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.

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Product Indication Date Designated
adeno-associated virus serotype 6 containing human LAMAN cDNA Alpha-mannosidosis Feb 15, 2018
selumetinib Neurofibromatosis Type 1 Feb 14, 2018
recombinant humanized IgG4 monoclonal antibody against pHERV-W Envelope protein Chronic inflammatory demyelinating polyradiculoneuropathy/polyneuropathy (CIDP) Feb 14, 2018
double stranded oligomer ADS-001 RNA interference-based liver targeted therapeutic Alpha-1 antitrypsin deficiency Feb 14, 2018
inter-Alpha-Inhibitor-Proteins (IaIp) (Human) Necrotizing enterocolitis (NEC) Feb 13, 2018
tilorone dihydrochloride Ebola infection Feb 07, 2018
a self-complementary adeno-associated virus serotype 9 expressing human codon-optimized CLN1 under control of CBh Neuronal ceroid lipofuscinosis type 1 (CLN1) Feb 07, 2018
anti-human epidermal growth factor receptor 2 (HER2) antibody drug conjugate Gastric cancer including cancer of gastroesophageal junction Feb 01, 2018
2-(3,7-Dimethyl-octa-2, 6-dienyl)�6-ethylamino-3-hydroxy-5-pentyl-[1,4]benzoquinone Huntington�s Disease (HD) Feb 01, 2018
glucagon analog conjugated with human immunoglobulin G4 fragment Congenital hyperinsulinism (CHI) Feb 01, 2018
fostamatinib Autoimmune hemolytic anemia (AIHA) Jan 31, 2018
recombinant humanized monoclonal antibody targeting the anti-m�llerian hormone receptor II Anti -Mullerian Hormone Receptor II (AMHRII)-expressing ovarian cancer Jan 30, 2018
neihulizumab Acute graft-versus-host disease (aGvHD) Jan 25, 2018
humanized IgG4-based anti-CD20 x anti-CD3 bispecific monoclonal antibody Follicular lymphoma (FL) Jan 25, 2018
branaplam Spinal muscular atrophy (SMA) Jan 23, 2018
2-{4-[(9R)-9-Hydroxy-2-(3-hydroxy-3-methylbutyloxy)-9-(trifluoromethyl)-9H-fluoren-4-yl]-1H-pyrazol-1-yl}-2-methylpropanamide monohydrate Pulmonary arterial hypertension Jan 23, 2018
riluzole oral soluble film (ROSF) Amytrophic lateral sclerosis (ALS) Jan 23, 2018
octreotide acetate (intranasal) Acromegaly Jan 23, 2018
inclisiran Homozygous familial hypercholesterolemia (HoFH) Jan 22, 2018
humanized immunoglobulin G1 (IgG1) T-cell bispecific (TCB) antibody targeting carcinoembryonic antigen (CEA) Gastric cancer, including gastroesophageal junction cancer Jan 22, 2018