OCU400

Extended indication	Patients with retinitis pigmentosa (RP) associated with RHO mutations and patients with any other RP associated mutation with a clinical phenotype of RP
Therapeutic value	No estimate possible yet
Registration phase	Clinical trials

Product

Active substance	OCU400
Domain	Neurological disorders
Reason of inclusion	New medicine (specialité)
Main indication	Eye disorders
Extended indication	Patients with retinitis pigmentosa (RP) associated with RHO mutations and patients with any other RP associated mutation with a clinical phenotype of RP
Manufacturer	Ocugen
Mechanism of action	Gene therapy
Route of administration	Ocular
Therapeutical formulation	Concentrate for solution for injection
Budgetting framework	Intermural (MSZ)

Registration

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
ATMP	Yes
Submission date	September 2025
Expected Registration	October 2026
Orphan drug	Yes
Registration phase	Clinical trials
Additional remarks	Primary completion (estimated): 30 juni 2025.

Therapeutic value

Therapeutic value	No estimate possible yet
References	NCT06388200

Expected patient volume per year

Patient volume	2,000 - 3,100 Market share is generally not included unless otherwise stated.
References	1: https://www.orpha.net/ ; 2: https://www.lumc.nl/actueel/2023/gentherapie-voor-retinitis-pigmentosa-werkt-op-gekweekt-netvlies/; 3: Diverse Genetic Landscape of Suspected Retinitis Pigmentosa in a Large Korean Cohort Yoon-Jeon et al. Genes (Basel). 2021 May; 12(5): 675.;
Additional remarks	De wereldwijde prevalentie van retinitis pigmentosa wordt geschat op 1:3.000 tot 1:5000 (1). In Nederland wordt het patiëntvolume geschat op 4.000 (2). De RHO mutatie is de meest voorkomende 5 tot 10% van de gevallen. Het percentage van genetische mutaties dat gediagnosticeerd wordt in patiënten met een familie historie van de ziekte (70,7%) ligt hoger dat dat van sporadische gevallen van de ziekte (51,3%) (3).

Patient volume

2,000 - 3,100

Market share is generally not included unless otherwise stated.

References

1: https://www.orpha.net/ ; 2: https://www.lumc.nl/actueel/2023/gentherapie-voor-retinitis-pigmentosa-werkt-op-gekweekt-netvlies/; 3: Diverse Genetic Landscape of Suspected Retinitis Pigmentosa in a Large Korean Cohort Yoon-Jeon et al. Genes (Basel). 2021 May; 12(5): 675.;

Additional remarks

De wereldwijde prevalentie van retinitis pigmentosa wordt geschat op 1:3.000 tot 1:5000 (1). In Nederland wordt het patiëntvolume geschat op 4.000 (2). De RHO mutatie is de meest voorkomende 5 tot 10% van de gevallen. Het percentage van genetische mutaties dat gediagnosticeerd wordt in patiënten met een familie historie van de ziekte (70,7%) ligt hoger dat dat van sporadische gevallen van de ziekte (51,3%) (3).

Expected cost per patient per year

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.