日本時間の4月20日（水）21:00-24:30において、バイオマリン社のR&D dayがニューヨークで開催されました。
各被験者群の1年間の投与結果において、投与前のベースラインと比較して、投与後の年間成長速度は、46%-65%増加し、一貫性のある効果が認められました。
今回得られたデータは以下のとおりです。
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) will host an R&D Day from 8:00 am (ET) to 11:30 pm (ET) on Wednesday, April 20, 2016 in New York City.
About one year of data is included for each cohort. The data shows the durable and consistent effects on mean annualized growth velocity for up to 12 months with increases of 46%-65% from the baseline.
The new data is below.
Table 1: Subject Disposition and Demographics
| Category | Cohort 3 (n=10) |
Cohorts 1 and 2 (n=12)* |
| Subjects Enrolled and Treated at 15 µg/kg/day | 10 (100%) | 12 (100%) |
| Subjects Who Completed 6 Months at 15 µg/kg/day | 10 (100%) | 12 (100%) |
| Subjects Who Completed 12 Months at 15 µg/kg/day | 10 (100%) | N/A |
| Age (years) at Enrollment | ||
| Mean (SD) | 8.0 (1.63) | 7.6 (1.88) |
| Min, Max | 6, 11 | 5, 10 |
| Gender (n, %) | ||
| Male | 4 (40%) | 6 (50%) |
| Female | 6 (60%) | 6 (50%) |
*Subjects increased dose to 15 µg/kg/day after at least 6 months at 2.5 and/or 7.5 µg/kg/day; 4 of original 16 subjects in Cohorts 1 and 2 did not initiate dosing at 15 µg/kg/day due to subject decision to withdraw from the study (2), declining extension study (1), and growth plate closure (1)
Source : The press release”BioMarin Provides Program Update on Vosoritide in Achondroplasia” on Apr 20, 2016
Table 2: Vosoritide Summary of Efficacy Results from Phase 2 Study in Children with Achondroplasia
| Efficacy Analysis: Annualized Growth Velocity | |||||||||
| Time Point |
6 Months | 12 Months ** | 6 Months |
||||||
| Annualized Growth Velocity
|
Cohort 3 15 µg/kg/daily (n=10) |
Cohort 3 15 µg/kg/daily (n=10) |
Cohorts 1, 2 15 µg/kg/daily (n=12) |
||||||
| Baseline | |||||||||
| Mean (SD), cm/Year | 4.0 (2.3) | 4.0 (2.3) | 3.6 (0.96) | ||||||
| Median | 4.1 | 4.1 | 3.5 | ||||||
| Post-Treatment | |||||||||
| Mean, (SD), cm/year | 6.1 (1.1) | 5.9 (0.92) | 5.9 (1.6) | ||||||
| Median | 5.9 | 5.6 | 5.6 | ||||||
| Change from Baseline | |||||||||
| Mean (SD), cm/year | 2.0 (2.0) | 1.9 (2.0) | 2.3 (1.9) | ||||||
| Nominal p-value* | 0.01 | 0.02 | 0.002 | ||||||
| Percent increase from Baseline | 50 | % | 46 | % | 65 | % | |||
| Based on means (%) | |||||||||
* Nominal p-value, not adjusted for multiplicity
** Mean Annualized Growth Velocity change from baseline increases to 2.0 cm/year (50% increase) if one patient who missed majority of doses between 6 and 12 months is excluded
Source : The press release”BioMarin Provides Program Update on Vosoritide in Achondroplasia” on Apr 20, 2016
Finally, at the conference, the key takeways of vosoritide are summarized below
Vosoritide for Achondroplasia
- Durable effect demonstrated at 12 months with 15 μg/kg dose, confirming prior 6 month data
- New 6 month data on 12 addisonal patients at 15 μg/kg dose consistent with prior results
- Initial safety profile at 30 μg/kg/ dose similiar to 15 μg/kg
- Expect to start Phase 3 trial by the year
Source : The presentation at R&D day on April 20th in NewYork
はじめまして
子供が栄護君と同じ病気と分かってから
こちらのブログで勇気を頂いています。
新薬の効果が出ているとのこと、
前向きな気持ちになれますね！
情報発信して頂いて心強いです。
有り難うございます。
たなかさん、はじめまして。
本件は、2015年6月に発表された半年間の投与結果からのアップデートです。
1年間においても良好な結果がでました。
私の発信に関して、色んなご意見があるようですが、ACHのお子さんを持つ家族に勇気や希望を提示することが本旨ですのでコメントを嬉しく拝読させて頂きました。