J&J’s Darzalex Faspro is first US therapy for rare blood disorderJohnson & Johnson’s Darzalex Faspro has become the first product approved by the FDA to treat light chain Share XJ&J’s Darzalex Faspro is first US therapy for rare blood disorderhttps://pharmaphorum.com/news/jjs-darzalex-faspro-is-first-us-therapy-for-rare-blood-disorder/
Sarepta helps bankroll new gene therapy startup AavantiBioSarepta chief commercial officer Bo Cumbo has left to head up gene therapy venture – AavantiBio – with Share XSarepta helps bankroll new gene therapy startup AavantiBiohttps://pharmaphorum.com/news/sarepta-helps-bankroll-new-gene-therapy-startup-aavantibio/
Chord gets $16m to challenge Alexion with oral NMOSD drugSwiss biotech Chord Therapeutics has come out of stealth mode with a $16 million first-round financing that will Share XChord gets $16m to challenge Alexion with oral NMOSD drughttps://pharmaphorum.com/news/chord-gets-16m-to-challenge-alexion-with-oral-nmosd-drug/
EMA starts rapid review of Bluebird’s gene therapy for rare disease CALDBluebird bio could be just a few months away from approval of its gene therapy for rare disease Share XEMA starts rapid review of Bluebird’s gene therapy for rare disease CALDhttps://pharmaphorum.com/news/ema-starts-rapid-review-of-bluebirds-gene-therapy-for-rare-disease-cald/
Novartis’ bid to expand use of SMA gene therapy delayed after FDA trial requestAnalysts have said the FDA’s request for a new trial will delay Novartis’ intrathecal formulation of its spinal Share XNovartis’ bid to expand use of SMA gene therapy delayed after FDA trial requesthttps://pharmaphorum.com/news/novartis-bid-to-expand-use-of-sma-gene-therapy-delayed-after-fda-trial-request/
NICE relents and okays Akcea’s rare disease drug Waylivra for NHS usePatients with the rare disease familial chylomicronemia syndrome (FCS) will be able to access treatment with Akcea’s Waylivra Share XNICE relents and okays Akcea’s rare disease drug Waylivra for NHS usehttps://pharmaphorum.com/news/nice-relents-and-okays-akceas-rare-disease-drug-waylivra-for-nhs-use/
Regeneron’s cholesterol drug evinacumab claims February FDA dateThe FDA has started a speedy review of Regeneron’s ANGPTL3-targeting antibody evinacumab for a rare, inherited disorder that Share XRegeneron’s cholesterol drug evinacumab claims February FDA datehttps://pharmaphorum.com/news/regenerons-cholesterol-drug-evinacumab-claims-february-fda-date/
Chiesi’s flagship Fabry drug heads for FDA verdict in early 2021The FDA has started its review of Israeli biotech Protalix BioTherapeutics and partner Chiesi’s Fabry disease therapy pegunigalsidase Share XChiesi’s flagship Fabry drug heads for FDA verdict in early 2021https://pharmaphorum.com/news/chiesis-flagship-fabry-drug-heads-for-fda-verdict-in-early-2021/
Mallinckrodt’s liver disease drug terlipressin edges FDA advisory panelAn FDA advisory committee has narrowly voted in favour of approval of Mallinckrodt’s terlipressin for hepatorenal syndrome type Share XMallinckrodt’s liver disease drug terlipressin edges FDA advisory panelhttps://pharmaphorum.com/news/mallinckrodts-liver-disease-drug-terlipressin-edges-fda-advisory-panel/
