WASHINGTON, March 21, 2024 /PRNewswire/ — Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), welcomed today’s decision by the Food and Drug Administration (FDA) to approve DUVYZAT™(givinostat) a histone deacetylase (HDAC) inhibitor indicated for individuals diagnosed with Duchenne six years of age and older. DUVYZAT™ marks a significant milestone as the first nonsteroidal drug approved for Duchenne that is applicable regardless of the specific genetic variant. It is the eighth approved therapy for Duchenne in the United States. DUVYZAT™ will be made available and marketed in the United States by ITF Therapeutics, LLC.
“PPMD is thrilled that the FDA has approved DUVYZAT™, an HDAC inhibitor therapy that works by targeting pathogenic processes to reduce inflammation and loss of muscle. Today’s approval expands the therapeutic options for individuals with Duchenne,” said PPMD Founding President and CEO Pat Furlong.
“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 also want to commend FDA leadership for recognizing the potential of DUVYZAT™ to make a difference in the lives of individuals living with Duchenne,” Furlong added.
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.
“Going forward, PPMD remains committed to working with all stakeholders and the community to do all we possibly can to strive for access for all patients who are eligible for DUVYZAT™. It is not enough to clear the FDA. We must make sure that all those who can benefit from these innovative therapies are able to access them as they reach the market,” said Furlong. “Additionally, PPMD believes that it will take a combination of therapies to fully halt progression and will continue to support the development of additional tools and resources to accelerate the development and approval of additional therapies and treatments for Duchenne and Becker muscular dystrophy.”
For three decades, PPMD has been dedicated to working with researchers, clinicians, industry, and families to advance treatments for all people living with Duchenne and Becker. The organization employs a comprehensive approach to identify opportunities to accelerate development of all therapies in Duchenne, including regulatory, research, and patient-recruitment counsel to help expedite the progress of investigational products.
PPMD’s patient-report registry, The Duchenne Registry, as well as PPMD’s Duchenne Outcomes Research Interchange, serve as resources for researchers and industry to access aggregated, de-identified information provided by patients and families. Additionally, PPMD remains committed to its sector-leading patient engagement efforts, which include multiple patient preference studies and a revised community-generated draft guidance document, recently updated to reflect advancements in knowledge, understanding, care, clinical trials, and approvals. PPMD also takes a lead role in advocating for national newborn screening efforts for Duchenne, Becker, and carrier identification, as well as policy efforts around access and reimbursement.
ABOUT PARENT PROJECT MUSCULAR DYSTROPHY:
Duchenne is a genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) fights every single battle necessary to end Duchenne.
We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won eight FDA approvals.
Everything we do—and everything we have done since our founding in 1994—helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and YouTube.
SOURCE Parent Project Muscular Dystrophy (PPMD)
