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rare disease

Showing 11 posts of 56 posts found.

DMD

The Pharmafocus debate: Can ultra-orphan drugs be made cheaper?

January 12, 2016
Manufacturing and Production, Research and Development, Sales and Marketing Drug pricing, Duchenne Muscular Dystrophy, Translarne, access to medicines, ataluren, muscular dystrophy, rare disease, ultra orphan drugs, ultra-orphan drugs

The recent reimbursement wrangle between PTC Therapeutics and NICE, the UK healthcare watchdog, has once again raised difficult questions about …

Feature
EU flags

Horizon receives European go-ahead for Ravicti

December 2, 2015
Research and Development, Sales and Marketing Horizon Pharma, Ravicti, horizon, orphan drug, rare disease, urea cycle disorders

The European Commission has approved Ravicti for rare urea cycle disorders.  Ravicti, (glycerol phenylbutyrate), is an oral liquid developed by Horizon …

News
Shire

Shire to acquire Dyax in $5.9 billion deal

November 2, 2015
Manufacturing and Production, Research and Development, Sales and Marketing Dyax, M&A, MA, R&D, Shire, hereditary angioedema, rare disease

Shire has struck a deal to acquire the biotech firm Dyax for $5.9 billion, in order to acquire exclusive rights …

News
PM David Cameron

ABPI hits back at Prime Minister’s criticism of high drug prices

October 15, 2015
Sales and Marketing ABPI, Drug pricing, Duchenne Muscular Dystrophy, NICE, Translarna, ataluren, david cameron, prime minister, rare disease

The ABPI has responded to comments made by UK Prime Minister David Cameron during yesterday’s Prime Minster’s Questions, in which …

News
Shire building

Shire goes public with rejected $30bn Baxalta bid

August 5, 2015
Research and Development, Sales and Marketing Baxalta, Shire, acquisitions, baxter, mergers, rare disease

Shire has confirmed that it made an all-share $30 billion offer to buy Baxalta last month, citing ’a strong strategic …

News

Alexion makes $8.4 billion Synageva purchase

May 7, 2015
Research and Development, Sales and Marketing Alexion, Shire, nps, rare disease, synageva

Rare disease specialist firm Alexion is splashing out $8.4 billion to buy fellow US orphan drug developer Synageva biopharmaceuticals. The …

News
Shire image

Shire lifted by rare disease approvals

January 26, 2015
Sales and Marketing HGT-2310, Hunter syndrome, SHP609, Shire, fast track, idursulfase-IT, rare disease

Shire’s investigational drug for a rare neurological disease has been accepted into the FDA’s fast-track approval process. The drug, SHP609, …

News
NHS image

MPs back ultra-rare disease treatment

March 12, 2014
Research and Development, Sales and Marketing BIA, NHS, UK, bates, personalised, rare disease, research

 

News
Department of health

Coalition launches rare disease strategy

November 22, 2013
Research and Development, Sales and Marketing DoH, NHS, R&D, rare disease

The Department of Health has launched the UK’s first strategy on rare diseases as part of a bid to boost …

News
shire_uk

Shire and Santaris extend rare disease alliance

August 28, 2013
Research and Development, Sales and Marketing Locked Nucleic Acid, Santaris, Shire, rare disease

News
Pfizer image

FDA holds up Pfizer’s rare disease drug tafamidis

June 19, 2012
Research and Development, Sales and Marketing FDA, Pfizer, rare disease, tafamidis

The FDA has sent Pfizer a Complete Response Letter about its rare disease drug tafamidis meglumine. The US regulator is …

News

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