Bluebird bio placed the company’s Phase I/II and Phase III trials of LentiGlobin gene therapy for sickle cell disease (SCD) on temporary suspension.
At the 2021 virtual JP Morgan Healthcare Conference, Vertex Pharmaceuticals’ chief executive officer Reshma Kewalramani said the company is looking to buy “mid- and late-stage assets.”
The American Society of Hematology (ASH) Annual Meeting & Exposition began December 5 with numerous presentations, abstracts and posters.
Shares of bluebird bio fell in trading after the company announced that the sickle cell disease gene therapy treatment bb1111 will not be submitted to the U.S. Food and Drug Administration for two more years.
The Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion recommending conditional marketing authorization of Novartis’ Adakveo (crizanlizumab) for the prevention of recurrent vaso-occlusive crises, or pain crises, in patients with sickle cell disease aged 16 years and older.
Sickle cell disease drugs made by Novartis and Global Blood Therapeutics (GBT) may not be cost effective at current prices, a draft report published on Friday suggested, widening the debate over U.S. healthcare affordability.
South San Francisco-based Global Blood Therapeutics struck a deal with Syros Pharmaceuticals that is worth up to $375 million to discover, develop and commercialize novel therapies for the two types of blood disorders.
The U.S. Food and Drug Administration approved Oxbryta (voxelotor), a drug from Global Blood Therapeutics Inc. developed to treat sickle cell disease in adults and children 12 years or older.
Novartis AG won U.S. approval for the company’s experimental sickle cell disease drug Adakveo.
Pfizer announced that the company’s Phase III RESET clinical trial evaluating rivipansel in sickle cell disease (SCD) failed to meet the study’s primary and key secondary efficacy endpoints.