Petition to expedite FDA approval of MCO-010 for Retinitis Pigmentosa, a rare disease

Recent signers:
Josefa Fariña moreno and 19 others have signed recently.

The Issue

RETINITIS PIGMENTOSA (RP), a rare disease, and related retinal abnormalities are devastating genetic disorders affecting over **1 in 4,000 people worldwide**, leading to progressive vision loss and eventual blindness. There are **no FDA-approved treatments** to halt or reverse this condition.

#LightForRP  # PriorityReviewBLA #ApproveMCO010 #RareDisease 

Patients face:  

- **Physical challenges**: Loss of independence, mobility, and ability to perform daily tasks.  

- **Psychological suffering**: Depression, anxiety, and isolation due to irreversible vision loss.  

- **Financial burden**: Costs of assistive devices, caregivers, and lost productivity.  

MCO-010, developed by Nanoscope Therapeutics, represents a **transformative breakthrough**. As a **broad-spectrum optogenetic therapy**, it has the potential to restore functional vision across diverse genetic mutations, offering hope where none currently exists.  

 

We respectfully urge the FDA to:  

1. **Expedite the priority review** the priority review of the Biological License Application (BLA) for MCO-010.   

2. **Prioritize approval** of this therapy to address the urgent unmet medical need.  

3. **Engage collaboratively** with Nanoscope Therapeutics to streamline the regulatory process without compromising safety.  

**Every day without treatment deepens the crisis for millions living in darkness.**  

 

SUPPORTING FACTS

- **Clinical Promise**: MCO-010 has demonstrated success in early trials, restoring light sensitivity and functional vision in blind patients.  

- **Broad Applicability**: Unlike gene-specific therapies, MCO-010 works independent of genetic mutations, making it accessible to a wide population.  

- **Global Impact**: Over 2 million people worldwide suffer from RP and related retinal disorders.  

Submitted By:

** The Global Retinitis Pigmentosa (RP) and Genetic Retinal Disorders Patients Community**  

**WE, THE UNDERSIGNED, URGENTLY REQUEST THE FDA TO PRIORITIZE THE REVIEW AND APPROVAL OF MCO-010, A GROUNDBREAKING OPTOGENETIC THERAPY FOR IRREVERSIBLE VISION IMPAIRMENT AND BLINDNESS.**  

 

4,757

Recent signers:
Josefa Fariña moreno and 19 others have signed recently.

The Issue

RETINITIS PIGMENTOSA (RP), a rare disease, and related retinal abnormalities are devastating genetic disorders affecting over **1 in 4,000 people worldwide**, leading to progressive vision loss and eventual blindness. There are **no FDA-approved treatments** to halt or reverse this condition.

#LightForRP  # PriorityReviewBLA #ApproveMCO010 #RareDisease 

Patients face:  

- **Physical challenges**: Loss of independence, mobility, and ability to perform daily tasks.  

- **Psychological suffering**: Depression, anxiety, and isolation due to irreversible vision loss.  

- **Financial burden**: Costs of assistive devices, caregivers, and lost productivity.  

MCO-010, developed by Nanoscope Therapeutics, represents a **transformative breakthrough**. As a **broad-spectrum optogenetic therapy**, it has the potential to restore functional vision across diverse genetic mutations, offering hope where none currently exists.  

 

We respectfully urge the FDA to:  

1. **Expedite the priority review** the priority review of the Biological License Application (BLA) for MCO-010.   

2. **Prioritize approval** of this therapy to address the urgent unmet medical need.  

3. **Engage collaboratively** with Nanoscope Therapeutics to streamline the regulatory process without compromising safety.  

**Every day without treatment deepens the crisis for millions living in darkness.**  

 

SUPPORTING FACTS

- **Clinical Promise**: MCO-010 has demonstrated success in early trials, restoring light sensitivity and functional vision in blind patients.  

- **Broad Applicability**: Unlike gene-specific therapies, MCO-010 works independent of genetic mutations, making it accessible to a wide population.  

- **Global Impact**: Over 2 million people worldwide suffer from RP and related retinal disorders.  

Submitted By:

** The Global Retinitis Pigmentosa (RP) and Genetic Retinal Disorders Patients Community**  

**WE, THE UNDERSIGNED, URGENTLY REQUEST THE FDA TO PRIORITIZE THE REVIEW AND APPROVAL OF MCO-010, A GROUNDBREAKING OPTOGENETIC THERAPY FOR IRREVERSIBLE VISION IMPAIRMENT AND BLINDNESS.**  

 

The Decision Makers

FDA, the Center for Biologics Evaluation and Research (CBER)
FDA, the Center for Biologics Evaluation and Research (CBER)
ocod@fda.hhs.gov
Center for Drug Evaluation and Research (CDER)
Center for Drug Evaluation and Research (CDER)
Industry.Biologics@fda.hhs.gov

Supporter Voices

Petition Updates