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22 March 2019 - The U.S. FDA today issued the draft guidance, Rare Diseases: Natural History Studies for Drug Development.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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Optimism on Orkambi in England as talks to resume

12 March 2019 - Scotland's deal puts pressure on NHS England and NICE. ...

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Cystic fibrosis breakthrough: vital drug could be made available as early as next month

8 March 2019 - A wonder drug that could dramatically improve the lives of thousands of cystic fibrosis patients may be ...

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Campaigners call for NHS access to Orkambi as MP inquiry gets underway

8 March 2019 - Patients and campaigners took to the streets in London yesterday demanding NHS access to Vertex’ cystic ...

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High price of cystic fibrosis drug not political, firm's boss tells MPs

8 March 2019 - CEO of company that makes Orkambi denies being ‘political minion’ for Donald Trump. ...

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In UK and Ireland, SMA patients demand reimbursement for Spinraza

6 March 2019 - A drug appraisal committee of Britain’s NICE met in Manchester, England, for the third and final ...

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FDA grants rare paediatric disease designation to odiparcil for the treatment of MPS VI

5 March 2019 - Inventiva eligible to receive priority review voucher upon approval of odiparcil for the treatment of MPS VI. ...

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How to fix a problem like Orkambi? Solutions suggested ahead of showdown

5 March 2019 - Approaches used in France and Germany could help break deadlock. ...

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FDA is working to bridge gaps and meet needs for rare disease product development

28 February 2019 - There are approximately 7,000 rare diseases affecting an estimated 30 million people in the United States.  ...

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Vertex chief Jeffrey Leiden to weigh in on Orkambi inquiry

4 March 2019 - Vertex’s long-running spat with NHS England has amounted to a legal inquiry scheduled to begin this Thursday, ...

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Akcea and Ionis receive positive EU CHMP opinion for Waylivra (volanesorsen)

1 March 2019 - First and only therapy approved for FCS, a serious and rare disease with no approved treatment options. ...

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BioMarin receives positive CHMP opinion in Europe for Palynziq (pegvaliase injection) for treatment of patients with phenylketonuria aged 16 and older

1 March 2019 - Decision on marketing authorisation application expected Q2 '19. ...

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They can’t get their medicines. So they’re moving to Scotland.

26 February 2019 - Families in England are preparing to leave the country in a desperate bid to obtain a cystic ...

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