Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2016-06-02 | atesidorsen (2'-O-(2-methoxyethyl) phosphorothioate antisense oligonucleotide targeting the growth hormone receptor) | Coté Orphan Consulting UK (UK) Antisense Therapeutics (Australia) | acromegaly | Granting of the orphan status in the US |
2016-09-19 | 3-(5-amino-2-methyl-4-oxoquinazolin-3(4H)-yl)piperidine-2,6-dione hydrochloride | Celgene (USA - NJ) | diffuse large B-cell lymphoma |
Granting of the orphan status in the US |
2016-06-02 | allogeneic donor-derived ex-vivo expanded T lymphocytes transduced with a retroviral vector containing inducible caspase 9 and truncated CD19 | QRC Consultants (UK) | Granting of the orphan status in the EU | |
2016-06-02 | citric acid monohydrate | CATS Consultants (Germany) | Granting of the orphan status in the EU | |
2016-06-02 | donor T lymphocytes depleted ex vivo of host alloreactive T cells using photodynamic treatment | Kiadis Pharma (The Netherlands) | hematopoietic stem cell transplantation | Granting of the orphan status in the EU |
2016-06-02 | eflornithine HCL | Orbus Therapeutics (USA - CA) | glioma |
Granting of the orphan status in the EU |
2016-06-02 | humanised monoclonal antibody targeting interleukin-15 | Dr Alain Vicari | eosinophilic oesophagitis |
Granting of the orphan status in the EU |
2016-07-18 | melatonin | Therapicon (Italy) | neonatal sepsis |
Granting of the orphan status in the EU |
2016-08-01 | melatonin | Therapicon (Italy) | necrotising enterocolitis |
Granting of the orphan status in the EU |
2016-06-02 | molgramostim | Serendex Pharmaceuticals (Denmark) | acute respiratory distress syndrome |
Granting of the orphan status in the EU |
2016-06-02 | cyclocreatine | Pharma Gateway (Sweden) | creatine deficiency syndromes |
Granting of the orphan status in the EU |
2016-06-02 | diclofenamide | Sun Pharmaceutical Industries (India) | periodic paralysis |
Granting of the orphan status in the EU |
2016-06-02 | modified mRNA encoding the UGT1A1 protein | Alexion Pharmaceuticals (USA - CT) | Crigler-Najjar syndrome |
Granting of the orphan status in the EU |
2016-06-02 | pyridoxine and L-pyroglutamic acid | FGK Representative Service (Germany) | fragile X syndrome |
Granting of the orphan status in the EU |
2016-06-02 | recombinant protein derived from the saliva of the Ornithodoros moubata tick | Akari Therapeutics (USA - NY - UK) | Guillain-Barré syndrome |
Granting of the orphan status in the EU |
2016-06-02 | teriparatide | Alacrita (UK) | hypoparathyroidism |
Granting of the orphan status in the EU |
2016-07-18 | 3-[4-(1H-imidazol-1-ylmethyl)phenyl]-5-(2-methylpropyl)thiophene-2-[(N-butyloxylcarbamate)- sulphonamide] sodium salt | Vicore Pharma (Sweden) | idiopathic pulmonary fibrosis |
Granting of the orphan status in the EU |
2017-01-25 | adeno-associated viral vector serotype 2.7m8 containing the ChrimsonR-tdTomato gene | GenSight Biologics (France) | retinitis pigmentosa | Granting of the orphan status in the EU |
2016-07-18 | dimethyl fumarate | Immungenetics (Germany) | bullous pemphigoid | Granting of the orphan status in the EU |
2016-07-18 | poly(oxy-1,2-ethanediyl), alpha-(carboxymethyl)-omega-methoxy-, amide with arginase 1 [cobalt cofactor] (synthetic human) (1:10), trimer | ERA Consulting (Germany) | hyperargininaemia | Granting of the orphan status in the EU |
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