Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2016-12-08 | (6aR, 10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid | TMC Pharma Services (UK) | systemic sclerosis |
Positive opinion for the granting of the orphan status in the EU |
2017-01-12 | 3-pentylbenzeneacetic acid sodium salt | ProMetic Pharma SMT (UK) | Alström syndrome | Granting of the orphan status in the EU |
2016-12-08 | antroquinonol | Biological Consulting Europe (UK) | pancreatic cancer |
Positive opinion for the granting of the orphan status in the EU |
2016-12-08 | autologous dendritic cells incubated ex vivo with zebularine and factor VIII | Idogen (Sweden) | hemophilia A |
Positive opinion for the granting of the orphan status in the EU |
2016-12-08 | doxorubicin hydrochloride in a lipid-based pegylated nanoparticle modified with a 31-aminoacid peptide targeting nucleolin | TREAT U, S.A (Portugal) | malignant mesothelioma | Granting of the orphan status in the EU |
2017-11-27 | genetically modified adeno-associated viral vector serotype 9 expressing shRNA as well as a codon-optimised shRNA-insensitive wildtype PABPN1 | Clinipace (Germany) Benitec BioPharma (Australia) | oculopharyngeal muscular dystrophy | Granting of the orphan status in the EU |
2016-12-08 | human donor hematopoietic stem and progenitor cells that have been treated ex vivo with the protein transduction domain of the HIV-1 transactivation protein fused to MYC transcription factor | Coté Orphan Consulting UK (UK) | hematopoietic stem cell transplantation |
Positive opinion for the granting of the orphan status in the EU |
2016-12-08 | human hepatoma cell line HepaRG in bioartificial liver | Hep-Art Medical Devices (The Netherlands) | acute liver failure |
Positive opinion for the granting of the orphan status in the EU |
2017-01-12 | humanised IgG1 monoclonal antibody against the receptor-binding site of human placental growth factor | Oncurious (Belgium) | medulloblastoma | Granting of the orphan status in the EU |
2016-12-08 | pentosan polysulfate sodium | Kyoto Tech (Japan) | interstitial cystitis | Positive opinion for the granting of the orphan status in the EU |
2016-12-08 | Pr-D-Cys-Met-Pip-Arg-Leu-Arg-Sar-Cys-Lys-Arg-Pro-Tyr-Tle-Leu-OH | Vect-Horus (France) | perinatal asphyxia |
Positive opinion for the granting of the orphan status in the EU |
2016-12-08 | recombinant adeno-associated viral vector serotype 9 containing the human N-alphaacetylglucosaminidase gene | Ser-mes Planificación SL (Spain) | mucopolysaccharidosis type IIIB (Sanfilippo Bsyndrome) |
Positive opinion for the granting of the orphan status in the EU |
2016-12-08 | recombinant IgG degrading enzyme of Streptococcus pyogenes | Hansa Medical (Sweden) | prevention of graft rejection following solid organ transplantation |
Positive opinion for the granting of the orphan status in the EU |
2016-12-08 | trans-resveratrol | Luis Pereira de Almeida (Portugal) | spinocerebellar ataxia |
Positive opinion for the granting of the orphan status in the EU |
2018-03-23 | biosimilar version of insulin glargin | Mylan (USA - PA) Biocon (India) | type 1 diabetes, type 2 diabetes | Granting of a Market Authorisation in the EU |
2016-05-18 | (tetrahydro-N,N-dimethyl-2,2-diphenyl-3-furanmethanamine hydrochloride) | Anavex Life Sciences (USA - NY) | Rett Syndrome |
Granting of the orphan status in the US |
2016-06-13 | cannabidiol | GW Research (UK) | infantile spasms |
Granting of the orphan status in the US |
2016-11-20 | (2-((2-chlorophenyl)(phenyl)amino)-N-(7-(hydroxyamino)-7-oxoheptyl)pyrimidine-5-carboxamide) | Acetylon Pharmaceuticals (USA - NJ), a Celgene subsidiary (USA - NJ) | multiple myeloma |
Granting of the orphan status in the US |
2016-07-20 | (S)-7-(1acryloylpiperidin-4-yl)-2-(4-phenoxyphenyl)-4,5,6,7-tetrahydropyrazolo[1,5-a]pyrimidine-3-carboxamide | BeiGene USA (USA - NY) | chronic lymphocytic leukemia |
Granting of the orphan status in the US |
2016-07-07 | 1-(2-chlorophenyl)-1-(S)-hydroxy-2-(S)-carbamoyloxy-propane | Bio-Pharm Solutions (Republic of Korea) | status epilepticus |
Granting of the orphan status in the US |
© 2024 Biopharmanalyses - Powered by Samacom+