Ocugen’s OCU410ST receives rare pediatric disease designation for the treatment of ABCA4-associated retinopathies

Author(s):

Martin David Harp

Key Takeaways

OCU410ST has received a rare pediatric disease designation for ABCA4-associated retinopathies, potentially allowing Ocugen to obtain a priority review voucher (PRV).
The AAV delivery platform for the RORA gene targets key pathways in Stargardt disease, including lipofuscin formation and oxidative stress.
The PRV program, crucial for rare pediatric disease innovation, faces criticism for benefiting large companies and is set to sunset unless reauthorized.
Ocugen plans to initiate a phase 2/3 trial and submit a biologics license application in 2027.

ABCA4-associated retinopathies include Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3.

(Image Credit: AdobeStock/mod)

The US Food and Drug Administration (FDA) has granted a rare pediatric disease designation (RPDD) to Ocugen’s OCU410ST for the treatment of ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3.¹

Previously, OCU410ST received orphan drug designations for the treatment of ABCA4-associated retinopathies from the FDA and European Medicines Agency.

OCU410ST uses an adeno-associated virus delivery platform for the retinal delivery of the RORA gene. RORA regulates pathophysiological pathways linked to Stargardt disease, such as lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks.¹

Shankar Musunuri, PhD, MBA, chairman, CEO, and cofounder of Ocugen, commented on the designation in a company press release, saying, “This latest designation for OCU410ST reaffirms the urgency of providing a therapeutic option to patients [with Stargardt disease] who have no FDA-approved treatment available.”

With this designation for OCU410ST, Ocugen may be awarded a priority review voucher (PRV) if the US Congress reauthorizes the PRV program. If awarded, a PRV can be redeemed to receive priority review for a different product or sold to another sponsor, and it typically sells for about $100 million or more.

Priority Review Voucher

The program shortened the FDA review period, cutting the review time from the standard 10 months to 6 months.
PRV Program awarded 53 vouchers across 39 different rare pediatric diseases.
350 additional Rare Pediatric Disease (RPD) designations granted between 2020 and 2022.
AbbVie paid $350 million for a PRV from United Therapeutics in 2015, which was used for Rinvoq.

The PRV program was signed into law in 2012 but has since expired, and the FDA began sunsetting the program as of December 20, 2024. The agency stated it will not award any new vouchers unless the drug held the RPDD prior to the sunset date. The FDA can award vouchers until 2026 for eligible drugs if Congress has not reauthorized the PSV program by then.²

Critics of the program claimed it primarily benefited large pharmaceutical companies rather than driving innovation for rare diseases. They also claimed the system was being exploited for financial gain rather than serving its original purpose.^3,4

Although support for the program lies in the belief that it is critical for incentivizing innovation in rare pediatric diseases, such diseases are particularly complex to research because conducting clinical trials with children is “challenging."^3,4

Ocugen noted it plans to initiate a phase 2/3 pivotal confirmatory trial in the coming weeks and submit a biologics license application in 2027.

References:

Ocugen announces rare pediatric disease designation granted for OCU410ST—modifier gene therapy for the treatment of Stargardt disease. News release. Ocugen, Inc. May 27, 2025. Accessed May 27, 2025. https://www.globenewswire.com/news-release/2025/05/27/3088484/0/en/Ocugen-Announces-Rare-Pediatric-Disease-Designation-Granted-for-OCU410ST-Modifier-Gene-Therapy-for-the-Treatment-of-Stargardt-Disease.html
Rare pediatric disease designation and priority review voucher programs. FDA. Updated September 27, 2024. Accessed May 27, 2025. https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/rare-pediatric-disease-designation-and-priority-review-voucher-programs#2024update
The end of the FDA’s rare pediatric disease priority review voucher program. ProPharma. March 11, 2025. Accessed May 27, 2025. https://www.propharmagroup.com/thought-leadership/fda-rare-pediatric-disease-priority-review-voucher-prv-program/
Armstrong A. Rare disease biotechs left in a lurch as Congress fails to renew priority review program. BioSpace. March 5, 2025. Accessed May 27, 2025. https://www.biospace.com/business/rare-disease-biotechs-left-in-a-lurch-as-congress-fails-to-renew-priority-review-program

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Ocugen’s OCU410ST receives rare pediatric disease designation for the treatment of ABCA4-associated retinopathies

Key Takeaways

Priority Review Voucher

References:

Rare pediatric disease designation and priority review voucher programs. FDA. Updated September 27, 2024. Accessed May 27, 2025. https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/rare-pediatric-disease-designation-and-priority-review-voucher-programs#2024update

The end of the FDA’s rare pediatric disease priority review voucher program. ProPharma. March 11, 2025. Accessed May 27, 2025. https://www.propharmagroup.com/thought-leadership/fda-rare-pediatric-disease-priority-review-voucher-prv-program/

Armstrong A. Rare disease biotechs left in a lurch as Congress fails to renew priority review program. BioSpace. March 5, 2025. Accessed May 27, 2025. https://www.biospace.com/business/rare-disease-biotechs-left-in-a-lurch-as-congress-fails-to-renew-priority-review-program

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