Elenco delle ultime designazioni di farmaco orfano e/o autorizzazioni all’immissione in commercio

Sono disponibili sul sito dell’EMA delle informazioni dettagliate sulle applicazioni del farmaco con designazione orfana in Europa.
Un elenco completo dei farmaci orfani designati ed autorizzati in Europa è disponibile su: ec.europa.eu

Learn more about the Orphan designation process in Europe

Autorizzazioni all’immissione in commercio
Elenco delle ultime designazioni di farmaco orfano

Elenco delle ultime designazioni di autorizzazioni all’immissione in commercio

Consulta un elenco di medicinali orfani attualmente autorizzati nell'UE.

Medicinal Product	Marketing Authorisation Holder	Therapeutic Indication	Date of Marketing Authorisation
Isturisa® (osilodrostat)	Novartis Europharm Limited	Cushing Syndrome	09/01/2020
Polivy® (polatuzumab vedotin)	Roche Registration GmbH	Diffuse large B-cell lymphoma (DLBCL)	16/01/2020
Givlaari® (givosiran)	Alnylam Netherlands B.V.	Acute hepatic porphyria	02/03/2020
Trepulmix® (treprostinil)	SciPharm Sàrl	Chronic thromboembolic pulmonary hypertension (CTEPH)	03/04/2020
Zolgensma® (onasemnogene abeparvovec)	AveXis EU Limited	Spinal muscular atrophy (SMA)	18/05/2020
Reblozyl® (luspatercept)	Celgene Europe B.V.	Beta thalassaemia & Myelodysplastic syndromes	25/06/2020
Daurismo® (glasdegib)	Pfizer Europe MA EEIG	Newly-diagnosed acute myeloid leukaemia (AML)	26/06/2020
Pretomanid FGK (pretomanid)	FGK Representative Service GmbH	Adults with drug-resistant tuberculosis	31/07/2020
Hepcludex (bulevirtide)	MYR GmbH	Chronic (long-term) hepatitis delta virus (HDV) infection in adults	31/07/2020
Idefirix (imlifidase)	Hansa Biopharma AB	Prevent the body from rejecting a newly transplanted kidney	25/08/2020
Kaftrio (ivacaftor / tezacaftor / elexacaftor)	Vertex Pharmaceuticals (Ireland) Limited	Cystic fibrosis	21/08/2020
Blenrep (belantamab mafodotin)	GlaxoSmithKline (Ireland) Limited	Multiple Myeloma	25/08/2020
Ayvakyt (avapritinib)	Blueprint Medicines (Netherlands) B.V.	Gastrointestinal stromal tumour (GIST)	24/09/2020
Arikayce liposomal (amikacin sulfate)	Insmed Netherlands B.V.	Adults with a lung infection caused by Mycobacterium avium complex (MAC)	27/10/2020
Adakveo (crizanlizumab)	Novartis Europharm Limited	Anemia, Sickle Cell	28/10/2020
Obiltoxaximab SFL (obiltoxaximab)	SFL Pharmaceuticals Deutschland GmbH	Anthrax	18/11/2020
Oxlumo (lumasiran)	Alnylam Netherlands B.V.	Hyperoxaluria, Primary	19/11/2020
Libmeldy (autologous CD34+ cells encoding ARSA gene)	Orchard Therapeutics (Netherlands) BV	Metachromatic leukodystrophy (MLD)	17/12/2020
Fintepla® (fenfluramine)	Zogenix ROI Limited	Seizures associated with Dravet syndrome	18/12/2020

Elenco delle ultime designazioni di farmaco orfano

2020

Jan - Feb - Mar - Apr - May - Jun - Jul - Aug - Sep - Oct - Nov - Dec

2019

Jan - Feb - Mar - Apr - May - June - July - Sep - Oct - Nov - Dec

2018

Jan - Feb - Mar - Apr - May - June - July - Sep - Oct - Nov - Dec

2017

Jan - Feb - Mar - Apr - May - Jun - July- Sep - Oct - Nov - Dec

Overview of Orphan Medicinal Product Designation Procedure since 2000

Including and prior to 2018 the EMA also published the detailed information summarised below:

Year	Applications submitted	Positive COMP opinions	Applications withdrawn	Negative COMP opinions	Designations granted by the Commission	Orphan medicinal products authorised	Orphan designations included in authorised therapeutic indication
2018	203	152	79	3	126	12	15
2017	260	144	96	2	138	14	15
2016	330	220	82	2	209	14	14
2015	258	177	94	1	190	14	21
2014	329	196	61	2	187	15	16
2013	201	136	60	1	136	7	8
2012	197	139	52	1	148	10	12
2011	166	111	45	2	107	5	5
2010	174	123	51	2	128	4	4
2009	164	113	23	0	106	9	9
2008	119	86	31	1	73	6	7
2007	125	97	19	1	98	13	13
2006	104	81	20	2	80	9	11
2005	118	88	30	0	88	4	4
2004	108	75	22	4	73	6	6
2003	87	54	41	1	55	5	5
2002	80	43	30	2	49	4	4
2001	83	62	27	1	64	3	3
2000	72	26	6	0	14	0	0
Total	3211	2134	881	28	2121	164	185