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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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. ...

Read more →

Orphazyme announces U.S. FDA acceptance and priority review of new drug application for arimoclomol for Niemann-Pick disease Type C

16 September 2020 - If approved, arimoclomol would become the first approved therapy in the U.S. for people with Niemann-Pick disease ...

Read more →

South Korea to shorten licensing review period new drugs on rare diseases by 25 days

8 September 2020 - Korean health regulators will shorten licensing review period from 115 days to 90 days for investigational ...

Read more →

Impact of rarity on Canadian oncology health technology assessment and funding

11 August 2020 - The pan-Canadian Oncology Drug Review evaluates new cancer drugs for public funding recommendations.  ...

Read more →

Rare disease drugs strategy, standard coverage in CLHIA’s 2020 budget wish list

12 August 2020 - The Canadian Life and Health Insurance Association is recommending the federal government develop a strategy for ...

Read more →

Rare disease drove two women on a mission to change FDA drug review process

19 July 2020 - Melissa Goetz and Lindsey Sutton are campaigning to improve the US drug review process for rare diseases. ...

Read more →