FDA 授予 Octant 在研口服小分子 OCT-980 孤儿药资格，用于视网膜色素变性
FDA grants orphan drug status to Octant’s OCT-980 for RP
FDA 已授予 Octant 在研口服小分子 OCT-980 孤儿药资格，用于治疗视网膜色素变性（RP），具体涵盖视紫红质相关常染色体显性视网膜色素变性（RHO-adRP）。OCT-980 通过纠正蛋白错误折叠、恢复视紫红质正常细胞内转运发挥作用，目前正在美国开展 Phase I/II 研究。该资格可带来获批后七年美国市场独占权及临床试验税收抵免等激励。
The regulatory status specifically includes RHO-adRP, the indication for which the candidate is presently being evaluated.
The US Food and Drug Administration (FDA) has granted orphan drug designation to Octant’s OCT-980, an investigational oral small molecule, to treat retinitis pigmentosa (RP).
The regulatory status specifically includes rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP), the indication for which the candidate is presently being evaluated.
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Orphan status is granted to support the development of therapies for rare diseases and conditions.
This designation offers financial and regulatory incentives, including eligibility for seven years of US market exclusivity upon approval, potential tax credits on qualified clinical trials, and relief from certain agency application fees, subject to applicable legal requirements.
OCT-980 is created to address the disorder at its origin. It works by correcting underlying protein misfolding and restoring the normal cellular trafficking of rhodopsin.
The therapy is currently undergoing clinical assessment in a Phase I/II study.
Octant co-founder and CEO Sri Kosuri said: “People with RHO-adRP are living with progressive vision loss and no approved treatment options today.
“This designation is another step forward as we advance OCT-980. With our Phase Ib/II study underway at US sites, we are focused on generating the clinical data needed to establish OCT-980 as an oral, disease-modifying option for RHO-adRP.”
RP represents a collection of inherited retinal disorders characterised by gradual vision loss that can culminate in total blindness. RHO-adRP is driven by disease-linked variants in the rhodopsin gene.
Octant is a clinical-stage drug discovery company developing correctors for conditions involving protein misfolding.
Its platform, The Navigator, combines generative chemistry, high-throughput synthetic biology, and AI and machine learning to target complex cellular mechanisms in human cells.
In addition to OCT-980, the company is advancing pipeline programmes across rare diseases and oncology.
来源：Pharmaceutical Technology · pharmaceutical-technology.com