Orphan Drugs for Rare Cancers: Proposals for Action at the

Agence Fédérale des Médicaments et des Produits de Santé
Federal Agency for Medicines and Health Products
www.fagg.be or www.afmps.be
Orphan Drugs for Rare Cancers: Proposals for Action at
the European Level
All cancers, rare cancers? The advent of tissue products
and
d vaccines
i
in
i cancer treatment
t
t
t
Dr André LHOIR
COMP Member
b
AL
05.10.2010
05.10.2010
1
Agence Fédérale des Médicaments et des Produits de Santé
Orphan Medicinal Products
EU Orphan Regulation 141/2000
The Orphan Designation always refer to a specific
medicinal product for the diagnosis or prevention
or treatment
t t
t off a specific
ifi condition.
diti
•Life-threatening
•Life
threatening or chronically debilitating
condition
evale ce max
a 5
5/10.000
0.000
• Prevalence
• +- 800 designations since 2000 and > 50 MAs
• 10 yyears exclusivityy ((+2 if Ped-Inv-Plan OK))
• Acces to EU funding (DG Research…)
• Protocol assitance (EMA)
AL
05.10.2010
2
Agence Fédérale des Médicaments et des Produits de Santé
Orphan Medicinal Products
Member States
•National incentives
•Several national plans for rare diseases / orphan
drugs
•Several national plans for cancer
•Several plans for children….
AL
05.10.2010
3
Agence Fédérale des Médicaments et des Produits de Santé
OD by therapeutic field
COMP opinions by therapeutic area (2009)
Immunology
7%
Other
23%
Musculoskeletal
and nervous
system
13%
Metabolism
5%
Cardiovascular
Anti-infectious and respiratory
3%
4%
Oncology
45%
>300 (out of 800) designations for cancer conditions
>80 different types of cancer actually designated !
AL
05.10.2010
4
Agence Fédérale des Médicaments et des Produits de Santé
ATMPs with OD
7%
52%
41%
AL
05.10.2010
tissue
cell
gene
57 Positive
P i i OD out off 788 (7
(7.2%)
2%)
In 2010 already 9.8%
–4
4 Tissue engineered
–23 Cell therapy
–29 Gene therapy
–1 gene-tissue (?)
5
Agence Fédérale des Médicaments et des Produits de Santé
ATMPs that have OD
7
6
5
4
cell
gene
tissue
3
2
1
0
2001 2002 2003 2004 2005 2006 2007 2008 2009 2010
AL
05.10.2010
6
Agence Fédérale des Médicaments et des Produits de Santé
Products designated in 2010 (i)
Lentiviral vector containing human MYO7A gene
Gene therapy; CAT Dec
09
Treat of retinitis pigmentosa
Autologous dendritic cells pulsed with recombinant human fusion protein (mucin 1glutathione S transferase) coupled to oxidised polymmanose
Cell therapy;
CAT June 10
Treat of ovarian cancer
Adenovirus associated viral vector serotype 10 carrying the human N-sulfoglucosamine
N sulfoglucosamine
sulfohydroxilase and sulfatase modifying factor 1 cDNAs
Cell therapy; CAT Dec
09
Treat of Sanfiippo A (MPS
IIIA)
Allogeneic human dermal fibroblasts
Cell / tissue; CAT Feb 10 Treat Epidermolysis bullosa
AL
05.10.2010
7
Agence Fédérale des Médicaments et des Produits de Santé
Products designated in 2010 (ii)
Allogeneic T cells encoding an exogenous
TK gene
Cell therapy;
CAT Feb 10
Treat AML
Autologous bone marrow derived
mononuclear cell fraction
Cell therapy;
CAT certification May 10
Treat tromboangiitis obliterans
AL
05.10.2010
8
Agence Fédérale des Médicaments et des Produits de Santé
orphan medicinal conditions (bio)markers in subsetting ?
example : C
C--Cure 709
• Clonal, cytotoxic, melanoma HLA-A2 restricted
CD8 T-cell
T cell line expressing MART-1
MART 1 T-cell
T cell receptor
• treatment of malignant melanoma in HLA-A2
positive patients
Subset
S
b t
• Prevalence melanoma: 7.2 per 10,000
• +/- 50% of Caucasians carry the HLA-A2 allele
• <100% of melanomas express Mart-1
• Subset: 3.6 per 10.000
AL
05.10.2010
9
Agence Fédérale des Médicaments et des Produits de Santé
ATMPs without OD
4 oncology
•Hepatocellular
p
carcinoma ((JX-594;;
GT)
•Non-small cell lung cancer (TG4010;
GT)
•Colon cancer Stage II (Oncovax; CT)
•Prostate cancer - castration resistant
(Prostavac; GT)
AL
05.10.2010
10