Announced details
Treatment and clinical trials for pediatric patients with achondroplasia (ACH) aged 5–14 years
After changing to subcutaneous administration of 1 mg/kg once weekly, no new safety concerns through 3 months in the first subject
Of the 12 patients who completed the domestic Phase II clinical trial, 11 participated in the long-term dosing study, 7 remain on treatment, and 3 transitioned to 1 mg/kg once-weekly dosing (all as of the current date)
Orphan drug designation
Ministry of Health, Labour and Welfare
No impact on full-year earnings for the fiscal year ending March 2027
The company does not address Contract Value / Offering price.
| Item | Details |
|---|---|
| Names of Products / Services Covered | umedaptanib pegol (RBM-007, anti-FGF2 aptamer) |
| Applicable area | Treatment and clinical trials for pediatric patients with achondroplasia (ACH) aged 5–14 years |
| Award/certification name | Orphan drug designation |
| Awarding / certifying body | Ministry of Health, Labour and Welfare |
| Quantitative Effects | After changing to subcutaneous administration of 1 mg/kg once weekly, no new safety concerns through 3 months in the first subject |
| Usage history | Of the 12 patients who completed the domestic Phase II clinical trial, 11 participated in the long-term dosing study, 7 remain on treatment, and 3 transitioned to 1 mg/kg once-weekly dosing (all as of the current date) |
| Impact on Business Performance | No impact on full-year earnings for the fiscal year ending March 2027 |
Items this document does not state (11)
These are items the company does not state in this document. We do not leave them blank, nor fill them in from other sources or by inference.
The reasons the company gives
The company's own explanation, not something inferred from the numbers.To evaluate long-term safety using the same dosage and administration as the domestic Phase III clinical trial (WAKABA trial), and to supplement the safety information from the WAKABA trial in advance.