4D Molecular Therapeutics (4DMT) has received regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration (FDA) for 4D-150 for diabetic macular oedema.
The therapy is tailored to deliver a multi-year sustained release of anti-vascular endothelial growth factor (anti-VEGF) with a single intravitreal injection.
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It is intended to alleviate the burden of frequent injections for individuals suffering from wet age-related macular degeneration (AMD) and diabetic macular oedema.
The designation, part of the US 21st Century Cures Act, aims to expedite the development and review of regenerative medicine therapies.
It provides benefits similar to the breakthrough therapy and fast track designation programmes, including more frequent interactions with the agency to facilitate the development of the drug.
4D-150 leverages the company’s intravitreal vector, R100, invented using 4DMT’s Therapeutic Vector Evolution platform.
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By GlobalDataDiabetic macular oedema is a diabetic retinopathy complication and is marked by macula swelling and inflammation due to blood vessel leakage, resulting in loss of vision.
4DMT CEO and co-founder David Kirn stated: “The RMAT designation is based on the review of our results to date from our ongoing 4D-150 SPECTRA diabetic macular oedema study, underscoring the potential of 4D-150 to sustain visual acuity improvements while dramatically reducing treatment burden for patients.
“This designation in diabetic macular oedema follows the RMAT designation granted for 4D-150 in wet AMD, and to our knowledge, 4D-150 is the first investigational medicine to be granted the designation in both indications.
“We look forward to continuing our ongoing collaboration with the FDA to advance 4D-150 into Phase III development with an aligned-upon single Phase III trial for approval in diabetic macular oedema, combined with our two wet AMD 4FRONT Phase III clinical trials.”
In January 2024, the company received a rare paediatric disease designation from the FDA for its cystic fibrosis gene therapy candidate, 4D-710.
