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NICE draft guidance does not recommend risdiplam for treating spinal muscular atrophy

2 June 2021 - NICE has today published draft guidance for public consultation which does not recommend risdiplam (Evrysdi, Roche) for ...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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Scholar Rock receives fast track designation from the U.S. FDA for apitegromab for the treatment of patients with spinal muscular atrophy

24 May 2021 - Builds on Priority Medicines (PRIME) designation recently granted by the EMA recognising the unmet medical needs of ...

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Apellis announces U.S. FDA approval of Empaveli (pegcetacoplan) for adults with paroxysmal nocturnal hemoglobinuria

14 May 2021 - Empaveli was superior to Soliris for the change from baseline in haemoglobin level at Week 16 in ...

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Fulcrum Therapeutics announces U.S. FDA grants fast track designation to losmapimod for the potential treatment of facioscapulohumeral muscular dystrophy

12 May 2021 - Fulcrum Therapeutics today announced that the U.S. FDA has granted fast track designation to losmapimod for ...

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Gilead readies to sell anticancer drug Trodelvy in Korea

4 May 2021 - Gilead’s antibody-drug conjugate Trodelvy (sacituzumab govitecan) has won the Ministry of Food and Drug Safety’s designation ...

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Vertex announces European Commission approval for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor to treat cystic fibrosis patients 12 years and older with at least one F508del mutation in the CFTR gene

28 April 2021 - New indication includes people ages 12 years and older who have one copy of the F508del mutation ...

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Biogen to expand access to its ALS drug, but move may come too late for some patient

27 April 2021 - After weeks of controversy, Biogen has agreed to provide an experimental drug for combating ALS to ...

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Alnylam submits new drug application with U.S. FDA for vutrisiran for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults

19 April 2021 - Alnylam has submitted a new drug application to the U.S. FDA for the approval of vutrisiran for ...

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Citrine Medicine and Sinopharm Group announce strategic partnership to broaden and accelerate access to rare disease drugs in China

19 April 2021 - Citrine Medicine and Sinopharm Group today announced a strategic partnership to accelerate the commercialisation of rare ...

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Masterton woman feels abandoned after medical funding rejected

9 April 2021 - Masterton's Allyson Lock feels she is not part of the "team of five million" after her ...

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