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BioMarin Pharmaceutical Inc. — Rare Disease Pipeline | Kisho | Kisho

BioMarin Pharmaceutical Inc.

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This biopharmaceutical company develops therapies for rare diseases, with 8 FDA-approved drugs and 25 orphan drug designations across 48 conditions, including hemophilia A and phenylketonuria.

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
Becker muscular dystrophy	Exon 52 specific phosphorothiate oligonucleotideExon 55 specific phosphorothioate oligonucleotideexon 53 specific phosphorothioate oligonucleotideExon 45 specific phosphorothioate oligonucleotideExon 44 specific phosphorothioate oligonucleotide	Orphan Designation	-
Bicervical bicornuate uterus with patent cervix and vagina	Vimizim	Orphan Designation	-
Duchenne muscular dystrophy	fully modified 18-mer oligonucleotide with a TEG modification at its 5' end that binds to exon 51 of dystrophin pre-mRNAExon 52 specific phosphorothiate oligonucleotideExon 55 specific phosphorothioate oligonucleotideexon 53 specific phosphorothioate oligonucleotideExon 45 specific phosphorothioate oligonucleotideExon 44 specific phosphorothioate oligonucleotideExon 51 specific phosphorothioate oligonucleotide	Orphan Designation	-
Hunter-McAlpine craniosynostosis	Aldurazyme	Orphan Designation	-
Huntington disease	Brineura	Orphan Designation	-
Noonan syndrome 1	vosoritide	Orphan Designation	-
Noonan syndrome 4	vosoritide	Orphan Designation	-
Noonan syndrome 8	vosoritide	Orphan Designation	-
Noonan syndrome 9	vosoritide	Orphan Designation	-
Noonan syndrome-like disorder with loose anagen hair 2	non-replicating AAV-based gene therapy vector with a single stranded DNA genome engineered to carry an expression cassette encoding for human cardiac myosin binding protein-C (cMyBP-C; hMYBPC3 transgene)	Orphan Designation	-
Toriello-Lacassie-Droste syndrome	non-replicating AAV-based gene therapy vector with a single stranded DNA genome engineered to carry an expression cassette encoding for human cardiac myosin binding protein-C (cMyBP-C; hMYBPC3 transgene)	Orphan Designation	-
achondroplasia	Voxzogo	Orphan Designation	-
acquired adult-onset immunodeficiency	Roctavian	Orphan Designation	-
adult embryonal tumor with multilayered rosettes, c19mc-altered	Roctavian	Orphan Designation	-
alpha 1-antitrypsin deficiency	oxoindoline carboxamide compound	Orphan Designation	-
carbon monoxide-induced delayed encephalopathy	VimizimAldurazyme	Orphan Designation	-
cardiofaciocutaneous syndrome 1	Brineura	Orphan Designation	-
cardiomyopathy, familial hypertrophic, 31	Naglazyme	Orphan Designation	-
glycogen storage disease II	reveglucosidase alfa	Orphan Designation	-
glycogen storage disease IXb	Voxzogo	Orphan Designation	-
glycogen storage disease VI	Voxzogo	Orphan Designation	-
hemophilia A	Roctavian	Orphan Designation	FDA Approved
hereditary angioedema	adeno-associated virus serotype 5 (AAV5) vector containing the hSERPING cDNA sequence encoding human C1-esterase inhibitor (C1-INH)	Orphan Designation	-
hyperphenylalaninemia due to tetrahydrobiopterin deficiency	KuvanPALYNZIQ	Orphan Designation	-
hypochondroplasia	vosoritide	Orphan Designation	-
microcephaly-intellectual disability-sensorineural hearing loss-epilepsy-abnormal muscle tone syndrome	Brineura	Orphan Designation	-
mild hyperphenylalaninemia	Kuvan	Orphan Designation	-
mitochondrial DNA depletion syndrome 6 (hepatocerebral type)	BrineuraVoxzogo	Orphan Designation	-
monosomy X	Voxzogo	Orphan Designation	-
mosaic monosomy X	vosoritideVoxzogo	Orphan Designation	-
mucopolysaccharidosis	Naglazyme	Orphan Designation	-
mucopolysaccharidosis type 6	Naglazyme	Orphan Designation	-
mucopolysaccharidosis type 6, rapidly progressing	Naglazyme	Orphan Designation	-
mucopolysaccharidosis type 6, slowly progressing	Naglazyme	Orphan Designation	-
mucopolysaccharidosis type 7	Vimizim	Orphan Designation	-
mucopolysaccharidosis-plus syndrome	VimizimAldurazyme	Orphan Designation	-
multisystemic smooth muscle dysfunction syndrome	VimizimAldurazyme	Orphan Designation	-
myofibrillar myopathy 8	Naglazyme	Orphan Designation	-
natal teeth-intestinal pseudoobstruction-patent ductus syndrome	VimizimAldurazyme	Orphan Designation	-
neonatal encephalomyopathy-cardiomyopathy-respiratory distress syndrome	non-replicating AAV-based gene therapy vector with a single stranded DNA genome engineered to carry an expression cassette encoding for human cardiac myosin binding protein-C (cMyBP-C; hMYBPC3 transgene)	Orphan Designation	-
neuronal ceroid lipofuscinosis 2	Brineura	Orphan Designation	-
nut midline carcinoma	Roctavian	Orphan Designation	-
palmoplantar keratoderma-sclerodactyly syndrome	Aldurazyme	Orphan Designation	-
phenylketonuria	adeno-associated virus vector encoding human phenylalanine hydroxylase	Orphan Designation	-
phenylketonuria	Kuvan	Orphan Designation	-
primary hyperoxaluria type 1	5-[[trans-4-[(trifluoromethoxy)phenyl]cyclohexyl]oxy]-1H-1,2,3-triazole-4-carboxylic acid	Orphan Designation	-
symptomatic form of muscular dystrophy of Duchenne and Becker in female carriers	Exon 52 specific phosphorothiate oligonucleotideExon 55 specific phosphorothioate oligonucleotideexon 53 specific phosphorothioate oligonucleotideExon 45 specific phosphorothioate oligonucleotide	Orphan Designation	-
tetrahydrobiopterin-responsive hyperphenylalaninemia/phenylketonuria	Kuvan	Orphan Designation	-

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