VOSORITIDE

Description
Sources: https://pubmed.ncbi.nlm.nih.gov/34694597|https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214938s000lbl.pdf|https://www.ema.europa.eu/en/documents/product-information/voxzogo-epar-product-information_en.pdf

Vosoritide (VOXZOGO®; BMN-111) is a modified recombinant human C-type natriuretic peptide (CNP) analogue, being developed by BioMarin Pharmaceutical for the treatment of achondroplasia. Achondroplasia is caused by a gain-of-function mutation in the fibroblast growth factor receptor 3 gene (FGFR3), which is a negative regulator of bone growth. Vosoritide acts to restore chondrogenesis through its binding to natriuretic peptide receptor B (NPR-B), resulting in the inhibition of downstream signalling pathways of the overactive FGFR3 gene. Vosoritide was approved in August 2021 in the EU for the treatment of achondroplasia in patients aged ≥ 2 years whose epiphyses are not closed; the diagnosis of achondroplasia should be confirmed by appropriate genetic testing. In November 2021, Voxzogo was also approved by FDA to improve growth in children with achondroplasia.

Originator

Approval Year

Targets

Primary Target	Pharmacology	Condition	Potency
Atrial natriuretic peptide receptor B 2 Target ID: CHEMBL1795 Sources: https://pubmed.ncbi.nlm.nih.gov/25650377	Agonist 2752		4.9 nM [EC50]

Conditions

Condition	Modality	Targets	Highest Phase	Product
Achondroplasia 1 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214938s000lbl.pdf	Primary		Approved 8583	Voxzogo Approved Use Indicated to increase linear growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses. Launch Date 2021

C_max

Value	Dose	Analyte	Population
749 pg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	2.5 μg/kg single, subcutaneous dose: 2.5 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
1820 pg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	5 μg/kg single, subcutaneous dose: 5 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
6750 pg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	10 μg/kg single, subcutaneous dose: 10 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
8830 pg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	15 μg/kg single, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
749 pg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	2.5 μg/kg 1 times / day multiple, subcutaneous dose: 2.5 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
804 pg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	2.5 μg/kg 1 times / day multiple, subcutaneous dose: 2.5 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
736 pg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	2.5 μg/kg 1 times / day multiple, subcutaneous dose: 2.5 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
1410 pg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	5 μg/kg 1 times / day multiple, subcutaneous dose: 5 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
1420 pg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	5 μg/kg 1 times / day multiple, subcutaneous dose: 5 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
1400 pg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	5 μg/kg 1 times / day multiple, subcutaneous dose: 5 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
549 pg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=197	2.5 μg/kg single, subcutaneous dose: 2.5 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
1360 pg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=197	7.5 μg/kg single, subcutaneous dose: 7.5 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
4750 pg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=197	15 μg/kg single, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
16800 pg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=197	30 μg/kg single, subcutaneous dose: 30 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
7.18 mg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=208	15 μg/kg 1 times / day multiple, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
4.71 mg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=208	15 μg/kg 1 times / day multiple, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
6.52 mg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=208	15 μg/kg 1 times / day multiple, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
5.72 mg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=208	15 μg/kg 1 times / day multiple, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
5.8 mg/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=208	15 μg/kg 1 times / day multiple, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN

AUC

Value	Dose	Analyte	Population
129 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	2.5 μg/kg single, subcutaneous dose: 2.5 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
83.2 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	5 μg/kg single, subcutaneous dose: 5 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
607 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	10 μg/kg single, subcutaneous dose: 10 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
687 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	15 μg/kg single, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
129 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	2.5 μg/kg 1 times / day multiple, subcutaneous dose: 2.5 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
63.8 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	2.5 μg/kg 1 times / day multiple, subcutaneous dose: 2.5 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
110 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	2.5 μg/kg 1 times / day multiple, subcutaneous dose: 2.5 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
74.2 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	5 μg/kg 1 times / day multiple, subcutaneous dose: 5 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
83 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	5 μg/kg 1 times / day multiple, subcutaneous dose: 5 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
69.5 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	5 μg/kg 1 times / day multiple, subcutaneous dose: 5 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
1.37 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=197	2.5 μg/kg single, subcutaneous dose: 2.5 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
32.2 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=197	7.5 μg/kg single, subcutaneous dose: 7.5 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
175 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=197	15 μg/kg single, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
689 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=197	30 μg/kg single, subcutaneous dose: 30 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
242 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=208	15 μg/kg 1 times / day multiple, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
161 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=208	15 μg/kg 1 times / day multiple, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
256 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=208	15 μg/kg 1 times / day multiple, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
205 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=208	15 μg/kg 1 times / day multiple, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
290 ng × min/mL OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=208	15 μg/kg 1 times / day multiple, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN

T_1/2

Value	Dose	Analyte	Population
53.8 min OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	10 μg/kg single, subcutaneous dose: 10 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
69.5 min OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=195	15 μg/kg single, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
24.4 min OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=197	15 μg/kg single, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
27 min OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=197	30 μg/kg single, subcutaneous dose: 30 μg/kg route of administration: Subcutaneous experiment type: SINGLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
21 min OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=208	15 μg/kg 1 times / day multiple, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
26.6 min OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=208	15 μg/kg 1 times / day multiple, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN
27.9 min OTHER GOV'T https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=208	15 μg/kg 1 times / day multiple, subcutaneous dose: 15 μg/kg route of administration: Subcutaneous experiment type: MULTIPLE co-administered:	VOSORITIDE plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: UNKNOWN food status: UNKNOWN

Doses

Dose	Population	Adverse events
15 ug/kg 1 times / day multiple, subcutaneous Studied dose Dose: 15 ug/kg, 1 times / day Route: subcutaneous Route: multiple Dose: 15 ug/kg, 1 times / day Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf	unhealthy, CHILD Health Status: unhealthy Age Group: CHILD Sex: M+F Food Status: UNKNOWN Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf	Disc. AE: Anxiety, Pain related injections... AEs leading to discontinuation/dose reduction: Anxiety (1 pt) Pain related injections (1 pt) Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf
15 ug/kg/day 1 times / day multiple, subcutaneous Studied dose Dose: 15 ug/kg/day, 1 times / day Route: subcutaneous Route: multiple Dose: 15 ug/kg/day, 1 times / day Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000OtherR.pdf#page=98	unhealthy, CHILD Health Status: unhealthy Age Group: CHILD Sex: M+F Food Status: UNKNOWN Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000OtherR.pdf#page=98	Disc. AE: Adverse event... AEs leading to discontinuation/dose reduction: Adverse event (1 pt) Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000OtherR.pdf#page=98
30 ug/kg/day 1 times / day multiple, subcutaneous Studied dose Dose: 30 ug/kg/day, 1 times / day Route: subcutaneous Route: multiple Dose: 30 ug/kg/day, 1 times / day Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf	unhealthy, CHILD Health Status: unhealthy Age Group: CHILD Sex: M+F Food Status: UNKNOWN Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf	Disc. AE: Adverse event... AEs leading to discontinuation/dose reduction: Adverse event (5 patients) Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf

AEs

AE	Significance	Dose	Population
Anxiety	1 pt Disc. AE	15 ug/kg 1 times / day multiple, subcutaneous Studied dose Dose: 15 ug/kg, 1 times / day Route: subcutaneous Route: multiple Dose: 15 ug/kg, 1 times / day Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf	unhealthy, CHILD Health Status: unhealthy Age Group: CHILD Sex: M+F Food Status: UNKNOWN Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf
Pain related injections	1 pt Disc. AE	15 ug/kg 1 times / day multiple, subcutaneous Studied dose Dose: 15 ug/kg, 1 times / day Route: subcutaneous Route: multiple Dose: 15 ug/kg, 1 times / day Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf	unhealthy, CHILD Health Status: unhealthy Age Group: CHILD Sex: M+F Food Status: UNKNOWN Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf
Adverse event	1 pt Disc. AE	15 ug/kg/day 1 times / day multiple, subcutaneous Studied dose Dose: 15 ug/kg/day, 1 times / day Route: subcutaneous Route: multiple Dose: 15 ug/kg/day, 1 times / day Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000OtherR.pdf#page=98	unhealthy, CHILD Health Status: unhealthy Age Group: CHILD Sex: M+F Food Status: UNKNOWN Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000OtherR.pdf#page=98
Adverse event	5 patients Disc. AE	30 ug/kg/day 1 times / day multiple, subcutaneous Studied dose Dose: 30 ug/kg/day, 1 times / day Route: subcutaneous Route: multiple Dose: 30 ug/kg/day, 1 times / day Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf	unhealthy, CHILD Health Status: unhealthy Age Group: CHILD Sex: M+F Food Status: UNKNOWN Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf

Overview

CYP3A4	CYP2C9	CYP2D6	hERG
inducer 1087 inhibitor 2252	inhibitor 2438	inhibitor 2507	inhibitor 2217

OverviewOther

Other Inhibitor	Other Substrate	Other Inducer
CYP3A5 136 CYP1A2 2153 CYP2B6 926 CYP2C8 1057 CYP2C19 2057	NEP 1 CYT 1	CYP1A2 832 CYP2B6 669 CYP3A5 92

Drug as perpetrator

Target	Modality	Activity
CYP1A2 2333	inducer 1966 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=40 Page: 40 \| 193	no
CYP1A2 2333	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=40 Page: 40.0	no
CYP2B6 1160	inducer 1966 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=40 Page: 40 \| 193	no
CYP2B6 1160	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=40 Page: 40 \|193	no
CYP2C19 2141	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=40 Page: 40 \| 193	no
CYP2C8 1117	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=40 Page: 40 \| 193	no
CYP2C9 2497	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=40 Page: 40 \| 193	no
CYP2D6 2546	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=40 Page: 40 \| 193	no
CYP3A4 2633	inducer 1966 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=40 Page: 40 \| 193	no
CYP3A4 2633	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=40 Page: 40 \| 193	no
CYP3A5 201	inducer 1966 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=40 Page: 40 \| 193	no
CYP3A5 201	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=40 Page: 40 \| 193	no

Drug as victim

Target	Modality	Activity	Metabolite	Clinical evidence
NEP 1	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=142 Page: 142.0	major
CYT 1	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=40 Page: 40.0	no

Tox targets

Target	Modality	Activity	Metabolite	Clinical evidence
hERG 2263	inhibitor 2237 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf#page=38 Page: 38 \| 102 \| 139

PubMed

Title	Date	PubMed

Sample Use Guides

In Vivo Use Guide

The recommended dosage of VOXZOGO is based on the patient’s actual body weight. Inject VOXZOGO at approximately the same time each day, if possible. The volume of VOXZOGO to be administered (injection volume) is based on the patient's actual body weight and the concentration of reconstituted VOXZOGO (0.8 mg/mL or 2 mg/mL). VOXZOGO must be reconstituted prior to use. For example, for body weight 44-59 kg: 0.6 mg/0.3 ml (vial strength for reconstitution: 1.2 mg)

Route of Administration: Other

In Vitro Use Guide

Vosoritide showed a half maximal effective concentration (EC50) of 11 nM in the 3T3 cell NPR-B guanylate cyclase assay.

Substance Class	Protein Created by `admin` on `Mon Mar 31 21:55:41 GMT 2025` Edited by `admin` on `Mon Mar 31 21:55:41 GMT 2025`
Protein Sub Type
Sequence Type	COMPLETE
Record UNII	7SE5582Q2P
Record Status	`Validated (UNII)`
Record Version

Download

Name

Type

Language

vosoritide [INN]

Preferred Name

English

VOSORITIDE

Official Name

English

VOSORITIDE [JAN]

Common Name

English

PRO-GLY-GLN-GLU-HIS-PRO-ASN-ALA-ARG-LYS-TYR-LYS-GLY-ALA-ASN-LYS-LYS-GLY-LEU-SER-LYS-GLY-CYS-PHE-GLY-LEU-LYS-LEU-ASP-ARG-ILE-GLY-SER-MET-SER-GLY-LEU-GLY-CYS 23-39 DISULFIDE

Common Name

English

VOSORITIDE [USAN]

Common Name

English

Vosoritide [WHO-DD]

Common Name

English

BMN-111

Code

English

VOXZOGO

Brand Name

English

MODIFIED RECOMBINANT HUMAN C-TYPE NATRIURETIC PEPTIDE (CNP)

Common Name

English

BMN 111 [WHO-DD]

Common Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

386012