Successful Use of

Existing Opportunities for

Orphan Drug Approval

Catarina Edfjäll, Ph.D.

VP, Head of EU and International Regulatory Affairs,

Shire HGT, Switzerland

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To be as brave as the people we help

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Orphan Drug Approval

~7000 Rare Diseases

~1% covered by treatment in EU

> 25 mio Patients still waiting…

OMP Marketing Authorisations in the EU

≈ 7/year

Orphan Drug Approval

Median review time*

235 days (FDA)

381 days (EMA)

* NEJM (2012): Downing et al.

We do have some tools – let’s use them!

• Conditional MA

• Accelerated Review

• Protocol Assistance

• Protocol Assistance

• Innovation Task Force

• Business Pipeline meetings

• EMA/HTA consultations

• Parallel Sci. Advise with FDA

• Pre-submission meetings

• EMA SME office

Accelerating Orphan Drug Development: The

VPRIV® Story

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Gaucher Disease

Collapsed

vertebrae

Pathologic

fracture

Erlenmeyer flask

deformity

Osteonecrosis/

Osteopenia

Bone crisis

To be as brave as the people we help

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Searching the globe for Gaucher patients …

• USA: Priority Review by the FDA (6 m)

• EU: Accelerated Review by the CHMP (8 m)

To be as brave as the people we help

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VPRIV approved in 39 countries

Can we reduce the

number of hurdles

for OMP Approval?

Flexibility

Predictability

Continuity

~60% of ODs

developed for

paediatric

indications

Let’s learn from each other!

Global Harmonisation of regulatory requirements

You don’t know if it works until

you try

Orphan Drug Approval

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If we can be as brave as the

people we help,

if we can be as brave as the

specialists we serve,

then we will be very brave indeed

Thank you for listening!