boys who could walk. Whether givinostat provides similar benefit in non-ambulatory patients has not been established in controlled trials, though some non-ambulatory patients may still benefit from reduced muscle inflammation and fibrosis.