Canadian baby with spinal muscular atrophy to get ‘world’s most expensive drug’

20 October 2020 - A little over a year ago, the idea of raising US$2.125 million to receive the “world’s ...

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Highlights from 12-15 October CHMP meeting

16 October 2020 - Ten new medicines recommended for approval. ...

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Novartis receives positive CHMP opinion for Leqvio (inclisiran), a potential first-in-class siRNA for the treatment of high cholesterol

16 October 2020 - If approved, inclisiran will be the first and only small interfering RNA (siRNA) in Europe for patients ...

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First treatment for rare condition primary hyperoxaluria type 1

16 October 2020 - EMA has recommended granting a marketing authorisation in the European Union for Oxlumo (lumasiran) for the ...

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Health Canada approves first-ever gene replacement therapy, Luxturna

15 October 2020 - Luxturna (voretigene neparvovec), is the first approved therapy for previously untreatable inherited retinal disease. ...

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Taysha Gene Therapies receives rare paediatric disease designation and orphan drug designation for TSHA-102 as a treatment for Rett syndrome

14 October 2020 - Program leverages novel miRARE platform technology used to control transgene expression on a cellular basis. ...

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Axovant Gene Therapies receives rare paediatric disease designation for AXO-AAV-GM1 for GM1 gangliosidosis

9 October 2020 - Company on-track to report data from on-going Phase 1/2 clinical study in Q4 2020. ...

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bluebird bio announces European Medicines Agency’s acceptance of marketing authorisation application for elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy for cerebral adrenoleukodystrophy

2 October 2020 - European Medicines Agency will evaluate eli-cel application under accelerated assessment. ...

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BioMarin receives FDA fast track designation for investigational gene therapy, BMN 307

2 October 2020 - Second investigational gene therapy in clinic, potential third therapy in PKU franchise. ...

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Pfizer receives FDA fast track designation for Duchenne muscular dystrophy investigational gene therapy

1 October 2020 - Pfizer today announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy ...

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bluebird bio’s LentiGlobin for sickle cell disease gene therapy (bb1111) granted priority medicines (PRIME) designation by European Medicines Agency

23 September 2020 - EMA’s PRIME program designed to optimise development and expedite evaluation of innovative medicines for patients with high ...

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FDA grants Spirovant Sciences orphan drug and rare paediatric disease designations for SPIRO-2101 for treatment of cystic fibrosis

24 September 2020 - Spirovant Sciences today announced that the U.S. FDA has granted orphan drug and rare paediatric disease ...

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Gyroscope Therapeutics granted FDA fast track designation for GT005, an investigational gene therapy for dry age-related macular degeneration

22 September 2020 - Gyroscope Therapeutics today announced that the U.S. FDA has granted fast track designation to GT005 for the ...

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Human Rights Commission grants case against PHARMAC for failing to fund rare muscular disorder drug

16 September 2020 - Newshub can reveal a ground-breaking Human Rights Commission case has been taken against Pharmac for failing ...

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GenSight Biologics submits EU marketing authorisation application for Lumevoq gene therapy to treat vision loss due to Leber hereditary optic neuropathy

15 September 2020 - First marketing authorisation application for a gene therapy treating a mitochondrial disease. ...

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