A drug or a biological product is designated as an ‘Orphan drug’ when authorized to prevent, diagnose or treat a rare disease or condition which are often serious or life-threatening. The indication of a drug is considered as an orphan when a substance is usually used in the treatment of a frequent disease but not intentionally developed to treat the rarer indication.

The few examples of rare diseases which require treatment by orphan drugs are:

Acute lymphoblastic leukaemia

1. Nelarabine (Brand name: Arranon)Manufactured by GlaxoSmithKline
2. Notuzumab ozogamicin (Brand name: Besponsa)Manufactured by Pfizer, Inc.

Adult On-set Still’s Disease – Canakinumab (Brand name: Ilaris) – Manufactured by Novartis

Parkinson Disease

1. Apomorphine HCL (Brand name: Apokyn®) – Manufactured by Ipsen Limited
2. Selegiline HCl (Brand name: Eldepryl)- Manufactured by Somerset Pharmaceuticals, Inc

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