PPMD is pleased to learn that DUVYZAT™ (givinostat) is now commercially available in the United States. DUVYZAT™ is a histone deacetylase (HDAC) inhibitor indicated for individuals diagnosed with Duchenne from six years of age and older that is being made available and marketed in the U.S. by ITF Therapeutics LLC. The drug was approved by the U.S. Food and Drug Administration (FDA) on March 21, 2024.

DUVYZAT™ works by targeting pathogenic processes to reduce inflammation and loss of muscle. It is the first nonsteroidal drug approved for Duchenne that is applicable regardless of the specific genetic variant. Currently, the drug is not indicated for the treatment of individuals aged five years and younger or patients with low platelet counts. DUVYZAT™ is not specific to genetic variants nor does it have any contraindications related to specific genetic variants (meaning all genetic variants should be eligible).

To find out if you or your child is eligible to receive DUVYZAT™, call your neuromuscular team, who will be able to provide you with the most appropriate information on next steps. 

Accessing DUVYZAT™

ITF Therapeutics also announced the launch of its patient services program, ITF ARC (Access, Resources and Care). According to the company, the program offers services to help patients and families navigate insurance challenges, personalized pharmacist support, financial and access assistance for eligible patients, educational materials, and other resources. For more information about patient support services offered through ITF ARC, visit www.DUVYZAT.com or call 1-855-448-3272.

You can also find information about DUVYZAT™ on PPMD’s Access & Coverage Resources page, as well as our DUVYZAT™FAQ surrounding the approval of DUVYZAT™, what this means, information on eligibility, access, and more. We will continue to update this FAQ as more information becomes available. 

View FAQ

PPMD is thrilled to see another approved therapeutic option for Duchenne become available to patients in the U.S. We remain committed to working towards community access to all approved products and will continue to work with all relevant stakeholders to ensure a favorable access environment for our community.

Read ITF Therapeutics’ community letter:

We are very pleased to share with you the exciting news that DUVYZAT™ (givinostat) is now commercially available in the U.S. for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older. To learn more about the commercial launch of DUVYZAT, please click here to see our press release.

Throughout the development of DUVYZAT we heard from so many families and members of the advocacy community about the critical need for new treatment options for DMD. As an HDAC inhibitor, DUVYZAT is a nonsteroidal therapy approved for the treatment of DMD and may be an option for appropriate patients regardless of their genetic mutation. The efficacy and safety of DUVYZAT were evaluated in the pivotal Phase 3 EPIDYS clinical trial, which was one of the largest Phase 3 clinical studies in DMD to date.

Over the past several months the entire ITF Therapeutics team has worked closely with many members of the DMD advocacy community to bring this new treatment option to patients as rapidly as possible. We are very grateful to you and to all the advocates, families, and clinicians who supported this effort. Patients and caregivers can now reach out to their healthcare providers to learn more about how to access treatment with DUVYZAT. To initiate treatment for eligible patients, healthcare providers must complete the Patient Start Form that is available on our dedicated website www.DUVYZAT.com. In addition to initiating the prescription fulfillment process, patients can also enroll in ITF ARC (Access, Resources and Care), the portfolio of patient support services available from ITF Therapeutics. Through ITF ARC, patients and caregivers can access services to help them navigate through insurance challenges, personalized pharmacist support, financial and access assistance for eligible patients, educational materials, and other resources. For more information about ITF ARC, please visit www.DUVYZAT.com/ITFARC.

The commercial launch of DUVYZAT represents a historic milestone in the treatment of DMD. It also reinforces our goal to be an effective partner and resource for all members of the advocacy community who are dedicated to improving care for patients. In the months ahead we will work to keep you updated on important news related to DUVYZAT. You can also reach out to us any time for any information you need. For general inquiries, contact us at generalinquiries@itftherapeutics.com, or reach out to Caroline Allen, Head of US Patient Advocacy and Communications, directly at c.allen@itftherapeutics.com.

Thank you for your interest in DUVYZAT.