Elpida Therapeutics and CureCMT4J granted Rare Pediatric and Orphan Drug Designations from the FDA for CMT4J
Los Angeles, CA and Ipswich, MA – [December 2, 2024] – Elpida Therapeutics, at the forefront in gene therapy for ultra-rare diseases, and CureCMT4J, a nonprofit dedicated to advancing treatments for Charcot-Marie-Tooth disease type 4J (CMT4J), are pleased to announce that their gene therapy program has received both Rare Pediatric Disease (RPDD) and Orphan Drug (ODD) designations from the U.S. Food and Drug Administration (FDA). These designations underscore the urgent need for treatment options for children with this progressive neurodegenerative disease and mark a major milestone for the CMT4J community.
The RPDD and ODD designations from the FDA highlight the critical need for innovative therapies targeting CMT4J, granting Elpida Therapeutics and CureCMT4J eligibility for valuable incentives that will help accelerate the development process and bring this promising therapy closer to patients. This program, funded in part by a grant from the California Institute for Regenerative Medicine (CIRM), also benefits from participation in the Accelerating Medicines Partnership® (AMP®) Bespoke Gene Therapy Consortium (BGTC) led by the Foundation for the National Institutes of Health (FNIH), which provides a collaborative framework to address the unique challenges of developing therapies for ultra-rare diseases.
“The BGTC team congratulates Elpida Therapeutics and CureCMT4J for being granted Rare Pediatric and Orphan Drug Designations from the FDA for CMT4J“ said P.J. Brooks, co-chair of the BGTC. “Making gene therapies for rare diseases more attractive to potential commercial sponsors is one of the ultimate goals of the BGTC projects,” said Dominique Pichard, M.D., director of the NCATS Division of Rare Diseases Research Innovation.
“This achievement is a testament to the dedication of our research and patient communities. Gaining these designations from the FDA is a critical step forward in our mission to develop a treatment for those living with CMT4J,” said Jocelyn Duff, Executive Director of CureCMT4J and mother to Talia, a CMT4J patient. “Our partnership with Elpida represents the hope of countless families facing this devastating disease.”
Terry Pirovolakis, CEO of Elpida Therapeutics, commented, “Receiving RPDD and ODD for our CMT4J gene therapy program reflects our commitment to driving meaningful advances for those impacted by ultra-rare diseases. We are deeply grateful for the FDA's support and look forward to continuing our work with CureCMT4J to develop a therapeutic pathway that could transform lives.”
With FDA designations now in place, Elpida and CureCMT4J are positioned to accelerate the path to a potential treatment, offering new hope for patients and families affected by this ultra-rare disease.
About Elpida Therapeutics
Elpida Therapeutics is dedicated to delivering life-saving therapies for ultra-rare diseases. Founded to tackle the unmet medical needs of children living with rare conditions, Elpida works to rapidly advance its therapeutic programs in partnership with leading researchers and institutions around the globe. [www.elpidatx.com]
About CureCMT4J
Founded by Jocelyn Duff, CureCMT4J is a nonprofit organization leading the charge to bring treatments to people with CMT4J and FIG4-related disorders. Through research funding, advocacy, and community support, CureCMT4J is dedicated to advancing treatments for CMT4J, offering hope to families impacted by this ultra-rare disease. [www.curecmt4j.org]
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About the California Institute for Regenerative Medicine (CIRM)
At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast-track the development of today’s most promising stem cell technologies. CIRM is one of the world’s largest institutions dedicated to helping people by bringing the future of regenerative medicine closer to reality. [https://www.cirm.ca.gov/]
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The Accelerating Medicines Partnership and AMP are registered service marks of the U.S. Department of Health and Human Services.