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Sepofarsen
Tier C — Watch / tradeSepofarsen
Proqr Therapeutics
Originator
-
First approval
-
Expiry (public)
No sales data
Peak sales
Nucleic acid drug
Type
Targets：Cep290
Indications (approved)：感觉障碍, 莱伯氏先天性黑蒙, 神经系统表现, 失明, 视觉障碍, 视网膜病变, 眼畸形, 遗传性眼睛疾病
Regional status
US
Phase III
2019-04-04
EU
Phase III
2019-04-04
China
-
Japan
-
Synthetic routes & intermediates
Routes not yet onboarded — the daily pipeline enriches by priority (approved small molecules first); this page updates automatically.
Frequently asked questions
What are the approved indications of Sepofarsen?
Approved indications include: 感觉障碍, 莱伯氏先天性黑蒙, 神经系统表现, 失明, 视觉障碍, 视网膜病变, 眼畸形, 遗传性眼睛疾病.
When does the Sepofarsen patent expire?
No public expiry year is available for Sepofarsen yet; this page updates once verified.
How do I get a quote for Sepofarsen intermediates?
Open the CAS profile of the target intermediate from this page, or submit an RFQ directly — the CAS enters the matching flow automatically and the supply network responds with quotes.
Source: cross-verified data (structured fields only) · 2026-10-08