European Medicines Agency validates BioMarin's marketing authorisation application for valoctocogene roxaparvovec to treat severe haemophilia A

23 December 2019 - Potential first gene therapy in Europe directed at any type of haemophilia. ...

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'Healthy lottery' fears as Novartis plans to give away world’s most expensive drug

19 December 2019 - Novartis aims to give away 100 doses of its $US2.1 million-per-patient ($3.05 million) Zolgensma for spinal ...

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Rocket Pharmaceuticals receives EMA PRIME eligibility for RP-L102 gene therapy for Fanconi anaemia

16 December 2019 - Milestone gives Fanconi anaemia program all accelerated regulatory tools in U.S. and EU, including FDA regenerative medicine ...

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Spinraza funding application reviewed by the Rare Disorders Subcommittee

17 December 2019 - PHARMAC has published the Rare Disorders Subcommittee meeting records which recommends Spinraza (nusinersen), a medication for ...

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FDA grants accelerated approval to first targeted treatment for rare Duchenne muscular dystrophy mutation

12 December 2019 - The U.S. FDA today granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy ...

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Sarepta Therapeutics announces FDA approval of Vyondys (golodirsen) injection for the treatment of Duchenne muscular dystrophy in patients amenable to skipping exon 53

12 December 2019 -  Vyondys 53 is Sarepta’s second RNA exon-skipping treatment for DMD approved in the U.S. ...

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Value-based pricing for gene therapy? Maybe not ready for haemophilia.

12 December 2019 - Insurers grappling with the million-dollar-plus costs of gene therapy have touted "value-based" arrangements that allow payment ...

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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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ICER to assess gene therapy for beta thalassaemia

5 December 2019 - Report will be subject of New England CEPAC meeting in July 2020; open Input now being accepted ...

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Family asking for help to purchase 'the most expensive drug in the world'

1 December 2019 - Ricardo Batista is desperately hoping for a cure for SMA, for his daughter Eva. ...

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FDA grants priority review to Roche’s risdiplam for spinal muscular atrophy

25 November 2019 - Filing submission includes 12-month data from pivotal FIREFISH and SUNFISH trials in a broad population of people ...

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Revised process for cell and gene therapies

21 November 2019 - CADTH has undertaken an internal review and established a novel process for the review of cell and ...

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BioMarin submits marketing authorisation application to EMA for valoctocogene roxaparvovec to treat severe haemophilia A

21 November 2019 - First marketing application submission for gene therapy directed at any type of haemophilia. ...

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Drug for ultra rare disease comes with ultra high price tag

21 November 2019 - Givlaari’s net price will be $442,000 after discounts included. ...

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FDA approves first treatment for inherited rare disease

20 November 2019 - Today, the U.S. FDA granted approval to Givlaari (givosiran) for the treatment of adult patients with acute ...

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