By the end of the quarter, the US Food and Drug Administration (FDA) had racked up 39 innovative drugs approvals, already exceeding last year’s total of 37. Among them are two bispecific antibodies redirecting T cells via the CD3 antigen to overexpressed tumor markers in multiple myeloma that received accelerated approvals in August: Talvey (talquetamab) from Johnson & Johnson links CD3 to GPCRC5D while Elrexfio from Pfizer links CD3 to BCMA. Extended approval for both is contingent on the results of follow-up studies. The end of the quarter saw another step in John Crowley’s journey to develop medicines for Pompe disease with the FDA approval of Amicus Therapeutics’ Pombiliti + Opfolda as a second enzyme replacement therapy (ERT). Crowley ran Genzyme’s early development program for the first ERT, Lumizyme (alglucosidase alfa), before leaving the company to enable two of his children with the disease to enter the trials. Pombiliti is an α-glucosidase variant designed for high muscle uptake while Opfolda reduces loss of enzyme activity in the blood.
The first quarter of 2024 sees reviews at FDA and European Medicines Agency (EMA) of the first CRISPR therapy for sickle cell disease, exagamglogene autotemcel from Vertex Pharmaceuticals and CRISPR Therapeutics. It gene edits the BCL11A gene, switching it off and allowing the production of fetal hemoglobin to compensate for the deficiencies in adult hemoglobin that occur in thalassemia and sickle cell disease. Patients with amyotrophic lateral sclerosis (ALS) may be facing another disappointment, as the FDA advisory committee was unimpressed with the performance of BrainStorm Cell Therapeutics’ NurOwn, a neurotrophic-factor-secreting mesenchymal bone marrow stromal cell therapy.
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