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Orfadin

Challenges for orphan medicines entering the European market

We explore the various challenges facing orphan medicines entering the European market and offer solutions to help improve patient access.

Petauri Evidence

- PMLiVE

What are “orphan drugs” and how do we navigate the challenges of writing about them?

The first in our 'Ask Alex' series, where Principal Medical Writer, Dr Alexandra Loveday answers some frequently asked questions about Medcomms. This week, she takes a look at 'orphan drugs',...

Mednet

- PMLiVE

Rare disease market access – Launching orphan drugs in secondary markets: the Nordic and MENA regions

Join us live at 1.00pm (GMT)/2.00pm (CET)/8.00am (EST) on Thursday 29th February, to explore rare disease market access challenges and learn more about these high potential markets.

Petauri Evidence

- PMLiVE

Sanofi and Sobi announce positive phase 3 trial results for severe haemophilia A

Results from the study showed once-weekly efanesoctocog alfa doses provided ‘clinically meaningful’ bleed protection for patients with severe haemophilia A

- PMLiVE

Orphan drugs market to soar to $224bn by 2024

Cell and gene therapies to fuel growth

- PMLiVE

‘Inflexible’ NICE blocking access to rare disease drugs

Spinraza most high profile example of access problem

Rare diseases and orphan drugs: Our experience and expertise

We have designed a complimentary infographic outlining the market opportunity as well as our experience and expertise in the growing area of rare diseases and orphan drugs, which includes links to...

Inizio

Malthus’ orphans

Firms focusing on small, high-profit niches risk falling into a 200-year-old trap

- PMLiVE

EMA hands UniQure orphan status first in Huntington’s

The Dutch biotech expects to file the treatment for approval later this year

Roche Basel Switzerland

Roche claims its own CAR-T first with orphan drug Actemra

Comes as FDA approves the first CAR-T therapy from Novartis

- PMLiVE

FDA moves to tackle orphan designation backlog

Some 200 requests are awaiting review by the US regulator

Shire Basingstoke

Shire finally bags EU approval for rare disease drug Natpar

Becomes first licenced treatment for hypoparathyroidism in Europe

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