FDA Grants RMAT Status to 4DMT's Diabetic Macular Edema Therapy

Diabetic macular oedema, a severe complication of diabetic retinopathy, leads to macula swelling and inflammation due to blood vessel leakage, ultimately causing vision loss. Addressing this condition, 4D Molecular Therapeutics (4DMT) has announced a significant milestone: the US Food and Drug Administration (FDA) has awarded its therapy, 4D-150, the regenerative medicine advanced therapy (RMAT) designation. This designation is designed to accelerate the development and review processes for regenerative therapies.

4D-150 is engineered to provide a long-term release of anti-vascular endothelial growth factor (anti-VEGF) through a single intravitreal injection. This innovative approach aims to reduce the frequency of injections required for patients with wet age-related macular degeneration (AMD) and diabetic macular oedema, thus decreasing the treatment burden. The therapy utilizes 4DMT's proprietary intravitreal vector, R100, a product of their Therapeutic Vector Evolution platform.

The RMAT designation, part of the US 21st Century Cures Act, presents several advantages akin to those of the breakthrough therapy and fast track designation programs. These benefits include increased interaction with the FDA, which can facilitate smoother and faster drug development and review processes. Such a designation highlights the potential of 4D-150 to not only maintain visual acuity in patients but also significantly lessen the number of treatments needed.

David Kirn, CEO and co-founder of 4DMT, commented on the RMAT designation, emphasizing that it is a testament to the promising results from their ongoing 4D-150 SPECTRA study focused on diabetic macular oedema. He noted that this was the second RMAT designation for 4D-150, with the first being for wet AMD. This makes 4D-150 the first investigational treatment to receive the RMAT designation for both diabetic macular oedema and wet AMD.

Kirn also expressed the company's commitment to working closely with the FDA to advance 4D-150 into Phase III trials. The plan includes a singular Phase III trial for diabetic macular oedema approval, alongside two wet AMD 4FRONT Phase III clinical trials. This collaborative effort aims to bring 4D-150 closer to becoming an approved treatment option, potentially transforming the management of these vision-threatening conditions.

In another development, 4DMT received a rare paediatric disease designation from the FDA for its cystic fibrosis gene therapy candidate, 4D-710, in January 2024. This designation underscores the diverse therapeutic avenues 4DMT is exploring, reinforcing its position in the field of advanced medicinal therapies. Through its innovative approaches, 4DMT continues to strive towards significant advancements in treating complex diseases, offering hope to patients with limited options.

Overall, the RMAT designation for 4D-150 represents a promising step forward in the treatment of diabetic macular oedema. By potentially reducing the frequency of necessary injections and improving patient outcomes, 4DMT is setting the stage for a new era in ocular treatment, further demonstrating its commitment to addressing unmet medical needs.