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Regulation of Advanced Therapies

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EuropaBio is the voice of the biotechnology industry in Europe. EuropaBio represents: ... Articular surface. Subchondral bone. 1. Biopsy. 2. Cell culture. 4. Rehab ... – PowerPoint PPT presentation

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Title: Regulation of Advanced Therapies


1
  • Regulation of Advanced Therapies
  • A Biotech SME Perspective
  • May 11, 2006

Gil Beyen CEO - TiGenix N.V. www.tigenix.com Memb
er of the EuropaBio Advanced Therapies Working
Group www.europabio.org
2
EuropaBio
  • EuropaBio is the voice of the biotechnology
    industry in Europe
  • EuropaBio represents
  • around 1500 SMEs
  • 68 large companies
  • 25 national biotechnology associations across
    Europe
  • 5 bio-regions
  • EuropaBio champions the responsible use of
    biotechnology to ensure that its potential is
    fully used to the benefit of people and their
    environment in all fields, including healthcare.

3
TiGenix an SME profile
  • Founded 2000, spin-off of Universies of Leuven
    Ghent
  • Financing Private - Venture backed (29 M)
  • Team 45 people
  • Focus Regenerative medicine for joint surface
    repair
  • Science Biology of stable hyaline cartilage
  • Approach Evidence Based Medicine
  •  From procedure to validated medicinal
    product 
  • Lead product ChondroCelect, autologous cell
    therapy product
  • for cartilage repair in Phase III clinical
    trial

SME
4
Major unmet medical need
Osteoarthritis
Age
Overweight
Genetics
Cartilage damage
  • More than 2 million patients per year with
    cartilage damage (US EU)
  • Cartilage damage known to be one of the main
    causes for OA
  • OA affecting over 10 of the population one of
    leading cause of disability at older age fast
    growing no effective treatments today
  • ? Need for biological solutions leading to
    durable repair
  • (JRC report market potential cartilage
    regeneration 2011 25 bn )

5
Autologous Chondrocyte Implantation (ACI)
Full thickness cartilage defect
1. Biopsy
2. Cell culture


3. Implantation

Articular surface Subchondral bone
?
4. Rehab
6
ChondroCelect Goal validated therapeutic
product for durable repair
Key challenges
Key responses
  • To produce cells that can build stable cartilage
    in vivo
  • To develop a consistent, reliable product with
    proven efficacy
  • To position the product in the treatment
    algorithm of joint surface defects
  • To obtain approval reimbursement
  • ChondroCelect cell technology
  • Proprietary potency assay
  • Extensive preclinical validation
  • GMP with batch release criteria
  • Prospective randomised GCP controlled clinical
    trials
  • Health economics studies

7
Condition Harmonised and Equitable Regulatory
Framework
  • High standards ? patient public confidence
  • Harmonised requirements
  • Adapted to specific needs
  • Data exclusivity IP protection
  • Possibility for branding pricing
  • Equitable for all parties
  • ? The bar can (and should) be high as long as
    at same level for all parties ...

? achievable
  • economic return condition for
  • innovation

8
The proposed ATP Regulation provides important
step forward (1)
  • Main positive points
  • Single legislation for all Advanced Therapy
    Medicinal Products
  • ... as a regulation
  • ... utilizing an existing product framework
  • ... tailor made to ATP
  • Centralised procedure ensuring harmonised
    standards
  • Data exclusivity IP protection foreseen
  • Financial incentives administrative help

9
The proposed ATP Regulation provides important
step forward (2)
  • Remaining concerns
  • Equal requirements for all parties (also for
    hospital-based) ...without hindering
    academic research
  • Will there be sufficient expertise and what role?
    Composition of the CAT and role of CAT advice
  • Implementation specifics (definitions, adaptation
    of the Clinical trials and GMP Directives)
  • Speed of implementation

10
Conclusions
  • Cell therapies and tissue engineering will change
    medical practices profoundly
  • Harmonized and equitable regulatory framework,
    conditio sine qua non for the necessary
    innovation and to ensure patients access to
    valuable new therapies
  • TiGenix (SME) and EuropaBio welcome the draft
    proposal ...
  • ... and look forward to contributing to a speedy
    adoption process

11
Thank you! More info Gil Beyen www.tigenix.c
om EuropaBio www.europabio.org
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