rare disease
The Pharmafocus debate: Can ultra-orphan drugs be made cheaper?
January 12, 2016Manufacturing 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 …
Horizon receives European go-ahead for Ravicti
December 2, 2015Research 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 …
Shire to acquire Dyax in $5.9 billion deal
November 2, 2015Manufacturing 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 …
ABPI hits back at Prime Minister’s criticism of high drug prices
October 15, 2015Sales 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 …
Shire goes public with rejected $30bn Baxalta bid
August 5, 2015Research 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 …
Alexion makes $8.4 billion Synageva purchase
May 7, 2015Research 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 …
Shire lifted by rare disease approvals
January 26, 2015Sales 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, …
Coalition launches rare disease strategy
November 22, 2013Research 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 …
FDA holds up Pfizer’s rare disease drug tafamidis
June 19, 2012Research 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 …
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