Vasoritide is a growth hormone drug . It is a new drug & approved by FDA in 25Nov 2021

1. VOSORITIDE
Dr. Pratap kumar sahoo
Professor
Presented by –Sandeep Kumar Kanungo
Regd NO-2161613003
Sikshya
‘O’
Anusandhan University,Bhubaneswar

2. INTRODUCTION
• vosoritide is a new drug indicated to
increase linear growth in pediatric
patients with achondroplasia who are 5
years of age.
• Achondroplasia is an autosomal
dominant genetic disorder that is a
primary skeletal dysplasia whose main
feature is dwarfism.
• It is developed by BioMarin
Pharmaceutical.
• It was approved in November 2021 by
the U.S. Food and Drug Administration.

4. MECHANISM
OF ACTION
• Vosoritide works by binding to a receptor
(target) called natriuretic peptide receptor
type B (NPR-B), which reduces the activity
of fibroblast growth factor
receptor (FGFR3).
• FGFR3 is a receptor that normally down-
regulates cartilage and bone growth when
activated by one of the proteins known
as acidic and basic fibroblast growth factor.
• It does so by inhibiting the development
(cell proliferation and differentiation)
of chondrocytes, the cells which is also
necessary for bone growth.

5. MECHANISM
OF ACTION
• Children with achondroplasia have one
of several possible FGFR3 mutations
resulting in overall reduced and bone
growth.
• The protein C-type natriuretic
peptide (CNP), naturally found in
humans, reduces the effects of over-
active FGFR3.
• Vosoritide is a CNP analogue with the
same effect but prolonged half-
life,allowing for once-daily administration

6. USE & ITS
SIDE EFFECT
• Vosoritide is a used to increase linear growth in
children with achondroplasia who are 5 years of age
and older with open growth plates (epiphyses).
• Vosoritide may cause serious side effects including a
temporary decrease in blood pressure in some
patients.
• To reduce the risk of a decrease in blood pressure
and associated symptoms (dizziness, feeling tired,
or nausea), patients should eat a meal and drink 8
to 10 ounces of fluid within 1 hour before receiving
the drug.

7. DOSAGE & ITS ADMINISTRATION
It is given in form of injection,lyophilized
powder for reconstitution for the treatment
of children with achondroplasia, the most
common form of human dwarfism.
It is administered in the subcutaneous route
which treats achondroplasia, a condition
that impairs bone growth.

8. RESULT
In study A between 30 and 42 months of treatment, children who took
15 micrograms per kilogram of vosoritide had an average growth rate
of 5.5 cm per year (this includes natural growth + vosoritide effect).
• Vosoritide increased growth rate by roughly 1.5 cm per year from the
start of the study.
• As this is an average, some children grew more and some grew less
than 1.5 cm per year.
• After 42 months of treatment, children with achondroplasia taking
vosoritide grew at a rate closer to children without achondroplasia,
who were of similar age and sex.

9. RESULT