- PMLiVE

FDA backs BioMarin drug for rare enzyme disorder

Vimizim is first drug approved in US under rare paediatric disease priority review

- PMLiVE

EMA to expand orphan drug HTA collaboration

Will further develop the concept of parallel scientific advice in 2014

- PMLiVE

Orphan drugs: A ‘responsible’ investment

There's growing sense of responsibility in the orphan drug market, but the market remains under-exploited by companies

- PMLiVE

Supportive strategies for demonstrating value

Improved planning will support better data generation and improve understanding of rare diseases and the orphan drugs that treat them

- PMLiVE

FDA approves sleep disorder drug for blind people

Vanda Pharmaceuticals wins backing for Hetlioz to improve a person’s body clock

The thing about small numbers

Marketers working in orphan drugs need to take a narrower, deeper approach to relationship building, and look to medical centres of excellence

Small diseases need big picture communications

An early commitment to communications planning in rare diseases is worth the risk

Orphan indication? No easy access

Health technology assessment for orphan drugs continues to present challenges for pharmaceutical companies

- PMLiVE

FDA panel backs Chelsea’s Northera for rare disease

Committee votes in favour of treatment for low blood pressure

Sanofi reception

Sanofi strikes $700m rare diseases deal

Company's Genzyme division will obtain significant rights to Alnylam’s pipeline of orphan drugs

- PMLiVE

US approval for Novo’s Factor XIII drug Tretten

Becomes first recombinant treatment licensed for a rare clotting disorder

- PMLiVE

Janssen’s Sirturo wins backing for conditional EU licence

CHMP recommends first-in-class multidrug-resistant tuberculosis treatment

Novartis building

Novartis myeloma drug clears phase III trial

Panobinostat on course to be first in new class of treatment for bone marrow cancer

- PMLiVE

EMA: more fee reductions for orphan drugs

Greater incentives introduced for larger companies

The logistics of orphan drugs

Managing a rare disease global access programme, the goal is to minimise risk and get the drug to the patient on time