A lifesaving therapy for children with a rare disease is now the world’s most expensive drug, raising questions about access

20 March 2024 - A new gene therapy for the fatal genetic disorder metachromatic leukodystrophy, or MLD, will carry a ...

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PTC Therapeutics provides key regulatory updates (March 2024)

19 March 2024 - BLA submitted to FDA for Upstaza for the treatment of AADC deficiency - ...

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FDA approves first gene therapy for children with metachromatic leukodystrophy

18 March 2024 - Today, the US FDA approved Lenmeldy (atidarsagene autotemcel), the first FDA approved gene therapy indicated for the ...

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PepGen receives US FDA orphan drug and rare paediatric disease designations for PGN-EDO51 for the treatment of Duchenne muscular dystrophy

13 March 2024 - PepGen today announced that the US FDA granted both orphan drug and rare paediatric disease designations for ...

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Medera’s Sardocor announces fast track designation and dosing of 3 patients in first in human HFpEF gene therapy trial

14 February 2024 - In the Phase 1/2A clinical trial for SRD-001, an adeno-associated virus mediated first in human gene therapy ...

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Sarepta Therapeutics announces US FDA acceptance of an efficacy supplement to expand the Elevidys indication

16 February 2024 - Priority review granted, with a review goal date of 21 June 2024. ...

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Rocket Pharmaceuticals announces update on FDA review timeline of Kresladi (marnetegragene autotemcel) for the treatment of severe leukocyte adhesion deficiency-I

13 February 2024 - New Prescription Drug User Fee Act date of 30 June 2024. ...

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Eplontersen granted US FDA fast track designation for patients with transthyretin-mediated amyloid cardiomyopathy

8 February 2024 - Ionis Pharmaceuticals announced today that the US FDA has granted fast track designation to Ionis and ...

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Ascidian Therapeutics announces fast tracks ACDN-01 in Stargardt disease and other ABCA4 retinopathies

29 January 2024 - Ascidian Therapeutics today announced that the US FDA has granted fast track ...

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Atsena Therapeutics receives rare paediatric disease designation from FDA for ATSN-101 gene therapy for GUCY2D associated Leber congenital amaurosis

16 January 2023 - Atsena Therapeutics today announced the US FDA has granted rare paediatric disease designation to ATSN-101, the company’s ...

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Vertex announces US FDA approval of Casgevy (exagamglogene autotemcel) for the treatment of transfusion-dependent beta thalassaemia

16 January 2024 - Approximately 1,000 patients in the US 12 years of age and older are now eligible for ...

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New medical treatments will use genetic scissors, and other clever tricks

13 November 2023 - New medicines to treat sickle cell disease and beta thalassaemia, two genetic blood disorders, will make headlines ...

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Sarepta Therapeutics submits efficacy supplement to expand the Elevidys label to include Duchenne muscular dystrophy patients without restriction to age or ambulatory status

22 December 2023 - Sarepta has also submitted the EMBARK post-marketing requirement to the FDA seeking conversion of the Elevidys accelerated ...

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Wainua (eplontersen) granted first ever regulatory approval in the US for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis

21 December 2023 - US FDA approval based on NEURO-TTRansform Phase 3 results showing Wainua demonstrated consistent and sustained benefit improving ...

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Ocugen announces OCU400 receives regenerative medicine advanced therapy designation for treatment of retinitis pigmentosa associated with RHO mutations

19 December 2023 - Ocugen today announced that the FDA has granted regenerative medicine advanced therapy designation to Ocugen’s investigational product ...

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