FDA Grants MDA Kickstart Program Rare Pediatric Disease Designation and Orphan Drug Designation in Congenital Myasthenic Syndrome
The program assembles a collaborative team of in-house and external experts in research, clinical care, and regulatory affairs to navigate gene therapy programs through the drug development process.
According to a recent announcement, the FDA has granted the
"Receiving these important designations from the FDA for our pilot MDA Kickstart indication is an essential first step in supporting our ability to bring life-changing treatments to patients living with this rare disease,"
The MDA Kickstart program, launched in 2023, is a novel initiative designed to bridge the therapeutic gap for neuromuscular diseases affecting fewer than 1000 patients in the United States. Its primary goal is to accelerate the transition from early-stage research to clinical trials, reducing risk and streamlining the development of gene therapies for ultra-rare conditions. The program's first project, initiated in September 2024 in collaboration with UC Davis and biomanufacturing partner Forge Biologics, focuses on CMS caused by mutations in the CHAT gene.
CMS linked to CHAT gene mutations is an extremely rare condition, with fewer than 250 known cases in the United States. It is characterized by severe muscle weakness and potentially life-threatening episodes of respiratory failure. Ricardo A. Maselli, MD, a neurologist at UC Davis Health, has developed a gene therapy that targets this condition by replacing the defective CHAT gene. This approach offers a promising therapeutic option for patients who currently lack approved treatments.
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“For many of the young patients that I see in the clinic battling CMS, these designations are a crucial first step towards developing an effective therapy. These designations unlock a pathway to treatments that can profoundly change their lives and it's nice to see the FDA acknowledge CMS as a condition of unmet need,” Maselli, said in a statement.1
In partnership with Forge Biologics, MDA will leverage advanced AAV gene therapy process and analytical development manufacturing services. All production activities will be conducted at Forge’s 200,000-square-foot cGMP facility in Columbus, Ohio, utilizing the company’s proprietary technology to ensure high-quality manufacturing standards.
"We are excited to support the MDA Kickstart Program and help advance these critical projects to the next stage of development through our leading AAV manufacturing expertise and services," John Maslowski, president and chief executive officer at Forge, said in a statement.1 Collaborating with MDA embodies our mission at Forge to help deliver potentially life-changing treatments to patients."
Additionally, the rare pediatric disease designation granted to the MDA Kickstart program for CMS reflects ongoing federal efforts to incentivize the development of therapies for rare childhood diseases. However, the Priority Review Voucher program supporting these efforts is set to expire on December 20, 2024, unless Congress acts to extend it, underscoring the need for continued advocacy to maintain support for rare disease innovation.
"We’re proud to have made an impact with advocates for the reauthorization of the Rare Pediatric Disease PRV program as it is essential for continuing progress in rare disease treatment,"
“These designations enable us to synchronize a development plan with regulatory initiatives and incentives at an early stage.” Marina Kolocha, PharmD, PMP, project manager of the MDA Kickstart program, said in a statement.1
At the
Registration for the 2025 MDA Conference is now open! The meeting is set to be held at the Hilton Anatole, Dallas, Texas, from March 16 to 19, 2025. To register and for more information, head to
REFERENCES
1. MDA Kickstart Program Receives FDA Rare Pediatric Disease Designation and Orphan Drug Designation for Congenital Myasthenic Syndrome. News Release. MDA. Published October 24, 2024. Accessed December 17, 2024. https://www.mda.org/press-releases/mda-kickstart-program-receives-fda-rare-pediatric-disease-designation-and-orphan-drug-designation
2. Hesterlee S. MDA Kickstart Program for Ultra-Rare Gene Therapy Development. Presented at: MDA Clinical & Scientific Conference; March 19-22, 2023; Dallas, TX. Ultra Rare Track session.
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