Rare Disease

News

Merck's Welireg becomes first drug for rare VHL tumours in U...

Merck & Co has extended its range of cancer medicines in the US after claiming FDA approval for Welireg – a drug it acquired as part of its $2.2 billion takeover of Peloton Therapeutics

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Gene therapy specialist bluebird exits "untenable" European ...

Europe's market has proved so hostile to gene therapies when it comes to pricing and reimbursement for gene therapies that bluebird bio has decided to quit the market altogether, according

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Sanofi extends its Pompe range with first Nexviazyme OK

Sanofi has added another string to its Pompe disease therapy bow, after getting FDA approval for Nexviazyme, its latest therapy for the rare, inherited disorder.

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Sanofi swoops on partner Translate, buying the mRNA firm for...

Sanofi has been forging ever-closer ties with its mRNA partner Translate Bio since the two started working together in 2018, and has now gone all in, offering $3.2 billion to buy the compan

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Ipsen forges $1bn alliance with Exicure on neurodegenerative...

Ipsen's new chief executive David Loew pledged to ramp up spending on the company's pipeline when he took the helm last year, and a flurry of licensing deals in the last few weeks shows tha

News

Boost for AZ as Ultomiris gets CHMP nod for expanded label

Fresh from its takeover of Alexion, AstraZeneca has picked up a recommendation in the EU for an expansion of the label of Ultomiris, one of the main assets behind the