Module 5 Presentations

05/07/2024

Thalidomide (3)

Why?

• The drug was not tested for use in pregnancy in animal models • In many countries it was placed on the market as non-prescription drug

• The original indication – sedative or tranquiliser • Off-label use – morning sickness during pregnancy • Insufficient pharmacovigilance system

The Organisation for Professionals in Regulatory Affairs

Thalidomide tragedy (1956-1961)

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Thalidomide (4)

Revival • Pharmion ltd. (Celgene) submitted new application 22.1.2007 • Centralised procedure • Orphan indication • Indication - Multiple myeloma • Approved 16.4.2008

Manymore indicationsapproved under“orphan status“

Graft versus host disease

Crohn`s disease

Erythema Nodosum Leprosum

Kaposi sarcoma

Severe recurrent aphthous stomatitis

Myelodysplastic syndrome

Primary brain malignancies

Telangiectasia

HIV-associated wasting syndrome

Mycobacterial infection

Recurrent aphthous ulcers

Hematopoietic stem cell transplantation

The Organisation for Professionals in Regulatory Affairs

Thalidomide tragedy (1956-1961)

10

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