Mum delivers 65 roses to Downing Street in bid to speed up NHS availability of vital cystic fibrosis drugs

16 May 2019 - Calls for NHS access to potentially life-saving drugs are growing, one year to the day after ...

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MPs call for 'life-changing' Kuvan to be made affordable

11 May 2019 - A number of MPs are calling on a drug company to make a "life-changing" treatment affordable ...

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FDA undercuts $375,000 drug in surprise move

8 May 2019 - The US FDA created a workaround this week that effectively undercuts the $375,000 price tag of ...

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Akcea and Ionis announce approval of Waylivra (volanesorsen) in the European Union

7 May 2019 - Akcea Therapeutics and Ionis Pharmaceuticals announced today that Waylivra has received conditional marketing authorisation from the ...

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European Commission approves Palynziq (pegvaliase injection) for treatment of phenylketonuria in patients aged 16 years or older

6 May 2019 - First enzyme substitution therapy approved in Europe to treat the underlying cause of phenylketonuria in patients aged ...

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NZORD supports rare medicine petitions to government

23 April 2019 - The National Organisation for Rare Disorders supports petitions for more effective, equitable outcomes for people with ...

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Priority review vouchers have helped to improve access to drugs targeting neglected diseases

22 April 2019 - The FDA priority review vouchers for neglected tropical diseases are aimed to incentivise pharmaceutical companies to ...

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Call for funding applications for medicines for rare disorders

17 April 2019 - PHARMAC invites pharmaceutical suppliers to submit funding applications for medicines for rare disorders. ...

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Family faces deportation from Australia after son diagnosed with cystic fibrosis

10 April 2019 - Anthony and Christine Hyde's application for permanent residence in Australia has been rejected - because of their ...

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Cystic fibrosis scandal: drugs deal moves a step closer to saving children

27 March 2019 - Officials are set to meet again “soon” with US drugs giant Vertex to “break the current impasse” ...

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Drug makers continue to clamour for FDA approval of orphan drugs

28 March 2019 - Orphan drugs may serve small groups of patients, but they generate big numbers at the Food ...

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600 years’ supply of cystic fibrosis drug destroyed in price row

27 March 2019 - Nearly 8,000 packs of Orkambi, the breakthrough medicine for cystic fibrosis, have been destroyed by the ...

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FDA takes new steps to advance natural history studies for accelerating novel treatments for rare diseases

22 March 2019 - The U.S. FDA today issued the draft guidance, Rare Diseases: Natural History Studies for Drug Development.  ...

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Is Orkambi deal close? Vertex meets again today with NHS England

21 March 2019 - Campaigners hopes rise after three year wait. ...

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Vertex receives approval for Symdeko (tezacaftor/ivacaftor and ivacaftor) in Australia, to treat the underlying cause of cystic fibrosis in people aged 12 and older with certain CFTR gene mutations

12 March 2019 - A new treatment option for patients with two copies of the F508del mutation, the most common mutation ...

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