Novartis agrees to buy Avidity Biosciences for $12bn
Novartis has signed an agreement for the acquisition of all outstanding shares of US-based Avidity Biosciences for $12bn in cash…
Genetic Disorders
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Harmony’s cannabidiol drug flops in pivotal Fragile X Syndrome trial
Harmony Biosciences’ transdermal cannabidiol ZYN002 suffered a setback as a Phase III trial failed to meet the primary endpoint in…
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Stealth Bio’s Forzinity finally approved as first Barth syndrome therapy
After a rocky regulatory pathway filled with delays and a previous rejection, the US Food and Drug Administration (FDA) has…
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Ionis and Sobi’s Tryngolza gains approval in EU for FCS treatment
Ionis Pharmaceuticals and Sobi have announced the approval of Tryngolza (olezarsen) in the European Union (EU) for adults with familial…
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Health Canada extends Evkeeza approval for children with HoFH
Ultragenyx Pharmaceuticals has announced the extension of approval by Health Canada for Evkeeza (evinacumab) as an adjunct to diet and…
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FDA approves KalVista’s Ekterly for hereditary angioedema
The US Food and Drug Administration (FDA) has approved KalVista Pharmaceuticals' Ekterly (sebetralstat) as the first and only oral on-demand…
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Vertex wins European approval for Alyftrek, bolstering cystic fibrosis stronghold
Vertex Pharmaceuticals has won approval in Europe for its next-generation cystic fibrosis therapy Alyftrek (deutivacaftor/tezacaftor/vanzacaftor), shoring up the company’s dominance…
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Newborn screening strategy needs modernising as HHS disbands advisory committee
After sweeping cuts to US federal health agencies, the elimination of a committee responsible for recommending newborn screening regulations is…
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Sanofi’s Qfitlia approved by FDA as first haemophilia therapy for all patients
Sanofi has won US Food and Drug Administration (FDA) approval for haemophilia treatment Qfitlia (fitusiran), as the company aims to…
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FDA approves Gomekli as first neurofibromatosis drug for adults
The US Food and Drug Administration (FDA) has approved SpringWorks Therapeutics’ Gomekli (mirdametinib) to treat neurofibromatosis type 1 (NF1), a…
