March 16 (Reuters) - BioMarin Pharmaceutical ( BMRN )
will discontinue mid-stage trials of its bone disorder treatment
after reports of several cases of patients' hip joint slipping
out of place, the drugmaker said on Monday, sending its shares
down 4%.
* BioMarin was testing its drug Voxzogo in patients with
Turner Syndrome, SHOX-deficiency and Aggrecan (ACAN)-deficiency,
which are distinct genetic conditions that causes severe short
stature and in some cases abnormal bone development.
* Turner syndrome occurs in females missing part or all of
an X chromosome, SHOX deficiency results from a missing or
faulty growth-related gene, and ACAN deficiency is caused by
mutations in a gene important for cartilage structure.
* During the mid-stage trials, several patients faced
slipped capital femoral epiphysis, where the ball of the hip
joint slips off the thigh bone at the area where the bone is
still growing.
* BioMarin said it will continue testing the drug in
patients with Noonan syndrome where typical development stops in
various parts of the body, and in patients with a form of
unexplained short stature.
* The U.S. Food and Drug Administration approved the
injection Voxzogo in 2021 for patients with achondroplasia, a
form of dwarfism.
* Last month, the FDA approved Ascendis Pharma's ( ASND )
once-weekly injection Yuviwel, making it the second treatment
for achondroplasia to gain U.S. approval.