1 0 years of the Orphan Regulation in Europe conference - The - - PowerPoint PPT Presentation

An agency of the European Union

1 0 years of the Orphan Regulation in Europe conference - The experience up to date

03 May 2010

• S. Aarum MD PhD / S.Tsigkos MD PhD

Outcom es of applications for ODD

1113 applications

submitted

positive outcome in 2/3

• f the cases, high

success rate

few negative opinions

per year

withdrawals may

reapply at a later stage

20 40 60 80 100 120 140 160 180 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 number of applications

submitted positive opinions negative opinions withdrawals

W ithdraw als per therapeutic area

269 withdrawals most withdrawals within

• ncology (such as

malignant melanoma, pancreatic cancer)

Other 29% Immunology 5% Oncology 33% Cardiovascul ar and Respiratory 11% Anti- infectious 5% Musculoskel etal and nervous system 13% Metabolism 4%

Opinions per therapeutic area

immunology 10%

• ncology

46% antiinfectious 3% musculoskeletal and nervous system 12%

• ther

10% metabolism 10% cardiovascular and respiratory 9% immunology

• ncology

cardiovascular and respiratory antiinfectious metabolism musculoskeletal and nervous system

• ther

Prevalence of designated applications

More than a third

affect less than 1 in 10,000 in the EU

Would attract little

interest per se without OD incentives

designations up to 10/2009

36% 52% 12% less than 1 in 10,000 between 1 and 3 in 10,000 more than 3 in 10,000

Target populations ( age)

The story so far…

A simple framework that works 1113 applications submitted More than two thirds positive opinions 724 designated orphan products

Protocol Assistance

Protocol assistance is free scientific advice Yearly increase in the number of SA/ PA procedures Links to success in marketing authorisation

264 311 73 56 77 71 69 58 136 87 72 201 215 58 55 22 25 17

50 100 150 200 250 300 350 400 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009

Scientific Advice Protocol Assistance

Use of EU special contribution for OD

Protocol assistance is

• ne of the most utilized

incentives

Contrast to post-

authorization procedures and inspections

Use of EU special contribution for orphan medicines ( 2 0 0 9 )

Ma rk e ting a uthorisa tions 2 9 % Protocol a ssista nce 6 3 % I nspe ctions 4 % Post- a uthorisa tion proce dure s 4 %

OD Marketing Authorisations

2001 Fabrazyme for Fabry disease Replagal for Fabry disease Glivec for chronic myeloid leukaemia 2002 Tracleer for pulmonary arterial hypertension Trisenox for acute promyelocytic leukaemia Somavert for acromegaly Zavesca for Gaucher disease 2003 Carbaglu for hyperammonaemia Aldurazyme for Mucopolysaccharidosis Busilvex for haematopoietic progenitor cell transplantation Ventavis for pulmonary arterial hypertension Onsenal for Familial Adenomatous Polyposis 2004 Litak for Hairy cell leukaemia Lysodren for adrenal cortical carcinoma Pedea for Patent Ductus Arteriosus Photobarr for Barret’s oesophagus Wilzin for Wilson's disease Xagrid for Thrombocythaemia

OD Marketing Authorisations

2005 Orfadin for Hereditary tyrosinemia type 1 Prialt for chronic pain requiring intrathecal (IT) 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 leukaemia Nexavar for advanced renal cell carcinoma Sutent for gastrointestinal stromal tumour 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 leukaemia and chronic myeloid leukaemia Diacomit for severe myoclonic epilepsy in infancy Elaprase for mucopolysaccharidosis type II (Hunter syndrome) Inovelon for Lennox-Gastaut syndrome Cystadane for homocystinuria

OD Marketing Authorisations

2007 Revlimid for multiple myeloma Soliris for paroxysmal nocturnal haemoglobinuria Siklos for sickle cell syndrome Atriance for acute lymphoblastic leukaemia Increlex for primary insulin-like growth factor-1 deficiency due to molecular or genetic defects Gliolan for Intra-operative photodynamic diagnosis of residual glioma Yondelis for soft tissue sarcoma Tasigna for chronic myeloid leukaemia 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 leukaemia Kuvan for hyperphenylalaninaemia Mepact for osteosarcoma Vidaza for acute myeloid leukaemia and myelodysplastic syndromes

OD Marketing Authorisations

2009 Nymusa for primary apnoea in premature newborns Afinitor for renal cell carcinoma Mozobil for mobilization of progenitor cells prior to stem cell transplantation Cayston for gram negative bacterial lung infection in cystic fibrosis Arcalyst for Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Autoinflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS) Ilaris for Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Autoinflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS) Nplate for idiopathic thrombocytopenic purpura (ITP) Firdapse for Lambert-Eaton Myasthenic Syndrome 2010 Revolade for idiopathic thrombocytopenic purpura Tepadina for conditioning prior haematopoietic progenitor cell transplantation Arzerra for chronic lymphocytic leukemia

Distribution of Marketing authorizations per therapeutic area

62 orphan drugs

received marketing approval so far

More than one third

antineoplastic and immunomodulating, followed by agents for metabolic diseases

46 46% 25% 25% 10 10% 13 13% 4% 4% 2% 2%

antineoplastic and immunomodulating agents metabolism blood musculoskeletal and nervous system cardiovascular

• thers

The story so far…

A Simple Framework that has produced measurable output: 724 designated orphan products across the entire spectrum of human disease 62 Orphan Medicinal Products have received Marketing Authorisation

Thank you