March 22, 2024 / Care,Clinical Trials,Research

FDA Grants Approval to DUVYZAT™ (Givinostat)

PPMD is excited to learn that the FDA has granted approval to DUVYZAT™ (givinostat), a histone deacetylase (HDAC) inhibitor indicated for individuals diagnosed with Duchenne muscular dystrophy from six years of age and older. DUVYZAT™ will be made available and marketed in the United States by ITF Therapeutics, LLC. 

DUVYZAT™ works by targeting pathogenic processes to reduce inflammation and loss of muscle. DUVYZAT™ is the first nonsteroidal drug approved for Duchenne that is applicable regardless of the specific genetic variant. Currently, it 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. 

PPMD will also begin including DUVYZAT™ on our Access & Coverage Resources page in the coming weeks as more information becomes available. 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.

What Does This Approval Mean?

PPMD has requested to host a webinar with ITF Therapeutics on Wednesday, March 27th at 1:00 PM ET to provide the community with an opportunity to meet members of the ITF Therapeutics team to learn more about their organization and next steps to support the availability of DUVYZAT™. Register now and submit your questions in advance here.

Register Now

Additionally, the PPMD team has compiled a resource of FAQs surrounding the approval of DUVYZAT™ and what this means, including questions on eligibility and access. We will continue to update this FAQ as more information becomes available. 

View FAQ

For three decades, PPMD has been dedicated to working with researchers, clinicians, industry, and families to advance treatments for all people living with Duchenne. 

We extend our heartfelt gratitude to the patients and their families who participated in these trials, as well as to Italfarmaco and the many dedicated clinicians and scientists who have tirelessly worked to develop this therapy. 

We are thrilled to see the approval of another therapeutic option for Duchenne. As we strive to ensure that these approved therapies become accessible to all who need them, today, we unite to celebrate another remarkable milestone in the fight for every future.

Read PPMD’s press release

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