Orphan medicines in numbers

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1 3 May 2010 EMA/279601/2010 The success of ten years of orphan legislation The orphan legislation came into force in Its aim is to give patients suffering from rare diseases access to high quality of treatments by stimulating research and development of medicines for their conditions. The legislation provides a set of incentives to the pharmaceutical industry to develop 'orphan' medicines. These are medicines intended for the diagnosis, prevention or treatment of lifethreatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, or that, for economic reasons, would be unlikely to be developed without incentives. Ten years on, the European Medicines Agency has received over 1000 applications for orphan designation, which have been reviewed by the Agency s Committee for Orphan Medicinal Products (COMP). Over 700 medicines have been granted orphan status by the European Commission. By the end of April orphan designated medicines have been authorised for marketing in the European Union, potentially benefiting more than 2.6 million European patients suffering from rare diseases. These medicines cover a wide variety of rare diseases, including many genetic diseases and rare cancers, for which there are no satisfactory treatments. A large number of these diseases affect children and newborn babies. In the last few years the number of applications submitted to the Agency for orphan designation has increased significantly. The continued interest in the orphan designation process by pharmaceutical companies indicates that more orphan medicines will be coming to the market offering treatments for patients with rare diseases. 7 Westferry Circus Canary Wharf London E14 4HB United Kingdom Telephone +44 (0) Facsimile +44 (0)20 info@ema.europa.eu Website An agency of the European Union European Medicines Agency, Reproduction is authorised provided the source is acknowledged.

2 Status of orphan-designation applications A total of 1113 applications have been submitted to date for the designation of orphan medicines. A total of 760 positive opinions on orphan designation have been adopted by the COMP. A total of 269 applications have been withdrawn and 16 received a negative COMP opinion A total of 724 medicines have been granted orphan status by the European Commission. Orphan medicinal product designation procedures ( ) * Submitted Positive opinions Negative opinions Withdrawals Commission decisions * Figures for 2010 as per 30 April Orphan designation by therapeutic area The largest group of orphan medicines for which the COMP has adopted a positive opinion were for oncology treatments. COMP opinions by therapeutic area 10.3% 9.7% 12.4% 9.7% 3.3% 9.4% 45.2% Immunology Oncology Cardiovascular and respiratory Anti-infectious Metabolism Musculoskeletal and nervous system Other EMA/279601/2010 Page 2/8

3 Orphan designation by population More than half of the medicines that have received a positive opinion on orphan designation are for conditions that affect children, with 8.4 % of them for conditions that affect only children. COMP opinions by population 8.4% 41.8% 49.8% Medical conditions affecting children only Medical conditions affecting both children and adults Medical conditions affecting adults only Designated orphan medicinal products for the treatment of children and adults ( ) 100% 80% 60% 40% 20% 0% Medical conditions affecting adults only Medical conditions affecting both children and adults Medical conditions affecting children only EMA/279601/2010 Page 3/8

4 Orphan designation by prevalence of conditions The majority of conditions for which products have been given orphan designation affect between one and three in 10,000 people in the EU. COMP opinions by prevalence of condition 11.2% 35.6% less than 1 in 10,000 between 1 and 3 in 10,000 more than 3 in 10, % Marketing authorisations for orphan medicines A total of 114 marketing authorisation applications for orphan-designated medicines have been submitted to the European Medicines Agency since orphan-designated medicines have received a marketing authorisation valid across the EU. Marketing authorisations for orphan designated medicines: applications and outcomes ( ) * Marketing authorisation applications Negative outcomes (refusals and withdrawals) Marketing authorisation decisions * Figures for 2010 as per 30 April marketing authorisation applications are currently under review. EMA/279601/2010 Page 4/8

5 Marketing authorisations for orphan medicines by therapeutic area The largest group of orphan medicines that received a marketing authorisation were for oncology treatments. Marketing authorisations for orphan medicines by therapeutic area 4.8% 24.2% 3.2% 24.2% 8.1% 35.5% Immunology Oncology Cardiovascular and respiratory Anti-infectious Metabolism Musculoskeletal and nervous system Other List of authorised orphan-designated medicines per year 2001 Fabrazyme for Fabry s disease Replagal for Fabry s disease Glivec for chronic myeloid leukemia 2002 Tracleer for pulmonary arterial hypertension Trisenox for acute promyelocytic leukemia Somavert for acromegaly Zavesca for Gaucher s disease 2003 Carbaglu for hyperammonemia Aldurazyme for mucopolysaccharidosis Busilvex for hematopoietic progenitor cell transplantation EMA/279601/2010 Page 5/8

6 Ventavis for pulmonary arterial hypertension Onsenal for familial adenomatous polyposis 2004 Litak for hairy cell leukemia Lysodren for adrenal cortical carcinoma Pedea for patent ductus arteriosus Photobarr for Barret s oesophagus Wilzin for Wilson s disease Xagrid for thrombocythemia 2005 Orfadin for hereditary tyrosinemia type 1 Prialt for chronic pain requiring intrathecal analgesia Xyrem for cataplexy in patients with narcolepsy Revatio for pulmonary arterial hypertension 2006 Naglazyme for replacement therapy in patients with mucopolysaccharidosis VI Myozyme for glycogen storage disease type II (Pompe s disease) Evoltra for acute lymphoblastic leukemia Nexavar for advanced renal cell carcinoma Sutent for gastrointestinal stromal tumor and metastatic renall cell carcinoma Savene for anthracycline extravasation Thelin for idiopathic pulmonary arterial hypertension or pulmonary arterial hypertension Exjade for chronic iron overload due to blood transfusions Sprycel for acute lymphoblastic leukemia and chronic myeloid leukemia Diacomit for severe myoclonic epilepsy in infancy Elaprase for mucopolysaccharidosis type II (Hunter s syndrome) Inovelon for Lennox Gastaut syndrome Cystadane for homocystinuria EMA/279601/2010 Page 6/8

7 2007 Revlimid for multiple myeloma Soliris for paroxysmal nocturnal hemoglobinuria Siklos for sickle cell syndrome Atriance for acute lymphoblastic leukemia Increlex for primary insulin-like growth factor 1 deficiency due to molecular or genetic defects Gliolan for intraoperative photodynamic diagnosis of residual glioma Yondelis for soft tissue sarcoma Tasigna for chronic myeloid leukemia Torisel for renal cell carcinoma 2008 Thalidomide Celgene for multiple myeloma Volibris for pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension Firazyr for angioedema Ceplene for acute myeloid leukemia Kuvan for hyperphenylalaninemia Mepact for osteosarcoma Vidaza for acute myeloid leukemia and myelodysplastic syndromes 2009 Nymusa for primary apnea in premature newborns Afinitor for renal cell carcinoma Mozobil for mobilize progenitor cells prior to stem cell transplantation Cayston for Gram-negative bacterial lung infection in cystic fibrosis Arcalyst for cryopyrin-associated periodic syndromes, including familial cold autoinflammatory syndrome and Muckle-Wells syndrome Ilaris for cryopyrin-associated periodic syndromes, including familial cold autoinflammatory syndrome and Muckle-Wells syndrome Nplate for idiopathic thrombocytopenic purpura Firdapse for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) EMA/279601/2010 Page 7/8

8 2010 Revolade for the treatment of chronic immune (idiopathic) thrombocytopenic purpura (ITP) Tepadina for conditioning treatment prior to autologous or allogeneic haematopoietic progenitor cell transplantation Arzerra for the treatment of chronic lymphocytic leukaemia (CLL) EMA/279601/2010 Page 8/8