Rare Disease Day 2021: FDA shows sustained support of rare disease product development during the public health emergency

1 March 2021 - Rare Disease Day is a time to reflect on both the progress that has been made, and ...

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Recordati Rare Diseases: Carbaglu (carglumic acid) receives U.S. FDA approval for a new indication to treat acute hyperammonaemia associated with propionic acidemia and methylmalonic acidemia

26 January 2021 - Carbaglu is first and only FDA approved medication for hyperammonaemia associated with these rare conditions. ...

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Evaluating New Zealanders’ values for drug coverage decision-making: trade-offs between treatments for rare and common conditions

8 January 2021 - The objectives of this study were to measure the relative societal importance of values of New Zealanders ...

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IQWiG publishes 1,000th report

2 December 2020 - In the 16th year of its existence, IQWiG has published its 1,000th report: a dossier assessment ...

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Amicus Therapeutics initiates rolling biologic license application to the U.S. FDA for AT-GAA in late-onset Pompe disease

1 December 2020 - On track for completing the BLA submission in 1H2021. ...

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Alnylam announces innovative value-based agreement framework for Oxlumo (lumasiran) to accelerate access for patients with primary hyperoxaluria type 1 and deliver ultra-rare orphan disease pricing solutions to U.S. payers

24 November 2020 - Expedited access to Oxlumo aims to support children and adults living with PH1 who face inevitable disease ...

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FDA approves first drug to treat rare metabolic disorder

23 November 2020 - Today, the U.S. FDA approved Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria type 1, a ...

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Workshop on regulatory support for development of orphan medicines

23 November 2020 - On Monday, 30 November, EMA is hosting a workshop to discuss the benefits and impact of ...

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FDA approves first treatment for Hutchinson-Gilford progeria syndrome and some progeroid laminopathies

20 November 2020 - Today, the U.S. FDA approved Zokinvy (lonafarnib) capsules to reduce the risk of death due to Hutchinson-Gilford ...

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Mirum Pharmaceuticals broadens Expanded Access Program for maralixibat in Alagille syndrome to Europe and Australia

5 November 2020 - Maralixibat Expanded Access Program now available for patients with pruritus associated with Alagille syndrome in Australia and ...

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reVision Therapeutics announces US FDA grant of rare paediatric disease and orphan drug designation for REV-0100 for the treatment of Stargardt disease

28 October 2020 - reVision Therapeutics today announced that the US FDA has granted the Company's request to designate REV-0100 as ...

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Prevail Therapeutics receives U.S. FDA fast track designation for PR001 for the treatment of neuronopathic Gaucher disease

27 October 2020 - Prevail Therapeutics today announced that the U.S. FDA has granted fast track designation for the Company’s ...

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The estimation of health state utility values in rare diseases: overview of existing techniques

25 September 2020 - There are several techniques for estimating health state utility values, each of which presents pros and cons ...

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NICE U-turn sees Akcea's Waylivra win NHS funding

18 September 2020 - Akcea Therapeutics UK's Waylivra (volanesorsen), the first and only therapy for Familial Chylomicronaemia Syndrome (FCS), will ...

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FDA granted paediatric disease designation for OXi-4503

16 September 2020 - Treatment of acute myeloid leukaemia due to genetic mutations that disproportionately affect paediatric patients. ...

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