Reimbursement for orphan drug Spinraza imminent

18 March 2019 - Biogen’s Spinraza (nusinersen), a treatment for spinal muscular atrophy, is likely to obtain reimbursement as early ...

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Family of sick toddler launches petition urging PHARMAC to fund life-extending drug Spinraza

10 March 2019 - A Pukekohe family has launched a petition urging PHARMAC to fund a drug for spinal muscular ...

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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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Panel backs provincial funding for expensive drug to treat rare disease. But only for children.

4 March 2019 - Provincial governments should expand public funding for one of the world’s most expensive medications to cover ...

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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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Biogen responds to CADTH’s updated recommendation regarding coverage for Spinraza

1 March 2019 - CADTH has expanded their recommendations for public reimbursement for SMA patients. ...

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'Depriving sick babies of right to life' - NZORD boss blasts PHARMAC decision to defer Spinraza

28 February 2019 - Clinical experts deciding whether to fund a drug that would prolong the life of babies with ...

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Roche has a $5 billion chance to lead on drug prices

25 February 2019 - A deal for gene-therapy developer Spark could help reset what’s now an exorbitant corner of the market. ...

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ICER’s assessment finds Spinraza and Zolgensma provide substantial health benefits for people with spinal muscular atrophy

22 February 2019 - Current pricing of Spinraza would require a substantial discount to meet traditional cost-effectiveness ranges; Zolgensma’s value-based price ...

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Japan approves two gene treatments in bid to catch up

21 February 2019 - Potential revolutionary treatments have exorbitant price tags. ...

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Decision on spinal muscular atrophy treatment Spinraza deferred

18 February 2019 - PHARMAC's decision to defer a decision on funding the only treatment for spinal muscular atrophy (SMA) ...

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Sarepta announces FDA acceptance of golodirsen (SRP-4053) new drug application for patients with Duchenne muscular dystrophy amenable to skipping exon 53

14 February 2019 - FDA grants priority review status. ...

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RegenxBio receives rare paediatric disease designation for RGX-181 gene therapy for the treatment of CLN2 form of Batten disease

31 January 2019 - Novel, one-time investigational treatment for CLN2 disease designed to halt progression of this rare, paediatric, neurodegenerative disease. ...

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Statement from FDA Commissioner and Director of the Center for Biologics Evaluation and Research on new policies to advance development of safe and effective cell and gene therapies

15 January 2019 - The FDA is witnessing a surge of cell and gene therapy products entering early development, evidenced ...

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A revolutionary drug that could treat a rare and devastating disease is prohibitively expensive. But one state has a plan to pay for its potential $5 million price tag.

13 January 2019 - A one-time treatment for a devastating rare disease could be paid for with an instalment plan as ...

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