News NHS England explores funding options for cystic fibrosis drug

17 September 2019 - NHS England is “committed to exploring and developing alternative routes” to enable people with cystic fibrosis ...

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Vertex announces new access agreement with Scottish Government for Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor)

12 September 2019 - Eligible patients in Scotland will immediately have access to Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor) in combination with ...

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Batten disease treatment: parents win battle ahead of court date

11 September 2019 - Families of children with a rare degenerative disease have won their fight for funding for NHS ...

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Children with rare inherited condition to benefit from drug through managed access agreement

11 September 2019 - It is estimated that in the UK there are around 30 to 50 children living with the ...

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The FDA and Sarepta: a window into the real world of drug regulation

3 September 2019 - It is hard to discern the true state of drug regulation from the outside, but two ...

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European Commission approves Soliris (eculizumab) for the treatment of adults with neuromyelitis optica spectrum disorder

27 August 2019 - 98% of adult anti-aquaporin-4 antibody-positive patients treated with Soliris were relapse free compared to 63% receiving placebo ...

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FDA accepts new drug application for VX-445 (elexacaftor), tezacaftor and ivacaftor combination treatment

20 August 2019 - FDA grants priority review of the application and sets a PDUFA target action date of 19 ...

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Canadian drug price regulator may be flexible on rare diseases

14 August 2019 - Canada’s patented drug price regulator, set to gain new powers next year, may be “more forgiving” in ...

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Medicines access for cancer must also be Fair for Rare

30 July 2019 - Rare Disorders NZ welcomes the National Party’s recent announcement on cancer care but calls on both ...

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Aeglea BioTherapeutics receives FDA breakthrough therapy designation for pegzilarginase for treatment of arginase 1 deficiency

24 July 2019 - Designation follows recently reported ARG1-D Phase 1/2 data demonstrating clinical response in patients rreated with pegzilarginase. ...

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Patients, backed by ‘dark money’ group, get ready to debate Duchenne drug prices. It will not be boring.

24 July 2019 - Meetings of the Institute for Clinical and Economic Review don’t usually get one’s heart pumping.  ...

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Mum needs $100,000 to treat baby son with infantile hypophosphatasia

23 July 2019 - A baby boy will only live for a few months without a $100,000 medication that is ...

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Vertex submits new drug application to the U.S. FDA for triple combination regimen of VX-445 (elexacaftor), tezacaftor and ivacaftor in cystic fibrosis

22 July 2019 - Application supported by positive results from two global Phase 3 studies in people with CF ages 12 ...

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Perth boy battles cystic fibrosis, with a life extending drug frustratingly out of reach

15 July 2019 - In many ways Connor Barrett is just like any other kid - he loves Peppa Pig ...

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Irish cystic fibrosis family face €12,400 bill after deportation victory in Australia

15 July 2019 - An Irish family threatened with deportation from Australia because their son has cystic fibrosis have been ...

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