FDA Grants Breakthrough Therapy Status to Iveric Bio’s Zimura for AMD

In a significant development for the treatment of age-related macular degeneration (AMD), the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to Iveric Bio’s Zimura. This designation acknowledges the potential of Zimura to provide a significant improvement over existing treatments for AMD, a leading cause of vision loss in older adults. In this blog post, we will focus on the key points surrounding the FDA’s decision to grant Breakthrough Therapy status to Zimura.

1. Age-Related Macular Degeneration (AMD)

AMD is a progressive eye disease that affects the macula, the part of the retina responsible for central vision. It is the leading cause of vision loss in older adults, with two forms: dry AMD and wet AMD. Dry AMD is more common and occurs when the macula thins over time, leading to gradual vision loss. Wet AMD, although less common, is more severe and occurs when abnormal blood vessels grow under the macula, leaking fluid and causing rapid vision loss.

2. The Significance of Breakthrough Therapy Designation

Breakthrough Therapy designation by the FDA is granted to expedite the development and review process of drugs that demonstrate potential advantages over existing therapies for serious or life-threatening conditions. It is a recognition of the potential of the treatment to improve patient outcomes significantly. The designated therapy also receives enhanced guidance and support from the FDA throughout the development and review process.

3. Zimura’s Mechanism of Action

Zimura, developed by Iveric Bio, is an experimental drug that targets the Complement C5 protein, a key factor in the inflammatory pathway associated with the development and progression of AMD. By inhibiting Complement C5, Zimura aims to reduce inflammation and prevent damage to the retina, ultimately slowing or halting the progression of AMD and preserving vision in affected individuals.

4. Clinical Trial Results

The FDA’s decision to grant Breakthrough Therapy status to Zimura was based on positive results from clinical trials. Iveric Bio conducted a Phase IIb clinical trial evaluating the efficacy and safety of Zimura in patients with geographic atrophy, a type of advanced dry AMD. The trial demonstrated a statistically significant reduction in the rate of geographic atrophy progression in patients receiving Zimura compared to those receiving a placebo.

5. Implications for AMD Treatment

The grant of Breakthrough Therapy status to Zimura represents a major milestone in the treatment of AMD. Currently, treatment options for AMD are limited, with no available therapies to halt the progression of dry AMD. If approved by the FDA, Zimura has the potential to become a groundbreaking therapy for AMD patients, offering them a chance to preserve their vision and improve their quality of life.

6. Next Steps

With the Breakthrough Therapy designation, Iveric Bio will continue working closely with the FDA to efficiently advance the development of Zimura. The company plans to initiate a Phase III clinical program to further evaluate the drug’s efficacy and safety in a larger patient population. If the results continue to be promising, Zimura could potentially be approved for the treatment of AMD and become a game-changer in managing this debilitating eye disease.


The FDA’s granting of Breakthrough Therapy status to Iveric Bio’s Zimura underscores the potential of this innovative drug in revolutionizing the treatment of AMD. With its distinct mechanism of action and promising clinical trial results, Zimura offers hope to individuals affected by AMD, who currently face limited treatment options. As further research and development progress, Zimura has the potential to transform the landscape of AMD treatment, preserving vision and enhancing the quality of life for countless patients.