Section 3 of 6
Product information and disease description
63 evidence topics · 52 sources
Product description
Phase of product development
Product, sponsor, indication, and FDA orphan status: designation and approval status
| Field | Source quotation |
|---|---|
| Orphan designation | “Treatment of Stargardt disease” |
| Date designated | “09/19/2017” |
| FDA orphan approval status | “Not FDA Approved for Orphan Indication” |
FDA acceptance, review designation, and target action date
Additional regulatory designations
Product information
Generic, brand name and therapeutic class of product
Product, sponsor, indication, and FDA orphan status: product and sponsor
| Field | Source quotation |
|---|---|
| Generic name | “tinlarebant” |
| Sponsor | “Belite Bio, LLC” |
Names, drug class, and proposed mechanism of action: names and class
Dosage forms and strengths
Investigational dosage form
Average sales price and wholesale acquisition cost
Not applicable.
American hospital formulary service (AHFS), or other drug classification
No evidence found.
Indication
Investigational indication: orphan designation
Pharmacology
Mechanism of action
Names, drug class, and proposed mechanism of action: retinol delivery
Pharmacodynamics
Pharmacokinetics
Pharmacokinetics and drug interactions
Pharmacokinetics and drug interactions: food
Contraindications/Warnings/Precautions/Adverse effects
Warnings and precautions
Not applicable.
Special populations
Adolescents and young adults: studied age range
Hepatic and renal impairment
No evidence found.
Drug/Drug, drug/disease interactions
Effects of other drugs on Tinlarebant
Pharmacokinetics and drug interactions: coadministration
Publication recommendation concerning CYP3A inducers: not approved labeling
Investigator conclusion on coadministration and food
Effects of Tinlarebant on other drugs
No evidence found.
Dosing and administration
Dosage
DRAGON: investigational dose
Administration
Access and distribution
Anticipated patient financial support
Co-prescribed/Concomitant therapies
No evidence found.
Effect of Tinlarebant on quality measures
No evidence found.
Product comparison
Place of product in therapy
Disease description
Definition and etiology
Inherited retinal disease
Epidemiology
Incidence of Stargardt disease type 1
Incidence and prevalence: Netherlands nationwide registry: incidence
Prevalence of Stargardt disease type 1
Estimated U.S. patient population
Incidence and prevalence: Netherlands nationwide registry: prevalence
Natural history, survival, and mortality
Clinical course and visual prognosis: variability
ProgStar report no. 10: visual acuity change over 24 months
ProgStar report no. 17: progression of definitely decreased autofluorescence
Age at onset and prognosis by onset age
ProgStar report no. 1: baseline disease severity in the natural history cohorts
Pathophysiology
Diagnosis
Clinical and molecular diagnostic assessment
Mesopic microperimetry in Stargardt disease: application and reliability
Clinical presentation - signs and symptoms
Central vision: progressive visual loss
Long-term morbidity
Clinical course and visual prognosis: long-term acuity
Burden of Stargardt disease type 1
Humanistic burden and health-related quality of life
Vision-specific quality of life and functional vision: Brazil
Mental health outcomes in inherited retinal diseases: Stargardt disease subgroup
Patient-reported outcome measures in recessive Stargardt disease
Daily functioning, independence, and caregivers: daily functioning
Economic burden and healthcare resource utilization
Cost-of-illness studies of inherited retinal diseases: systematic review
Economic impact of Stargardt disease type 1 on families
Daily functioning, independence, and caregivers: caregiver needs
Patient testimony: costs of assistive technology
Economic impact of diagnostic testing
South Africa: time-limited 2025 genetic-testing offer
South Africa: additional genetic-counseling consultation cost
Approaches to treatment
Current treatment options and standard of care
Low-vision aids and vision rehabilitation
Stargardt disease: patient-care guidance: low-vision care
Lifestyle and light-exposure management
Stargardt disease: patient-care guidance: vitamin A supplementation
Genetic counseling
Limitations of current therapies
Summary
Current care includes low-vision aids, vision rehabilitation, and measures to limit avoidable retinal stress. The National Eye Institute describes no treatment for Stargardt disease, while the 2026 management review identifies supportive care as foundational. The 2018 technology assessment reported limitations in the size, duration, and quality of studies available at that time; it predates the completed DRAGON trial.
Treatments for dry age-related macular degeneration and Stargardt disease
Place in treatment, anticipated use, and care setting
Summary
Tinlarebant is being developed as an oral treatment for Stargardt disease type 1. DRAGON studied participants aged 12–20 years with established atrophic lesions and visual acuity of 20/200 or better. This trial population should not be equated with a finalized approved population. The sponsor anticipates confirmatory ABCA4 testing, specialty-pharmacy fulfillment, and patient-support programs. These are proposed access arrangements rather than an approved care pathway.
Heterogeneity of treatment effect
No evidence found.
Care management intervention strategies
Stargardt’s Connected Research Network inaugural meeting report
Other product development or post-marketing obligations required by the FDA
Not applicable.
Ongoing post-approval monitoring
Not applicable.