Aduhelm backlash threatens to reverse progress in FDA’s reviews of rare and ultra-rare disease drugs

14 September 2021 - The FDA’s approval of Aduhelm to treat Alzheimer’s disease has unleashed criticism about the decision and ...

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Albireo receives UK MHRA approval of Bylvay (odevixibat)

8 September 2021 - Bylvay now approved in U.S., EU and UK as first drug treatment for patients with progressive familial intrahepatic ...

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FDA, drug makers propose new pilots aimed at speeding rare disease drugs, as part of user fee update

23 August 2021 - The FDA and the drug industry have jointly agreed to create several new pilot programs aimed ...

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Health Canada releases What We Heard Report from the public engagement on the National Strategy for Drugs for Rare Diseases

26 July 2021 - Too many Canadians are struggling to access the drugs they need, especially if they have a rare ...

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bluebird bio receives EC approval for Skysona (elivaldogene autotemcel, Lenti-D) gene therapy for patients less than 18 years of age with early cerebral adrenoleukodystrophy without matched sibling donor

21 July 2021 - Skysona is the first and only gene therapy approved in the European Union to treat early CALD. ...

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PHARMAC 'cruel' for failing to fund drugs for rare illnesses — advocate

14 July 2021 - New Zealand’s drug-buying agency doesn’t value the lives of all people equally, according to those advocating for ...

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LEXEO Therapeutics receives rare paediatric disease designation and orphan drug designation for LX2006 for the treatment of Friedreich’s ataxia

30 June 2021 - Phase I/II clinical trial in patients with cardiomyopathy associated with Friedreich’s ataxia expected to initiate in 2021. ...

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AskBio receives FDA fast track designation for LION-101, a novel investigational AAV gene therapy for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9)

28 June 2021 - LGMD2I/R9 is a rare form of muscular dystrophy with no approved therapies. ...

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BioMarin receives positive CHMP opinion in Europe for vosoritide for the treatment of children with achondroplasia from age 2 until growth plates close

25 June 2021 - Temporary Authorization for Use (ATU) granted in France to allow access and reimbursement of vosoritide to begin ...

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FDA should lead the way on new ALS treatments, not Canada and Europe

22 June 2021 - Following the FDA’s recent decision to give the green light to aducanumab, the first treatment approved ...

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Rare disease patient advocates continue battle for affordable drugs

23 June 2021 - Beth Vanstone, mother of local Cystic Fibrosis Warrior Madi Vanstone and director of the CF Get ...

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Orphazyme provides regulatory update from FDA on arimoclomol for Niemann-Pick disease type C

18 June 2021 - Orphazyme today announced it has received a complete response letter from the U.S. FDA following its ...

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NICE draft guidance does not recommend risdiplam for treating spinal muscular atrophy

2 June 2021 - NICE has today published draft guidance for public consultation which does not recommend risdiplam (Evrysdi, Roche) for ...

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Examining the impact of different country processes for appraising rare disease treatments: a case study analysis

31 May 2021 - Conventional appraisal and reimbursement processes are being challenged by the increasing number of rare disease treatments with ...

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Ipsen confirms U.S. FDA accepts new drug application for palovarotene as the first potential treatment worldwide for fibrodysplasia ossificans progressiva

28 May 2021 - New drug application granted priority review status, with a decision anticipated on 30 November 2021. ...

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