Funding orphan medicinal products beyond price: sustaining an ecosystem

18 November 2019 - Following a similar legislation in the USA, the EU passed a legislation in 1999 to incentivise ...

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Rare Disease Fund now covers Pompe disease, a rare inherited neuromuscular disorder

3 November 2019 - The Rare Disease Fund now covers Singaporeans with Pompe disease - a rare inherited neuromuscular disorder ...

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FDA admits it goofed when granting orphan status to an opioid addiction treatment

8 November 2019 - In an unusual move, the FDA has acknowledged a mistake and revoked orphan drug status for ...

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Vertex announces EMA marketing authorisation application validation for VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination treatment in cystic fibrosis

31 October 2019 - Application supported by positive results from two global Phase 3 studies in people with cystic fibrosis ages ...

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Battle for life-saving drugs now harder as PHARMAC absorbs $5m rare disorders fund

29 October 2019 - A $5 million fund set up by PHARMAC in 2014 to address rare disorders no longer ...

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NHSE signs ‘definitive’ agreement for Vertex CF drugs

24 October 2019 - NHS England has announced a definitive agreement with Vertex Pharmaceuticals to make all three of their ...

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FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases

8 October 2019 - The U.S. FDA today announced that it has awarded 12 new clinical trial research grants totaling ...

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Mum 'hopeful' after baby Kaan Hunter starts $250k a year medication for rare bone disease

5 October 2019 - A mum whose baby was the first Kiwi to be diagnosed with a rare bone disease ...

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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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