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New medicines #51 - May 1, 2021 - May 7, 2021
A weekly overview of the development of new medicines.
- BioLineRx released data from its phase 3 trial evaluating Motixafortide, in combination with granulocyte colony stimulating factor (G-CSF, the standard of care in this indication), for hematopoietic stem-cell mobilization for autologous bone marrow transplantation in multiple myeloma patients, met primary and secondary endpoints.
- Chemocentryx released FDA briefing documents raising concerns including “complexities of the study design, as detailed in the briefing document, raise questions about the interpretability of the data to define a clinically meaningful benefit of avacopan and its role in the management of AAV”:
The panel voted 9-9 that the efficacy data supported approval, and 10-8 that the benefit-risk profile is adequate to support approval
The difference between the votes became apparent later, when one panel member voted in favor in the latter vote only if the benefit-risk was further evident in a confirmatory trial
The PDUFA date for a final decision by the FDA is set for July 7, 2021
- Sarepta Therapeutics released data from Part A of its phase 2 Momentum trial of SRP-5051 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping:
The trial found that the 30 mg/kg monthly dose of SRP-5051 produced eight times (6.55% vs 0.86%) the dystrophin production as weekly doses of Exondys 51, its currently approved treatment for DMD
Source: BPC (reformatted), SY, Bioworld