Shanghai U‑Yin Pharmaceutical (U‑Yin Pharma) announced that the National Medical Products Administration (NMPA) has approved its Investigational New Drug (IND) application for EHT102 Injection, a gene therapy for Autosomal Recessive Deafness 9 (DFNB9), marking one of the first Class 1 innovative drugs to successfully leverage NMPA’s new 30‑Day Review for Innovative Drug Clinical Trials policy enacted in September 2025.
Regulatory Milestone
Item
Detail
Company
Shanghai U‑Yin Pharmaceutical (private)
Drug
EHT102 Injection
Application
IND (Investigational New Drug)
Agency
NMPA (China)
Review Pathway
30‑Day Accelerated Review for Innovative Drugs
Approval Date
12 Jan 2026
Innovation
First gene therapy IND approved under new 30‑day policy
Indication
DFNB9 caused by biallelic OTOF gene mutations
Drug Profile & Mechanism of Action
Mechanism:AAV‑based gene therapy delivering functional OTOF (otoferlin) gene to cochlear hair cells, restoring synaptic vesicle fusion and auditory signal transmission
Disease Pathology: DFNB9 patients lack functional otoferlin protein, resulting in severe‑to‑profound congenital hearing loss and speech development barriers
Advantages:
Single‑administration intracochlear injection
Restores natural auditory function vs. artificial stimulation from cochlear implants
Targets root genetic cause rather than symptomatic treatment
Preclinical Data: In OTOF‑knockout mouse models, EHT102 achieved 70‑80 % hearing threshold restoration and synaptic structure normalization at 6 months post‑administration
Market Opportunity & Patient Landscape
Parameter
China
Global
Annual Births
9.5 million
135 million
DFNB9 Incidence (1:100,000)
~95 newborns
~1,350 newborns
Total Prevalent Cases
1,200‑1,500
18,000‑22,000
Currently Undiagnosed/Treated
70 %
65 %
Addressable Market (2035E)
450 patients
7,200 patients
Average Gene Therapy Cost
¥1.2 million
$1.8 million
Current Standard of Care: Hearing aids (ineffective in profound loss) and cochlear implants (provide artificial hearing, no natural sound quality)
Unmet Need:No approved therapies restore natural hearing; families face lifetime speech therapy costs exceeding $500,000 per patient
Speed Advantage: NMPA’s 30‑day review (vs. standard 60‑90 days) accelerates study start by 2‑3 months, enabling first patient dosed in Q1 2026
Manufacturing: U‑Yin’s Shanghai gene therapy facility (capacity 2,000 doses/year) GMP‑certified; dedicated AAV production line operational
Global Strategy: Plans FDA IND filing in H2 2026 leveraging China data; targeting rare pediatric disease priority review voucher worth ~$100 M
Financial Projections
Parameter
2026E
2027E
2028E
China DFNB9 Diagnoses
85
95
105
EHT102 Treatment Rate
0 %
55 %
75 %
Annual Revenue (¥)
–
¥62 million
¥94 million
Operating Margin
–
68 %
72 %
Cumulative R&D Investment
¥180 million
¥240 million
¥300 million
Pricing Strategy: Gene therapy pricing expected at ¥1.2‑1.5 million ($170K‑210K), comparable to Zolgensma and Luxturna
Market Access: Eligible for China Rare Disease Insurance (reimbursement up to 70 % in provinces like Zhejiang and Jiangsu)
Forward‑Looking Statements This brief contains forward‑looking statements regarding EHT102 clinical development timelines, regulatory pathways, and commercial forecasts. Actual results may differ due to clinical trial outcomes, manufacturing scale‑up challenges, and competitive dynamics in the hearing loss gene therapy space.-Fineline Info & Tech
By Fineline Cube