Achondroplasia causes short stature. A phase 3 trial working to evaluate the effectiveness of vosoritide will be completed by the end of next year (2019). The study includes patients aged 5-14 and will span 52 weeks in total. Earlier studies of the drug showed an increased growth of 6cm in patients who were treated for 42 months.
There is also a phase 2 trial going on focused on children under the age of 5. This study also includes infants.
Current treatments for achondroplasia can cause increased body disproportion in the limbs. Achondroplasia is caused by a FGFR3 mutation which blocks cells from developing. Vosoritide should reduce the effects of the overactive FGFR3 without causing an increase in disproportion.
BioMarin has been working on a treatment for hemophilia A. This type of the condition is caused a deficiency of clotting factor VIII. Their research uses a viral vector to deliver factor VIII. The trial is currently phase 1/2. Preliminary results showed a decrease in annual bleed rates of 97%! Preliminary results also indicated that most patients stopped requiring preventive injections of the factor after treatment.
Their goal is to file for FDA approval of the treatment, called valrox, in the second half of 2019. The company says they have been meeting with the FDA regularly and their confidence in this treatment is high.
These updates are promising for both the achondroplasia and hemophilia community! Stay tuned to hear how these clinical trials play out. We’re all rooting for FDA approval!
You can read more about these two clinical trials by clicking here!