Mum says 'miracle' drug saved ill baby Kaan Hunter's life

21 January 2020 - A baby who was given only four months to live after being diagnosed with an extremely ...

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FDA harnesses technology and collaboration to support rare disease product development

17 January 2020 - Announcing an orphan drug technology modernisation effort and the 2020 FDA Rare Disease Day meeting. ...

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Asfotase alfa in hypophosphatasia in children and adolescents: survival advantage for small children

15 January 2020 - Weak evidence does not allow a specific statement to be made about the extent of the added ...

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Does an orphan drug policy make a difference in access? A comparison of Canada and Australia.

2 January 2020 - Canada has been discussing whether to implement an orphan drug policy for more than 25 years. Recently, ...

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Vertex announces European Commission approval for Kalydeco (ivacaftor) in infants with cystic fibrosis ages 6 months to less than 12 months with certain mutations in the CFTR gene

10 December 2019 - Ivacaftor is the first and only approved medicine in Europe to treat the underlying cause of cystic ...

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Our drug policy often prioritises eliminating rare disease. One researcher asks: Is that the right goal?

26 November 2019 - Dr. Peter Bach wants to turn the entire philosophical underpinnings of America’s health care system upside ...

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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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