Blog
RSS FeedPosted by Michael Wonder on 18 Mar 2019
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 ...
Posted by Michael Wonder on 10 Mar 2019
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 ...
Posted by Michael Wonder on 06 Mar 2019
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 ...
Posted by Michael Wonder on 04 Mar 2019
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 ...
Posted by Michael Wonder on 04 Mar 2019
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. ...
Posted by Michael Wonder on 03 Mar 2019
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. ...
Posted by Michael Wonder on 27 Feb 2019
'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 ...
Posted by Michael Wonder on 25 Feb 2019
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. ...
Posted by Michael Wonder on 22 Feb 2019
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 ...
Posted by Michael Wonder on 21 Feb 2019
Japan approves two gene treatments in bid to catch up
21 February 2019 - Potential revolutionary treatments have exorbitant price tags. ...
Posted by Michael Wonder on 18 Feb 2019
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) ...
Posted by Michael Wonder on 15 Feb 2019
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. ...
Posted by Michael Wonder on 11 Feb 2019
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. ...
Posted by Michael Wonder on 15 Jan 2019
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 ...
Posted by Michael Wonder on 14 Jan 2019
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 ...