Treatment Database

The following database contains products that have been granted orphan status, or have been market approval in the EU or the USA.

You can filter the content by clicking on the titles: Product, Indication, Sponsor and Date. Clicking once, will filter the column alphabetically from A – Z and a second click on the same title will filter from Z – A.

Become a Partner.

Product Indication Date Designated
small molecule inhibitor of integrins avb6 and avb1 Idiopathic Pulmonary Fibrosis Aug 01, 2018
Ex-vivo fused autologous human bone marrow-derived mesenchymal stem cell with allogenic human myoblast Duchenne muscular dystrophy Jul 31, 2018
Adenovirus associated viral vector serotype 2/8 containing the humanCNGA3gene Achromatopsia Jul 31, 2018
Tetracosactide Duchenne muscular dystrophy Jul 31, 2018
Synthetic antisense oligonucleotide directed against human dystrophin pre-mRNA Duchenne muscular dystrophy Jul 31, 2018
Selumetinib Neurofibromatosis type 1 Jul 31, 2018
N-acetylgalactosamine-conjugated synthetic double-stranded oligomer specific to serpin family A member 1 gene Congenital alpha-1 antitrypsin deficiency Jul 31, 2018
Givinostat Becker muscular dystrophy Jul 31, 2018
Allogeneic bone marrow derived mesenchymal stromal cells, ex-vivo expanded Graft-versus-host disease Jul 31, 2018
Combination of carboplatin and sodium valproate Glioma Jul 31, 2018
2ʹ-O-(2-methoxyethyl) antisense oligonucleotide targeting microtubule-associated protein tau pre-mRNA Behavioural variant frontotemporal dementia Jul 31, 2018
anti-CD47 monoclonal antibody Multiple myeloma Jul 30, 2018
antibody drug conjugate of metal-binding motif-introduced trastuzumab antibody variant and monomethyl auristatin E Gastric Cancer Jul 30, 2018
ascorbic acid Cystic fibrosis Jul 30, 2018
arsenic trioxide Graft-versus-host disease Jul 30, 2018
human Alpha1-Proteinase inhibitor Prevention of graft-versus-host disease (GVHD) Jul 30, 2018
lenabasum Dermatomyositis Jul 18, 2018
recombinant adeno-associated viral vector containing a single-stranded transgene encoding a codon-optimized human SMN1 complementary deoxyribonucleic acid under the control of the chicken beta actin promoter Spinal Muscular Atrophy Jul 16, 2018
Recombinant human insulin receptor monoclonal antibody-N-heparan sulfamidase fusion protein Mucopolysaccharidosis Type IIIA, Sanfilippo A Syndrome Jul 12, 2018
lisocabtagene maraleucel Primary mediastinal large B-cell lymphoma Jul 12, 2018