Expert Interview
Checking in with a prescriber of Voxzogo (vosoritide) in Achondroplasia, as the first FDA approved drug to improve growth in children with most common form of dwarfism
Ticker(s): BMRN, ASNDInstitution: University Hospital Cologne
- Head of the Center for Rare Skeletal Disorders in Children and Adolescents at the University of Cologne & member of the Osteogenesis Imperfecta Federation Europe (OIFE) Medical Advisory Board
- Treats 52 patients with Achondroplasia older than 5 years of age and is currently prescribing Voxzogo to 26 patients.
- Specializes in the research of brittle bone disease and directs the special ambulance for rare pediatric skeletal diseases and pediatric rheumatology.
Please tell us about your clinical experience. How many patients with Achondroplasia do you see on a yearly basis? What is the standard of care, and what novel therapies are you excited about for the near future?
Added By: sara_adminHow often are any of the following procedures undertaken:-Spinal fusion to stabilize the spine- Spinal decompression to free up the compressed spinal cord or nerve roots -Guided-growth surgery/osteotomy to correct uneven growth or abnormal rotation of the bones.
Added By: sara_adminWhat has been your clinical experience and feedback in terms of improved patient growth after using Voxzogo?
Added By: sara_adminAre there other potential forms dwarfism where Voxzogo may be used as treatment?
Added By: sara_adminWhat percent of your patients have been inclined to try Voxzogo? And how well do you think patients would comply with a years-long, daily, injectable schedule?
Added By: sara_adminWhat are the potential risks associated with achondroplasia? How well do patients understand achondroplasia, and what percent of patients agree with treatment?
Baseline mean AGV in the placebo and Voxzogo groups was 4.06 cm/year and 4.26 cm/year, respectively. At week 52, the change from baseline in AGV was -0.17 cm/year for the placebo treated patients and 1.40 cm/year for the Voxzogo treated patients, resulting in a 1.57 cm/year improvement in AGV for Voxzogo patients. Could you discuss these results, how much of a positive impact would it have on the patients’ long term outcomes? How much would complications and need for surgery be reduced?
Added By: sara_adminOn a scale of 1-10, how would you rate your level of excitement for the future of this drug?
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