Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2015-07-28 | modified adenovirus serotype 5/35 containing a CMV promoter-driven transgene cassette with the human transgenes for a membrane-bound CD40 ligand and full length 4-1BBL | Lokon Pharma (Sweden) | pancreatic cancer | Granting of the orphan status in the EU |
2015-07-28 | sarizotan hydrochloride | Newron Pharmaceuticals (Italy) | Rett syndrome |
Granting of the orphan status in the EU |
2015-07-28 | triheptanoin | Ultragenyx Pharmaceutical (USA - CA) | carnitine palmitoyltransferase II deficiency | Granting of the orphan status in the EU |
2018-08-29 | triheptanoin | Ultragenyx Pharmaceutical (USA - CA) | long-chain 3-hydroxyacyl-coA dehydrogenase deficiency | Submission of an NDA |
2015-07-28 | triheptanoin | Ultragenyx Pharmaceutical (USA - CA) | mitochondrial trifunctional protein deficiency | Granting of the orphan status in the EU |
2015-05-12 | N-t-butylhydroxylamine | Andrew Lim, California Institute of Technology (USA – CA) | infantile neuronal ceroid lipofuscinosis |
Granting of the orphan status in the US |
2015-03-24 | STP-206 (Lactobacillus acidophilus and Bifidobacterium animalis subsp. Lactis) | Sigma-Tau Pharmaceuticals (USA – MD) | prevention of necrotizing enterocolitis (NEC) in premature infants with very low birth weight less than or equal to 1500 grams
|
Granting of the orphan status in the US |
2015-04-02 | triolimus | Co-D Therapeutics (USA – WI) | angiosarcoma |
Granting of the orphan status in the US |
2015-06-25 | human alpha1-proteinase | CSL Behring (Germany) | maintenance treatment, to slow the progression of emphysema in adults with documented severe alpha1-proteinase inhibitor deficiency (e.g. genotypes PiZZ, PiZ(null), Pi(null,null), PiSZ) |
Positive opinion for the granting of a Market Authorisation in the EU |
2015-07-28 | insulin detemir | Novo Nordisk (Denmark) | diabetes mellitus in adults, adolescents and children aged 2 years 1 year and above |
Granting of a Market Authorisation in the EU |
2015-09-23 | ferric citrate coordination complex | Keryx Biopharmaceuticals (USA - NY) | hyperphosphataemia in adult patients with chronic kidney disease |
Granting of a Market Authorisation in the EU |
2017-03-30 | guanfacine | Shire (UK - USA) Shionogi (Japan) |
|
Granting of a Market Authorisation in Japan |
2015-09-18 | sufentanil | Grunenthal (Germany) | post-operative pain |
Granting of a Market Authorisation in the EU |
2015-06-11 | eltrombopag | Novartis (Switzerland) | paediatric patients six years and older with chronic immune (idiopathic) thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy |
Granting of a Market Authorisation in the US |
2015-08-10 | glycyl-L-2-methylprolyl-L-glutamic acid | QRC Consultants (UK)/Neuren Pharmaceuticals (Australia) | Rett syndrome
|
Granting of the orphan status in the EU |
2015-11-09 | human allogeneic bone-marrow-derived osteoblastic cells | Bone Therapeutics (Belgium) | osteogenesis imperfecta |
Granting of the orphan status in the US |
2015-08-10 | insulin human | Sirius Regulatory Consulting (UK) | short bowel syndrome |
Granting of the orphan status in the EU |
2015-08-10 | (S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride | Khondrion (The Netherlands) | mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS syndrome) |
Granting of the orphan status in the EU |
2015-08-10 | 2-(2-phenylvinyl)-4-[4-methylpiperazin-1-yl)]-6-(5-methyl-2H-pyrazol-3-yl-amino)-pyrimidine L(+) tartrate salt | CASI Pharmaceuticals (USA - MD) Dr Ulrich Granzer (Germany) | hepatocellular carcinoma |
Granting of the orphan status in the EU |
2015-08-10 | adeno-associated viral vector serotype 8 containing the human MTM1 gene | Audentes Therapeutics (USA - CA) | X linked myotubular myopathy | Granting of the orphan status in the EU |
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