Rare diseases

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.

Despite substantial variability in the presented data and no well-controlled trial, the FDA advisory committee voted in favor of Stealth BioTherapeutics’ Barth syndrome therapy elamipretide, citing the urgent unmet need.

The pediatric patients, with a rare neurodegenerative disease, were treated with bluebird bio’s Skysona to slow the progression of neurologic dysfunction. Six patients developed myelodysplastic syndrome and one patient developed acute myeloid leukemia.

The FDA’s reviewers pointed out that Stealth’s elamipretide missed its primary efficacy endpoints in the main study used to establish its effectiveness.

The rejection is related to the timing of the FDA’s reinspection of Zealand’s third-party manufacturer, which previously had deficiencies.

With Monday’s data from SAPPHIRE, Scholar Rock is building toward regulatory submissions for apitegromab in spinal muscular atrophy in the first quarter of 2025.

Under the deal announced Friday, the Italian pharma will make an upfront payment of $825 million to Sanofi for global rights to a biologic for the treatment of cold agglutinin disease, with milestone payments of up to $250 million.

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.

One upcoming decision—on a perioperative PD-1 regimen for lung cancer—comes as the FDA considers an overhaul of trial designs in this treatment setting.