Biomarin released today the results of BMN-111 (now vosoritide) phase 2 study in children with achondroplasia.
In summary, the drug showed no relevant safety issues in all doses tested. Moreover, in terms of efficacy, as the developer has been emphasizing lately in public financial meetings, the largest dose (15mcg/kg/day) induced a 50% increase in growth velocity (Table 1).
| Table 1: BMN 111 (vosoritide) Summary of Efficacy Results from Phase 2 Study in Children with Achondroplasia | |||
| Efficacy Analysis: Annualized 6-Months Growth Velocity | |||
| Cohort 1 | Cohort 2 | Cohort 3 | |
| Growth Velocity | 2.5 µg/kg/daily | 7.5 µg/kg/daily | 15 µg/kg/daily |
| (n=8*) | (n=8) | (n=10) | |
| Baseline | |||
| Mean (cm/Year) | 3.8 | 2.9 | 4.0 |
| Post-Treatment | |||
| Mean (cm/year) | 3.4 | 4.2 | 6.1 |
| Change from Baseline | |||
| Mean (cm/year) | -0.4 | 1.3 | 2.0 |
| 95% Confidence Interval (cm/year) | -1.8, 1.1 | 0.1, 2.5 | 0.6, 3.4 |
| p-value** | 0.56 | 0.04 | 0.01 |
| Percent increase from Baseline | |||
| Based on means (%) | NM | 45 | 50 |
** p-value, provided for descriptive purposes and based on the paired t-test comparing post-treatment GV and baseline GV, not adjusted for multiple comparisons. From Biomarin's press release, available here.
What does it mean this 50% increase in growth velocity ?
Historical data show that normal pre-pubertal children grow an average of 6 cm/year. As we see in the table above, the average growth velocity in achondroplasia before the therapy was 4 cm. As we have reviewed in the last article of the blog, the expectations were that the drug could lead to an increase in the growth velocity that would put children under therapy in a rate closer or similar to the normal growth velocity average.
What are the next steps?
Biomarin announced that they are now looking forward to work with FDA to start the pivotal registration study (phase 3) later this year. They keep planning a fourth cohort with a larger dose of vosoritide (30mcg/kg/day). They also mentioned the will to perform a study in younger children.
You can read the press release here. You can also listen to the recorded audio conference, available here.
Historical data show that normal pre-pubertal children grow an average of 6 cm/year. As we see in the table above, the average growth velocity in achondroplasia before the therapy was 4 cm. As we have reviewed in the last article of the blog, the expectations were that the drug could lead to an increase in the growth velocity that would put children under therapy in a rate closer or similar to the normal growth velocity average.
What are the next steps?
Biomarin announced that they are now looking forward to work with FDA to start the pivotal registration study (phase 3) later this year. They keep planning a fourth cohort with a larger dose of vosoritide (30mcg/kg/day). They also mentioned the will to perform a study in younger children.
You can read the press release here. You can also listen to the recorded audio conference, available here.
Dr. Morrys reciba un gran saludo.
ReplyDeleteLas noticias son muy buenas y esperanzadoras.
Seguimos muy atentos a los siguientes pasos.
Ojalá se abran pruebas en nuestros países con niños de menor edad.
Qué opina de las pruebas que se quieren hacer con una dosis mayor, considera con los datos que podría devolver el atraso en el crecimiento ?
Un gran abrazo !
Un gran abrazo
Dr. Morrys como esta.
ReplyDeleteMil gracias por toda su información tan buena y positiva.
Estamos muy felices y nos sentimos bendecidos por el futuro que viene.
Nos gustaría seber con el conocimiento que tienes de Biomarin que debemos hacer para tratar que en la nueva fase de pruebas pueda ser incluido Colombia.
De igual forma queremos que Biomarin conozca de nuestro caso y que estamos dispuestos a que nuestro bebe Martin, que actualmente tiene ocho meses, pueda hacer parte de las pruebas de fase III, si no es factible en Colombia en los Estados Unidos, ya que si somos seleccionados nos trasladaríamos para allá.
Mil gracias por tu ayuda y consejo que nos puedas brindar.
Un abrazo.
Alvaro,
DeleteEl blog y todo el trabajo de divulgación es independiente. No tengo acceso directo a información de los estudios, solamente lo que esta públicamente accesible. Es muy dificil saber donde los estudios futuros serán realizados. Puede-se deducir que los centros actualmente activos estarán haciendo los nuevos estudios tambíen. Para conocerlos, debe visitar sus paginas en la internet (en clinicaltrials.gov, use el enlace en el canto derecho en esta pagina) o entonces hacer contacto directo con Biomarin, a través de la pagina del laboratorio (http://www.bmrn.com/patients-physicians/bpps.php). Saludos.