Sickle cell disease
In the wake of Pfizer’s voluntary market withdrawal of the popular sickle cell disease therapy, BioSpace looks at five investigational drugs currently making their way through the pipeline.
While ex vivo genome editing results in highly effective cell therapies, it can lead to off-target effects. Caribou Biosciences has come up with a novel approach for potentially more precise gene editing compared to all-RNA guides.
Since its inception in 1992, the FDA’s accelerated approval pathway has helped shepherd nearly 300 new drugs to the market. However, recent years have seen a number of high-profile market withdrawals and failed confirmatory trials.
Bristol Myers Squibb wins approval for the first novel schizophrenia drug in decades; Pfizer pulls Oxbryta from the market; new IVF and abortion laws could derail women’s health research; Roche touts CDK inhibitor deal and obesity pipeline and BioSpace heads to Meeting on the Mesa.
Pfizer’s sudden market withdrawal of sickle cell therapy Oxbryta, which some analysts predicted would reach $750 million in sales by the end of the decade, has left patients and healthcare providers with few options, while investors question the pharma giant’s dealmaking prowess.
Based on new safety signals, Pfizer on Wednesday announced that Oxbryta’s overall benefit “no longer outweighs the risk” in patients with sickle cell disease. Guggenheim Securities analysts said the setback increases investor frustrations with the company’s business development track record.
No patients have received Casgevy, CRISPR Therapeutics and Vertex Pharmeceuticals’ recently approved sickle cell gene therapy. Experts weigh in on the path to profit for the treatment and the therapeutic class in general.
Vertex has filed a complaint against the Department of Health and Human Services, seeking to make its fertility preservation program available to federally insured patients needing Casgevy treatment.
With 15 patients started across its three gene therapies, bluebird bio claims a 138% year-over-year revenue growth and aims to initiate up to around 100 new patients in the current year.
While Pfizer has ended one of its two Phase III studies for inclacumab in sickle cell disease, the company is still eyeing an approval for the antibody in the inherited blood disorder by 2026.
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