NICE widens its Zolgensma appraisal due to European Marketing Authorisation

11 September 2020 - England’s NICE is expanding its appraisal of Zolgensma, a gene therapy for spinal muscular atrophy, according ...

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Health ministers condemn Novartis lottery for Zolgensma, the world’s most expensive drug

12 February 2020 - Novartis has held the first draw to choose four babies who will receive its one-shot treatment ...

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NICE refuses Akcea’s Waylivra

3 January 2020 - NICE has issued an Evaluation Consultation Document, refusing Waylivra (volanesorsen) as a treatment for familial chylomicronaemia ...

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Biotech companies defend prices of one-off gene therapy

9 December 2019 - Latest treatments with price tags as high as $2 million require new financing models. ...

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AveXis rules out EU compassionate use of SMA gene therapy, says Biogen option is available

27 September 2019 - Novartis’ AveXis unit has given its reasons for refusing to supply its spinal muscular atrophy gene ...

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NHS funds eye gene therapy, after Novartis cuts £613,000 price

4 September 2019 - NICE has recommended the NHS should fund Novartis’ Luxturna for a rare eye disorder, after the ...

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NICE recommends novel gene therapy treatment for rare inherited eye disorder

4 September 2019 - It's estimated that 86 people would be eligible for treatment with voretigene neparvovec in England. ...

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'Amazing' gene-silencing drugs reach NHS

9 July 2019 - A new form of medicine called "gene-silencing" has been approved for use by the NHS in ...

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NICE Onpattro approval puts Alnylam back in hATTR running

 9 July 2019 - NICE has recommended the use of Alynylam’s Onpattro (patisiran), after issuing a preliminary ‘no’, back in ...

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Spinraza access agreement extended

3 July 2019 - More children with the rare genetic disorder spinal muscular atrophy can now be treated with Spinraza after ...

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Final NICE guidance issued for Akcea’s Tegsedi

24 May 2019 - Akcea Therapeutics UK has announced that NICE has published a final guideline for its nerve damage ...

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NHS chief hails SMA drug deal – and was that a swipe at Vertex?

15 May 2019 - Children in England with the rare muscle wasting disease spinal muscular atrophy will get access to ...

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NICE recommends first ever treatment for children with rare muscle wasting condition

15 May 2019 - Children with the rare genetic disorder spinal muscular atrophy can now be treated with nusinersen after NICE ...

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Akcea steals march on Alnylam with NICE approval

16 April 2019 - Advantage to Tegsedi over Onpattro. ...

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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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