Cell and gene therapies: FDA official on COVID-19 impact

10 June 2020 - Speaking at a session on cell and gene therapies at BIO Digital on Monday, a top US ...

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Passage Bio receives rare paediatric disease designation for PBGM01 for patients with GM1 gangliosidosis

21 May 2020 - PBGM01 to enter the clinic in fourth quarter of 2020. ...

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AveXis receives EC approval and activates “Day One” access program for Zolgensma, the only gene therapy for spinal muscular atrophy

19 May 2020 - Zolgensma (onasemnogene abeparvovec) is conditionally approved in Europe for the treatment of patients with spinal muscular atrophy ...

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AveXis receives positive CHMP opinion for Zolgensma, the only gene therapy for spinal muscular atrophy

28 March 2020 - AveXis, a Novartis company, today announced that the CHMP of the EMA has adopted a positive ...

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Alnylam announces approval of Givlaari (givosiran) in the European Union for the treatment of acute hepatic porphyria in adults and adolescents

3 March 2020 - Givlaari approved based on ENVISION Phase 3 study results showing a 74% reduction in the rate of ...

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MeiraGTx announces Priority Medicines (PRIME) and Advanced Therapy Medicinal Product designations granted by the EMA to AAV-RPGR gene therapy for the treatment of X-linked retinitis pigmentosa

2 March 2020 - PRIME designation granted based on clinical data from ongoing Phase 1/2 trial of AAV-RPGR. ...

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Translate Bio receives FDA fast track designation for MRT5005 for the treatment of cystic fibrosis

26 February 2020 - First inhaled mRNA therapeutic remains on track to report results from its ongoing multiple-ascending dose portion of ...

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Lysogene receives FDA fast track designation for LYS-SAF302 gene therapy in MPS IIIA

25 February 2020 - 17th patient dosed in the ongoing phase 2/3 AAVance study. ...

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BioMarin's biologics license application for valoctocogene roxaparvovec accepted for priority review by FDA with review action date of 21 August 2020

20 February 2020 - No advisory committee meeting currently planned to review the application. ...

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NDA accepted for filing by the FDA for antisense oligonucleotide viltolarsen

7 February 2020 - Nippon Shinyaku announced today that the U.S. FDA has accepted the filing of our new drug ...

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Orsini Pharmaceutical Services now providing FDA approved Vyondys 53 (golodirsen) injection for Duchenne muscular dystrophy

5 February 2020 - Commercial distribution of Vyondys 53 in the U.S. is currently underway. ...

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Alnylam receives positive CHMP opinion for Givlaari (givosiran) for the treatment of acute hepatic porphyria in adults and adolescents

31 January 2020 - Positive opinion is based on data from the pivotal ENVISION Phase 3 study. ...

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Highlights from the CHMP 27-30 January 2020 meeting

31 January 2020 - Fifteen new medicines recommended for approval. ...

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ProQR receives rare paediatric disease designation from FDA for QR-421a

30 January 2020 - Designation is for the treatment of patients with retinitis pigmentosa caused by mutations in exon 13 ...

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With new rules, FDA aims to spark competition, lower prices for expensive gene therapies

28 January 2020 - The FDA released a slew of gene therapy policies Tuesday aimed at encouraging drug makers to ...

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