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/
NICE backs NHS use of Takeda’s Adcetris for rare blood cancerTakeda’s antibody-drug conjugate Adcetris can now be routinely available on the NHS to patients with a rare form Share XNICE backs NHS use of Takeda’s Adcetris for rare blood cancerhttps://pharmaphorum.com/news/nice-backs-nhs-use-of-takedas-adcetris-for-rare-blood-cancer/
Sanofi hands back gene therapies to Oxford BiomedicaOxford Biomedica’s long-running alliance with Sanofi over a pair of gene therapies for ophthalmic diseases has come to Share XSanofi hands back gene therapies to Oxford Biomedicahttps://pharmaphorum.com/news/sanofi-hands-back-gene-therapies-to-oxford-biomedica/
