Beyond Achondroplasia

Growing together with Clara

BMN-111 Phase 3 ancillary cohort

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BioMarin press release 3rd August 2015

“A major event in the second quarter of BioMarin Pharmaceutical Inc. was the completion and results of the Phase 2 study with vosoritide (future BMN-111 commercial designation) for the treatment of achondroplasia. BioMarin related that vosoritide increased the mean annualized growth velocity by 50% in children in the highest dose cohort who received 15 micrograms per kilogram daily. BioMarin believes growth velocity is a leading indicator of improvement in many of the severe complications associated with the disorder, such as foramen magnum compression, sleep apnea, bowed legs, spinal stenosis, recurrent ear infections and obesity.  Based on efficacy and safety observed in the first 3 cohorts of the Phase 2 study BioMarin is planning for registration enabling studies at 15 micrograms per kilogram daily and have also begun enrollment in a 4th higher dose cohort with 30 micrograms per kilogram daily.”

Excerpt from the BioMarin Investors page

BioMarin is going to start an ancillary phase with a double dose of the one with better results (15mg/kg) with new children (number unknown at the moment).

In pre-clinical studies, monkeys received doses up to 40mg/kg.

If the next phase 3 study’s outcomes are good and with minimum side effects, BMN-111 can be approved by FDA and EMA for administration in children with achondroplasia under puberty age (supposedly between 13 to 17 years-old).

The label for Vosoritide probably will be similar to “Treatment for the complications of achondroplasia”, and not an exclusive label for growth.

Remember that the Phase 2 is to assess safety and, in a positive way, BioMarin reported not just safety but also reported growth in the partial report.

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