abacavir
Antiinfectives for systemic use (J05)US regulatory status: approved.
pharma · Cystic Fibrosis · Multiple Sclerosis · RARE
Ultragenyx UK
Ultragenyx UK Limited
Ultragenyx UK drug portfolio — 168 indexed compounds with therapeutic class and molecular targets from public FDA and regional approval metadata.
Newcastle upon tyne, NE128RL, GB, NOVATO, CA HQ
2024 Founded
2 Employees
Public company Type
RARE · NYSE Ticker
Company details
- Status
- Public
- HQ
- Newcastle upon tyne, NE128RL, GB, NOVATO, CA
- Founded
- 2024
- Employees
- 2
- Programs
- 201
- Drugs
- 168
- Patents
- 23
Ultragenyx UK pipeline
Portfolio lens on 168 drugs and development assets — regulatory status, targets, and deep links to NovaPharmaNews drug profiles.
Ultragenyx UK drug portfolio — 168 indexed compounds with therapeutic class and molecular targets from public FDA and regional approval metadata.
Drug portfolio
-
-
US regulatory status: approved.
-
abatacept
Antineoplastic and immunomodulating agents (L04)US regulatory status: approved. Molecular target: T-lymphocyte activation antigen CD80.
- Target
- T-lymphocyte activation antigen CD80
- Mechanism
- T-lymphocyte activation antigen CD80 inhibitor
-
US regulatory status: approved. Molecular target: Integrin alpha-IIb/beta-3.
- Target
- Integrin alpha-IIb/beta-3
- Mechanism
- Integrin alpha-V/beta-3 inhibitor
-
US regulatory status: approved. Molecular target: Synaptosomal-associated protein 25.
- Target
- Synaptosomal-associated protein 25
- Mechanism
- Synaptosomal-associated protein 25 inhibitor
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
US regulatory status: approved. Molecular target: Carbonic anhydrase 1.
- Target
- Carbonic anhydrase 1
- Mechanism
- Carbonic anhydrase XII inhibitor
-
US regulatory status: approved. Mechanism: Glutathione precursor.
- Mechanism
- Glutathione precursor
-
US regulatory status: approved. Molecular target: DNA polymerase catalytic subunit.
- Target
- DNA polymerase catalytic subunit
- Mechanism
- Human herpesvirus 1 DNA polymerase inhibitor
-
US regulatory status: approved. Molecular target: DNA polymerase catalytic subunit.
- Target
- DNA polymerase catalytic subunit
- Mechanism
- Human herpesvirus 1 DNA polymerase inhibitor
-
allogeneic t cells genetically modified with a retroviral vector encoding for a truncated form of the human low affinity nerve growth factor receptor (δlngfr) and the herpes simplex i virus thymidine kinase (hsv-tk mut2)
Antineoplastic and immunomodulating agents (L01)Antineoplastic and immunomodulating agents (L01) compound in Ultragenyx UK's portfolio. Mechanism: Somatic cell supplemental therapy.
- Mechanism
- Somatic cell supplemental therapy
-
allopurinol
Musculo-skeletal system (M04)US regulatory status: approved. Molecular target: Xanthine dehydrogenase/oxidase.
- Target
- Xanthine dehydrogenase/oxidase
- Mechanism
- Xanthine dehydrogenase inhibitor
-
US regulatory status: approved. Molecular target: Adrenergic receptor alpha.
- Target
- Adrenergic receptor alpha
- Mechanism
- Adrenergic receptor alpha antagonist
-
US regulatory status: approved. Mechanism: Reactive metabolite scavenging agent.
- Mechanism
- Reactive metabolite scavenging agent
-
US regulatory status: approved. Molecular target: Dihydrofolate reductase.
- Target
- Dihydrofolate reductase
- Mechanism
- Dihydrofolate reductase inhibitor
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
arginine
Various (V03)Various (V03) compound in Ultragenyx UK's portfolio.
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
US regulatory status: approved. Molecular target: Cyclooxygenase.
- Target
- Cyclooxygenase
- Mechanism
- Cyclooxygenase inhibitor
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
atropine sulfate
Sensory organs (S01)US regulatory status: approved. Molecular target: Muscarinic acetylcholine receptor M1.
- Target
- Muscarinic acetylcholine receptor M1
- Mechanism
- Muscarinic acetylcholine receptor M3 antagonist
-
US regulatory status: approved. Mechanism: Unknown.
- Mechanism
- Unknown
-
US regulatory status: approved. Molecular target: GABA-B receptor.
- Target
- GABA-B receptor
- Mechanism
- GABA-B receptor agonist
-
US regulatory status: approved. Molecular target: Betaine-homocysteine S-methyltransferase.
- Target
- Betaine-homocysteine S-methyltransferase
- Mechanism
- Betaine-homocysteine S-methyltransferase substrate
-
US regulatory status: approved. Molecular target: Glucocorticoid receptor.
- Target
- Glucocorticoid receptor
- Mechanism
- Glucocorticoid receptor agonist
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
botulinum toxin type a
Musculo-skeletal system (M03)Musculo-skeletal system (M03) compound in Ultragenyx UK's portfolio. Molecular target: Synaptosomal-associated protein 25.
- Target
- Synaptosomal-associated protein 25
- Mechanism
- Synaptosomal nerve-associated protein 25 (SNAP-25) hydrolytic enzyme
-
Development asset linked to Ultragenyx UK in public FDA and regional records. Mechanism: D2-like dopamine receptor agonist.
- Mechanism
- D2-like dopamine receptor agonist
-
US regulatory status: approved. Molecular target: D2-like dopamine receptor.
- Target
- D2-like dopamine receptor
- Mechanism
- D2-like dopamine receptor agonist
-
US regulatory status: approved. Molecular target: Histamine H1 receptor.
- Target
- Histamine H1 receptor
- Mechanism
- Histamine H1 receptor antagonist
-
burosumab
Musculo-skeletal system (M05)Musculo-skeletal system (M05) compound in Ultragenyx UK's portfolio. Molecular target: Fibroblast growth factor 23.
- Target
- Fibroblast growth factor 23
- Mechanism
- Fibroblast growth factor 23 inhibitor
-
US regulatory status: approved. Molecular target: Fibroblast growth factor 23.
- Target
- Fibroblast growth factor 23
- Mechanism
- Fibroblast growth factor 23 inhibitor
-
US regulatory status: approved. Molecular target: Vitamin D3 receptor.
- Target
- Vitamin D3 receptor
- Mechanism
- Vitamin D receptor agonist
-
US regulatory status: approved.
-
US regulatory status: approved. Mechanism: Dihydrofolate reductase inhibitor.
- Mechanism
- Dihydrofolate reductase inhibitor
-
US regulatory status: approved. Molecular target: Sodium channel alpha subunit.
- Target
- Sodium channel alpha subunit
- Mechanism
- Sodium channel alpha subunit blocker
-
US regulatory status: approved. Mechanism: Unknown.
- Mechanism
- Unknown
-
US regulatory status: approved. Molecular target: Bacterial penicillin-binding protein.
- Target
- Bacterial penicillin-binding protein
- Mechanism
- Bacterial penicillin-binding protein inhibitor
-
characterised viable autologous cartilage cells expanded ex vivo expressing specific marker proteins
Musculo-skeletal system (M09)Musculo-skeletal system (M09) compound in Ultragenyx UK's portfolio. Mechanism: Somatic cell supplemental therapy.
- Mechanism
- Somatic cell supplemental therapy
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
cidofovir
Antiinfectives for systemic use (J05)US regulatory status: approved. Molecular target: DNA polymerase catalytic subunit.
- Target
- DNA polymerase catalytic subunit
- Mechanism
- Human herpesvirus 1 DNA polymerase inhibitor
-
US regulatory status: approved. Molecular target: DNA.
- Target
- DNA
- Mechanism
- DNA inhibitor
-
cladribine
Antineoplastic and immunomodulating agents (L04)US regulatory status: approved. Molecular target: DNA.
- Target
- DNA
- Mechanism
- DNA inhibitor
-
US regulatory status: approved. Molecular target: Estrogen receptor.
- Target
- Estrogen receptor
- Mechanism
- Estrogen receptor alpha modulator
-
colchicine
Musculo-skeletal system (M04)US regulatory status: approved. Molecular target: Tubulin.
- Target
- Tubulin
- Mechanism
- Tubulin inhibitor
-
US regulatory status: approved.
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
covid-19 vaccine (inactivated, adjuvanted, adsorbed)
Antiinfectives for systemic use (J07)Antiinfectives for systemic use (J07) compound in Ultragenyx UK's portfolio. Mechanism: Biological vaccine - induces immune response.
- Mechanism
- Biological vaccine - induces immune response
-
US regulatory status: approved. Molecular target: DNA.
- Target
- DNA
- Mechanism
- DNA inhibitor
-
US regulatory status: approved. Molecular target: Peptidyl-prolyl cis-trans isomerase A.
- Target
- Peptidyl-prolyl cis-trans isomerase A
- Mechanism
- Cyclophilin A modulator
-
cytarabine
Antineoplastic and immunomodulating agents (L01)US regulatory status: approved. Molecular target: DNA.
- Target
- DNA
- Mechanism
- DNA polymerase (alpha/delta/epsilon) inhibitor
-
US regulatory status: approved. Molecular target: Sodium-dependent noradrenaline transporter.
- Target
- Sodium-dependent noradrenaline transporter
- Mechanism
- Norepinephrine transporter inhibitor
-
US regulatory status: approved. Molecular target: Glucocorticoid receptor.
- Target
- Glucocorticoid receptor
- Mechanism
- Glucocorticoid receptor agonist
-
US regulatory status: approved. Molecular target: GABA-A receptor; anion channel.
- Target
- GABA-A receptor; anion channel
- Mechanism
- GABA-A receptor; anion channel positive allosteric modulator
-
US regulatory status: approved. Molecular target: Carbonic anhydrase 1.
- Target
- Carbonic anhydrase 1
- Mechanism
- Carbonic anhydrase XII inhibitor
-
diphtheria (d), tetanus (t), pertussis (acellular, component) (pa), hepatitis b (rdna) (hbv), poliomyelitis (inactivated) (ipv) and haemophilus influenzae type-b (hib) conjugate vaccine (adsorbed)
Antiinfectives for systemic use (J07)Antiinfectives for systemic use (J07) compound in Ultragenyx UK's portfolio.
-
diphtheria (d), tetanus (t), pertussis (whole cell) (pw) and hepatitis b (rdna) (hbv) vaccine (adsorbed)
Antiinfectives for systemic use (J07)Antiinfectives for systemic use (J07) compound in Ultragenyx UK's portfolio.
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
US regulatory status: approved. Molecular target: Adrenergic receptor.
- Target
- Adrenergic receptor
- Mechanism
- Adrenergic receptor agonist
-
Development asset linked to Ultragenyx UK in public FDA and regional records. Mechanism: Angiotensin-converting enzyme inhibitor.
- Mechanism
- Angiotensin-converting enzyme inhibitor
-
enalapril maleate
Cardiovascular system (C09)US regulatory status: approved. Molecular target: Angiotensin-converting enzyme.
- Target
- Angiotensin-converting enzyme
- Mechanism
- Angiotensin-converting enzyme inhibitor
-
Development asset linked to Ultragenyx UK in public FDA and regional records. Mechanism: Prostanoid IP receptor agonist.
- Mechanism
- Prostanoid IP receptor agonist
-
US regulatory status: approved. Molecular target: Prostacyclin receptor.
- Target
- Prostacyclin receptor
- Mechanism
- Prostanoid IP receptor agonist
-
US regulatory status: approved. Molecular target: Estrogen receptor.
- Target
- Estrogen receptor
- Mechanism
- Estrogen receptor agonist
-
US regulatory status: approved. Molecular target: Estrogen receptor.
- Target
- Estrogen receptor
- Mechanism
- Estrogen receptor alpha agonist
-
US regulatory status: approved. Molecular target: Estrogen receptor.
- Target
- Estrogen receptor
- Mechanism
- Estrogen receptor alpha agonist
-
evinacumab
Cardiovascular system (C10)Cardiovascular system (C10) compound in Ultragenyx UK's portfolio. Molecular target: Angiopoietin-related protein 3.
- Target
- Angiopoietin-related protein 3
- Mechanism
- Angiopoietin-related protein 3 inhibitor
-
US regulatory status: approved. Molecular target: Angiopoietin-related protein 3.
- Target
- Angiopoietin-related protein 3
- Mechanism
- Angiopoietin-related protein 3 inhibitor
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
ferumoxytol
Blood and blood forming organs (B03)US regulatory status: approved. Mechanism: Unknown.
- Mechanism
- Unknown
-
filgrastim
Antineoplastic and immunomodulating agents (L03)US regulatory status: approved. Molecular target: Granulocyte colony-stimulating factor receptor.
- Target
- Granulocyte colony-stimulating factor receptor
- Mechanism
- Granulocyte colony stimulating factor receptor agonist
-
US regulatory status: approved. Molecular target: Lanosterol 14-alpha demethylase.
- Target
- Lanosterol 14-alpha demethylase
- Mechanism
- Cytochrome P450 51 inhibitor
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
US regulatory status: approved. Molecular target: Glucocorticoid receptor.
- Target
- Glucocorticoid receptor
- Mechanism
- Glucocorticoid receptor agonist
-
ganciclovir
Antiinfectives for systemic use (J05)US regulatory status: approved. Molecular target: DNA polymerase catalytic subunit.
- Target
- DNA polymerase catalytic subunit
- Mechanism
- Human herpesvirus 1 DNA polymerase inhibitor
-
guanfacine
Cardiovascular system (C02)Cardiovascular system (C02) compound in Ultragenyx UK's portfolio. Mechanism: Adrenergic receptor alpha-2 agonist.
- Mechanism
- Adrenergic receptor alpha-2 agonist
-
US regulatory status: approved. Molecular target: Adrenergic receptor alpha-2.
- Target
- Adrenergic receptor alpha-2
- Mechanism
- Adrenergic receptor alpha-2 agonist
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
human coagulation factor viii / human von willebrand factor
Blood and blood forming organs (B02)Blood and blood forming organs (B02) compound in Ultragenyx UK's portfolio.
-
US regulatory status: approved. Molecular target: Solute carrier family 12 member 3.
- Target
- Solute carrier family 12 member 3
- Mechanism
- Thiazide-sensitive sodium-chloride cotransporter inhibitor
-
hydrocortisone
Systemic hormonal preparations (H02)US regulatory status: approved. Molecular target: Glucocorticoid receptor.
- Target
- Glucocorticoid receptor
- Mechanism
- Glucocorticoid receptor agonist
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
iloprost
Blood and blood forming organs (B01)US regulatory status: approved. Molecular target: Prostacyclin receptor.
- Target
- Prostacyclin receptor
- Mechanism
- Prostanoid IP receptor agonist
-
US regulatory status: approved. Molecular target: Sodium-dependent noradrenaline transporter.
- Target
- Sodium-dependent noradrenaline transporter
- Mechanism
- Serotonin transporter inhibitor
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
Development asset linked to Ultragenyx UK in public FDA and regional records. Molecular target: Insulin receptor.
- Target
- Insulin receptor
- Mechanism
- Insulin receptor agonist
-
interferon alfa-2b
Antineoplastic and immunomodulating agents (L03)US regulatory status: approved. Molecular target: Interferon alpha/beta receptor.
- Target
- Interferon alpha/beta receptor
- Mechanism
- Interferon alpha/beta receptor agonist
-
interferon beta-1a
Antineoplastic and immunomodulating agents (L03)US regulatory status: approved. Molecular target: Interferon alpha/beta receptor.
- Target
- Interferon alpha/beta receptor
- Mechanism
- Interferon alpha/beta receptor agonist
-
US regulatory status: approved. Molecular target: Lanosterol 14-alpha demethylase.
- Target
- Lanosterol 14-alpha demethylase
- Mechanism
- Cytochrome P450 51 inhibitor
-
US regulatory status: approved. Mechanism: Colonic bacteria substrate.
- Mechanism
- Colonic bacteria substrate
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
US regulatory status: approved.
-
Development asset linked to Ultragenyx UK in public FDA and regional records. Mechanism: Gonadotropin-releasing hormone receptor agonist.
- Mechanism
- Gonadotropin-releasing hormone receptor agonist
-
US regulatory status: approved. Molecular target: Gonadotropin-releasing hormone receptor.
- Target
- Gonadotropin-releasing hormone receptor
- Mechanism
- Gonadotropin-releasing hormone receptor agonist
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
US regulatory status: approved. Molecular target: Type-1 angiotensin II receptor.
- Target
- Type-1 angiotensin II receptor
- Mechanism
- Type-1 angiotensin II receptor antagonist
-
Development asset linked to Ultragenyx UK in public FDA and regional records. Molecular target: Progesterone receptor.
- Target
- Progesterone receptor
- Mechanism
- Progesterone receptor agonist
-
US regulatory status: approved. Molecular target: Progesterone receptor.
- Target
- Progesterone receptor
- Mechanism
- Progesterone receptor agonist
-
melphalan
Antineoplastic and immunomodulating agents (L01)US regulatory status: approved. Molecular target: DNA.
- Target
- DNA
- Mechanism
- DNA inhibitor
-
methotrexate
Antineoplastic and immunomodulating agents (L04)US regulatory status: approved. Molecular target: Dihydrofolate reductase.
- Target
- Dihydrofolate reductase
- Mechanism
- Dihydrofolate reductase inhibitor
-
US regulatory status: approved. Molecular target: DNA.
- Target
- DNA
- Mechanism
- DNA inhibitor
-
US regulatory status: approved. Molecular target: Glucocorticoid receptor.
- Target
- Glucocorticoid receptor
- Mechanism
- Glucocorticoid receptor agonist
-
US regulatory status: approved. Molecular target: Glucocorticoid receptor.
- Target
- Glucocorticoid receptor
- Mechanism
- Glucocorticoid receptor agonist
-
Development asset linked to Ultragenyx UK in public FDA and regional records. Mechanism: Dopamine D2 receptor antagonist.
- Mechanism
- Dopamine D2 receptor antagonist
-
US regulatory status: approved. Molecular target: Sodium channel alpha subunit.
- Target
- Sodium channel alpha subunit
- Mechanism
- Sodium channel alpha subunit blocker
-
Development asset linked to Ultragenyx UK in public FDA and regional records. Mechanism: Adrenergic receptor alpha-1 agonist.
- Mechanism
- Adrenergic receptor alpha-1 agonist
-
US regulatory status: approved. Molecular target: Adrenergic receptor alpha-1.
- Target
- Adrenergic receptor alpha-1
- Mechanism
- Adrenergic receptor alpha-1 agonist
-
miglustat
Alimentary tract and metabolism (A16)US regulatory status: approved. Molecular target: Ceramide glucosyltransferase.
- Target
- Ceramide glucosyltransferase
- Mechanism
- Ceramide glucosyltransferase inhibitor
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
octreotide
Systemic hormonal preparations (H01)Systemic hormonal preparations (H01) compound in Ultragenyx UK's portfolio. Mechanism: Somatostatin receptor agonist.
- Mechanism
- Somatostatin receptor agonist
-
US regulatory status: approved. Molecular target: Somatostatin receptor.
- Target
- Somatostatin receptor
- Mechanism
- Somatostatin receptor agonist
-
Development asset linked to Ultragenyx UK in public FDA and regional records. Mechanism: Cyclooxygenase inhibitor.
- Mechanism
- Cyclooxygenase inhibitor
-
US regulatory status: approved. Molecular target: Polyunsaturated fatty acid 5-lipoxygenase.
- Target
- Polyunsaturated fatty acid 5-lipoxygenase
- Mechanism
- Arachidonate 5-lipoxygenase inhibitor
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
orlistat
Alimentary tract and metabolism (A08)US regulatory status: approved. Molecular target: Gastric triacylglycerol lipase.
- Target
- Gastric triacylglycerol lipase
- Mechanism
- Pancreatic lipase inhibitor
-
osilodrostat
Systemic hormonal preparations (H02)Systemic hormonal preparations (H02) compound in Ultragenyx UK's portfolio. Mechanism: Cytochrome P450 11B1 inhibitor.
- Mechanism
- Cytochrome P450 11B1 inhibitor
-
US regulatory status: approved. Molecular target: Cytochrome P450 11B1, mitochondrial.
- Target
- Cytochrome P450 11B1, mitochondrial
- Mechanism
- Cytochrome P450 11B1 inhibitor
-
US regulatory status: approved. Molecular target: Fatty acids.
- Target
- Fatty acids
- Mechanism
- Fatty acids hydrolytic enzyme
-
pandemic influenza vaccine (h5n1) (live attenuated, nasal)
Antiinfectives for systemic use (J07)Antiinfectives for systemic use (J07) compound in Ultragenyx UK's portfolio. Mechanism: Biological vaccine - induces immune response.
- Mechanism
- Biological vaccine - induces immune response
-
US regulatory status: approved. Molecular target: Sodium-dependent noradrenaline transporter.
- Target
- Sodium-dependent noradrenaline transporter
- Mechanism
- Norepinephrine transporter releasing agent
-
US regulatory status: approved. Molecular target: Sodium channel alpha subunit.
- Target
- Sodium channel alpha subunit
- Mechanism
- Sodium channel alpha subunit blocker
-
US regulatory status: approved. Molecular target: Dopamine receptor.
- Target
- Dopamine receptor
- Mechanism
- Dopamine receptor antagonist
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
potassium citrate
Alimentary tract and metabolism (A12)US regulatory status: approved.
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
prasterone
Genito-urinary system and sex hormones (G03)US regulatory status: approved. Mechanism: Unknown.
- Mechanism
- Unknown
-
Development asset linked to Ultragenyx UK in public FDA and regional records. Mechanism: HMG-CoA reductase inhibitor.
- Mechanism
- HMG-CoA reductase inhibitor
-
US regulatory status: approved. Molecular target: Glucocorticoid receptor.
- Target
- Glucocorticoid receptor
- Mechanism
- Glucocorticoid receptor agonist
-
US regulatory status: approved. Molecular target: Glucocorticoid receptor.
- Target
- Glucocorticoid receptor
- Mechanism
- Glucocorticoid receptor agonist
-
US regulatory status: approved. Molecular target: Thyrotropin-releasing hormone receptor.
- Target
- Thyrotropin-releasing hormone receptor
- Mechanism
- Thyrotropin-releasing hormone receptor agonist
-
Development asset linked to Ultragenyx UK in public FDA and regional records. Molecular target: Peptidyl-prolyl cis-trans isomerase FKBP1A.
- Target
- Peptidyl-prolyl cis-trans isomerase FKBP1A
- Mechanism
- FK506-binding protein 1A inhibitor
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
Development asset linked to Ultragenyx UK in public FDA and regional records. Molecular target: Bacterial DNA-directed RNA polymerase.
- Target
- Bacterial DNA-directed RNA polymerase
- Mechanism
- Bacterial DNA-directed RNA polymerase inhibitor
-
risperidone
Nervous system (N05)US regulatory status: approved. Molecular target: D(2) dopamine receptor.
- Target
- D(2) dopamine receptor
- Mechanism
- Serotonin 2c (5-HT2c) receptor antagonist
-
rituximab
Antineoplastic and immunomodulating agents (L01)US regulatory status: approved. Molecular target: B-lymphocyte antigen CD20.
- Target
- B-lymphocyte antigen CD20
- Mechanism
- B-lymphocyte antigen CD20 binding agent
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
sargramostim
Antineoplastic and immunomodulating agents (L03)US regulatory status: approved. Molecular target: Granulocyte-macrophage colony-stimulating factor receptor.
- Target
- Granulocyte-macrophage colony-stimulating factor receptor
- Mechanism
- Granulocyte-macrophage colony-stimulating factor receptor agonist
-
sirolimus
Antineoplastic and immunomodulating agents (L04)US regulatory status: approved. Molecular target: Peptidyl-prolyl cis-trans isomerase FKBP1A.
- Target
- Peptidyl-prolyl cis-trans isomerase FKBP1A
- Mechanism
- FK506-binding protein 1A inhibitor
-
US regulatory status: approved.
-
sodium benzoate
Alimentary tract and metabolism (A16)US regulatory status: approved. Mechanism: Unknown.
- Mechanism
- Unknown
-
sodium phenylbutyrate
Alimentary tract and metabolism (A16)US regulatory status: approved. Molecular target: Glutamine.
- Target
- Glutamine
- Mechanism
- Glutamine sequestering agent
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
somatropin
Systemic hormonal preparations (H01)US regulatory status: approved. Molecular target: Growth hormone receptor.
- Target
- Growth hormone receptor
- Mechanism
- Growth hormone receptor agonist
-
US regulatory status: approved. Molecular target: Growth hormone receptor.
- Target
- Growth hormone receptor
- Mechanism
- Growth hormone receptor agonist
-
US regulatory status: approved. Molecular target: Growth hormone receptor.
- Target
- Growth hormone receptor
- Mechanism
- Growth hormone receptor agonist
-
US regulatory status: approved.
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
US regulatory status: approved. Molecular target: Lead.
- Target
- Lead
- Mechanism
- Lead chelating agent
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
US regulatory status: approved. Mechanism: Diagnostic agent.
- Mechanism
- Diagnostic agent
-
US regulatory status: approved. Mechanism: Diagnostic agent.
- Mechanism
- Diagnostic agent
-
thalidomide
Antineoplastic and immunomodulating agents (L04)US regulatory status: approved. Molecular target: CRL4(CRBN) E3 ubiquitin ligase.
- Target
- CRL4(CRBN) E3 ubiquitin ligase
- Mechanism
- CRL4(CRBN) E3 ubiquitin ligase inhibitor
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
tiratricol
Systemic hormonal preparations (H03)Systemic hormonal preparations (H03) compound in Ultragenyx UK's portfolio. Molecular target: Thyroid hormone receptor.
- Target
- Thyroid hormone receptor
- Mechanism
- Thyroid hormone receptor agonist
-
US regulatory status: approved. Molecular target: Carbonic anhydrase 2.
- Target
- Carbonic anhydrase 2
- Mechanism
- GABA-A receptor; anion channel positive modulator
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
US regulatory status: approved. Mechanism: Supplement.
- Mechanism
- Supplement
-
Development asset linked to Ultragenyx UK in public FDA and regional records.
-
vestronidase alfa
Alimentary tract and metabolism (A16)Alimentary tract and metabolism (A16) compound in Ultragenyx UK's portfolio. Molecular target: Glycosaminoglycans.
- Target
- Glycosaminoglycans
- Mechanism
- Glycosaminoglycans hydrolytic enzyme
-
US regulatory status: approved. Molecular target: Glycosaminoglycans.
- Target
- Glycosaminoglycans
- Mechanism
- Glycosaminoglycans hydrolytic enzyme