ID	DDInter2202
	Drug Type	biotech
	Protein Chemical Formula	C176H290N56O51S3
	Protein Average Weight	4100.000
	CAS Number	1480724-61-5
	Description	Achondroplasia is an autosomal dominant genetic disease and the most common cause of dwarfism in humans.[A242277] It results from a gain-of-function missense mutation in _FGFR3_ that results in a dramatic suppression of bone growth, both in volume and in length.[A242273,A242277] Treatment for achondroplasia includes both surgical and pharmacological interventions, the latter of which includes C-type natriuretic peptide (CNP) analogs. Endogenous CNP, first described in 1998, is primarily responsible for the stimulation of chondrocytes and long bone growth via activity at the NPR-B receptor, making it an attractive target in the treatment of a condition like achondroplasia.[A242273] While the remarkably short half-life of endogenous CNP - 2 to 3 minutes due to its rapid degradation by endopeptidases - makes it ineffective as a therapeutic intervention,[A242273] the development of a peptidase-resistant formulation has allowed for its use as a viable treatment option in achondroplasia. Vosoritide is an analog of CNP with proline-glycine on its N-terminus to convey resistance to neutral endopeptidase.[L39229] It was approved for use under the brand name Voxzogo (BioMarin Pharmaceutical Inc.) in the EU in August 2021 and the US in November 2021,[L39245,L39241] becoming the first pharmacological intervention approved for the treatment of achondroplasia in both regions.
	ATC Classification	M05BX07
Sequences	None
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