ANNUAL REPORT 2012 - TiGenix

pharmacological products on the market to
effectively treat localized cartilage defects.
Current treatment options include :
- surgical treatments, such as debridement,
microfracture and mosaicplasty,
- cell-based therapies, such as Autologous
Chondrocyte Implantation (ACI) and
Characterized Chondrocyte Implantation
(CCI), and
- cell-free products, such as scaffolds and
gels.
Cell-based therapies are the only treatment
options that offer a repair treatment while
sparing the osteochondral region, thus
offering a focused and gentle approach.
In addition, in comparison to the other
treatment options mentioned above,
ChondroCelect is the only treatment that
offers :
- proven efficacy and effectiveness through
a level 1, Phase III, randomized controlled
trial 7,8,9 , as well as a compassionate use
program 10 . Thus far, circa 850 patients have
been treated with ChondroCelect;
- demonstrated long term (5 year) durability
of the treatment effect (5 year follow-up
RCT) for well-defined patient profiles 9 ;
- evidence supporting the use of
ChondroCelect for large lesions 10 ;
- the security of central European marketing
authorization (approved ATMP); and
- a dedicated, licensed cGMP cell expansion
facility.
Autologous Chondrocyte Implantation
(ACI) in Europe
The barriers to entry for ACI services and
cell-based products to the European
market have been relatively low in the
past, resulting in a number of smaller
companies penetrating the market. On
January 1, 2013, however, ATMP regulations
will start being enforced in the EU. As the
only ATMP, ChondroCelect should benefit
from this situation. However, Member
States can grant exemptions to regulate
the manufacturing and use of certain
non-routine produced ATMP’s outside the
scope of the Medicinal Product Directive
2001/23 (Hospital Exemption) to provide
patients access to custom-made, innovative
individual treatment in the absence of valid
therapeutic alternatives (i.e. where there is a
clear unmet medical need).
Genzyme/Sanofi is currently involved in a
Phase III trial for MACI, for which published
results are expected in 2013. Tetec, a
subsidiary of B. Braun, has recently started
a Phase III clinical trial for NovoCart 3D.
Completion is expected in 2019 11 . Co.don
has initiated a Phase III trial for its product
ChondroSphere; results are expected
in 2016 12 . Other companies include
Biotissue Tech (product BioSeed C), Anika
7
Saris DB, Vanlauwe J, Victor J, et al. Characterized chondrocyte implantation results in better structural repair when treating
symptomatic cartilage defects of the knee in a randomized controlled trial versus microfracture. Am J Sports Med. 2008 : 36(2) :235-46.
8
Saris DB, Vanlauwe J, Victor J, et al. Treatment of symptomatic cartilage defects of the knee : characterized chondrocyte
implantation results in better clinical outcome at 36 months in a randomized trial compared to microfracture. Am J Sports Med. 2009 :
37 Suppl 1 :10S-19S.
9
Vanlauwe J, Saris DB, Victor J, Almqvist KF, Bellemans J, Luyten FP. Five-year outcome of characterized chondrocyte implantation
versus microfracture for symptomatic cartilage defects of the knee : early treatment matters. Am J Sports Med. 2011 : 39(12) :2566-74.
10
Vanlauwe J, Huylebroek J, Van Der Bauwhede J, et
al. Clinical Outcomes of Characterized Chondrocyte
Implantation. Cartilage. 2012 : 3(2) : 173-180.
58 TiGenix I annual report 2012