Retinitis pigmentosa therapy receives rare pediatric disease designation
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Key takeaways:
- The FDA granted rare pediatric disease designation to VG901 for retinitis pigmentosa.
- An independent data safety monitoring board approved dose escalation in an ongoing trial assessing the therapy.
The FDA granted rare pediatric disease designation to VG901 for the treatment of retinitis pigmentosa caused by mutations in the CNGA1 gene, according to a press release from ViGeneron.
The designation allows the company to receive a priority review voucher upon marketing approval of VG901 that could be used to reduce the review period for any of ViGeneron’s drug candidates.
In addition, ViGeneron announced that an independent data safety monitoring board has unanimously approved dose escalation in an ongoing phase 1b clinical trial investigating the safety and preliminary efficacy of VG901. The therapy is designed to leverage the company’s vgAAV capsid to distribute the functional CNGA1 gene to retinal photoreceptor cells when delivered through intravitreal injection.
“This [rare pediatric disease designation] recognition from the FDA highlights the significant unmet medical need in retinitis pigmentosa and underscores VG901’s therapeutic potential as the first-in-class and only clinical-stage therapy targeting retinitis pigmentosa associated with mutations in the CNGA1 gene,” Caroline Man Xu, PhD, MBA, ViGeneron’s co-founder and CEO, said in the release. “In addition to the previously granted FDA orphan drug designation for VG901, the [rare pediatric disease designation] further supports our efforts to accelerate the development of VG901.”
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