“This approval represents a major advancement in the treatment of pediatric myasthenia gravis and provides hope to families navigating this complex disease,” said Sharon Hesterlee, PhD, Chief Research Officer at the Muscular Dystrophy Association (MDA). “The availability of Soliris for children underscores the importance of continued research and innovation in neuromuscular disease treatments.”
Donald S. Wood, PhD, President and CEO of MDA, highlighted the role of nonprofit organizations in advancing rare disease treatments. “The approval of Soliris for pediatric myasthenia gravis is a major milestone—proof of how far science has come thanks to donors and supporters who believe in the power of scientific possibility,” he said. “By funding research, advocating for the community, and working alongside industry partners, we are pushing the boundaries to bring life-changing therapies to people with neuromuscular diseases.”
vTv Therapeutics’ Cadisegliatin Program Resumes as FDA Lifts Clinical Hold
vTv Therapeutics announced that the FDA has lifted the clinical hold on its cadisegliatin clinical program, including the CATT1 Phase III trial for type 1 diabetes (T1D). Cadisegliatin, a liver-selective glucokinase activator, is being developed as a first-in-class oral adjunctive therapy to insulin and has been well tolerated in over 500 subjects with up to six months of treatment.
To expedite topline data collection, vTv plans to submit a protocol amendment reducing the trial duration from 12 months to 6 months while maintaining the original primary endpoint of assessing level 2 and 3 hypoglycemia rates at 6 months. This adjustment will accelerate the initiation of pivotal studies required for a future New Drug Application (NDA) submission.
“We are pleased that the FDA has lifted the clinical hold on our cadisegliatin program and are eager to resume our Phase III trial,” said Paul Sekhri, Chairman, President, and CEO of vTv Therapeutics. “By shortening the trial duration, we can obtain data sooner and advance cadisegliatin toward becoming the first oral adjunctive therapy to insulin for T1D.”
The FDA initially placed a hold on the CATT1 trial in July 2024 due to an unresolved chromatographic signal in an absorption, distribution, metabolism, and excretion (ADME) study. No patients had been dosed at the time, and past clinical trials showed no safety concerns. The hold was lifted on March 14, 2025, after vTv’s response demonstrated the signal was an experimental artifact.
ENCell’s EN001 Granted Orphan Drug Designation for Charcot-Marie-Tooth Disease
ENCell announced that its investigational stem cell therapy, EN001, has received an Orphan Drug Designation (ODD) from the FDA for the treatment of Charcot-Marie-Tooth disease (CMT). CMT is a progressive neuromuscular disorder with no approved treatments, making this designation a significant step toward addressing an unmet medical need.
EN001, developed using ENCell’s proprietary ENCT platform, enhances cell longevity and optimizes the secretion of therapeutic molecules. The therapy targets damaged nerves, promotes regenerative factor secretion, and supports remyelination. A Phase I trial in CMT type 1A patients showed no dose-limiting toxicity, serious adverse events, or injection-related reactions. Based on these results, a high-dose Phase Ib trial was initiated in December 2024, with completion expected in 2025.
“The FDA’s Orphan Drug Designation for EN001 is a significant milestone that will accelerate its clinical development,” an ENCell representative stated. “We are committed to completing the ongoing Phase Ib trial and ensuring timely access to this innovative treatment for CMT patients.”
Beyond CMT, EN001 is being explored for Duchenne Muscular Dystrophy (DMD) and Sarcopenia, positioning it as a promising next-generation stem cell therapy for muscular diseases.
FDA Accepts Sydnexis’ NDA for SYD-101 in Pediatric Myopia
Sydnexis announced that the FDA has accepted its New Drug Application (NDA) for SYD-101, a potential first-in-class treatment for the progression of pediatric myopia, with a Prescription Drug User Fee Act (PDUFA) target action date of October 23, 2025. If approved, SYD-101 would be the first FDA-approved pharmaceutical option for managing myopia progression in children across the U.S.
The NDA is supported by data from the STAR Study, the largest clinical trial conducted for pediatric myopia treatment, enrolling over 850 children aged 3 to 14. The study evaluated the safety and efficacy of Sydnexis’ proprietary low-dose atropine formulation over three years.
“SYD-101’s novel formulation was designed to deliver superior drug activity, maximum stability, and optimal comfort,” said Patrick Johnson, Ph.D., President of Sydnexis.
“The FDA’s acceptance of our NDA is a major step toward offering an innovative treatment for millions of children with progressive myopia,” said Perry Sternberg, CEO of Sydnexis. “We look forward to working with the FDA to bring a once-daily, safe, and effective eye drop to patients, families, and clinicians.”
Experts emphasize the growing need for an FDA-approved therapy for pediatric myopia. “Having a safe and effective treatment for myopic children would be a critical advancement in tackling this global epidemic,” said Dr. Gregory Ostrow, Director of Pediatric Ophthalmology at Scripps Clinic. Dr. Paul Karpecki, OD, FAAO, added, “A first FDA-approved option for early intervention is urgently needed to reduce the long-term risks associated with myopia.”
Cambium Bio’s Potency Assay Strategy Approved for Elate Ocular Phase III Trials
Cambium Bio has achieved a significant regulatory milestone, securing FDA approval for its potency assay strategy to support Phase III clinical trials of Elate Ocular, a novel biologic therapy for moderate to severe dry eye disease. Following a successful Type D meeting, the FDA endorsed Cambium Bio’s proposed cell-based assay, alongside EGF and PDGF-BB ELISA assays, as a suitable lot release potency assay through to a future Biologics License Application (BLA) submission.
Additionally, the FDA approved the company’s approach to establishing acceptance criteria for the cell-based EGF activity bioassay, which will be used to assess clinical trial material and the eventual commercial product. The agency also confirmed the bioassay’s suitability for incorporation into a comparability protocol, ensuring consistency between Phase I/II investigational products and the pathogen-inactivated Phase III formulation.
These positive outcomes mark one of the final Chemistry, Manufacturing, and Controls (CMC) hurdles before Cambium Bio moves forward with its pivotal Phase III trials, expected to begin in mid-2025. The company will now focus on finalizing the validation of this potency assay with its Contract Development and Manufacturing Organization (CDMO) to meet regulatory requirements ahead of trial initiation.
“The FDA’s agreement on our potency assay strategy represents a critical regulatory milestone as we prepare to advance Elate Ocular® into Phase III trials,” said Karolis Rosickas, CEO of Cambium Bio. “This validation underscores the strength of our CMC and analytical development approach and keeps us on track to initiate our registration-enabling Phase III program mid-year.”
