In a recent late-stage clinical trial, Sarepta Therapeutics’ gene therapy designed to treat Duchenne muscular dystrophy (DMD), a progressive muscle-wasting genetic disorder, failed to achieve its primary goal when administered to patients aged 4 to 7 years. Specifically, the therapy did not demonstrate statistical significance in the total assessment score of motor function when compared to patients who received a placebo over a 52-week period. DMD primarily affects young boys and typically results in a limited life expectancy beyond one’s thirties.
The gene therapy, known as Elevidys, had previously received accelerated approval from the U.S. health regulator for children aged 4 to 5 years who can walk, as opposed to the initial application seeking approval for all walking DMD patients. The company highlighted that Elevidys did meet all secondary study objectives with statistically significant outcomes and did not show any new safety concerns.
Sarepta plans to request an update from the U.S. Food and Drug Administration (FDA) to expand the therapy’s usage to other age groups based on the trial findings. Elevidys is a one-time treatment, representing a potential shift in the treatment paradigm for DMD patients, as current therapies necessitate ongoing use. Of the four “exon-skipping” therapies intended for a specific subset of DMD patients with specific genetic mutations, three are developed by Sarepta. These therapies require weekly infusions and function by skipping specific gene segments, known as exons, in order to promote the formation of shorter dystrophin protein forms.
Roche holds commercial rights to Elevidys outside the United States, while Sarepta retains responsibility for clinical development and shares the associated costs with Roche.
The disappointing results from Sarepta Therapeutics’ trial have led to a significant drop in the company’s stock value. On the other hand, it raised questions about how regulators handle gene therapies with unclear outcomes but high medical needs.
https://endpts.com/sareptas-confirmatory-duchenne-gene-therapy-study-fails-to-meet-primary-endpoint/
https://endpts.com/sareptas-duchenne-confirmatory-trial-results-set-up-showdown-between-fda-and-companys-bold-talk/
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
Related News
Exploring Tau Protein’s Role in Glaucoma: New Insights and Therapeutic Potential
Glaucoma, a chronic neurodegenerative disorder, leads to irreversible vision loss by damaging retinal ganglion cells (RGCs) and the optic nerve, often associated with increased intraocular pressure (IOP). Despite the benefits of IOP-lowering treatments, the underlying...
FDA-mandated CAR-T monitoring period could be halved, say researchers
In patients with diffuse large B-cell non-Hodgkin lymphoma (DLBCL), the two hallmark post-chimeric antigen receptor (CAR)-T therapy toxicities are extremely rare after two weeks, supporting a shorter, more flexible toxicity monitoring period, according to a study...
Ancestral CRISPR-Cas13 Ribonucleases Discovered: Implications for Genome Editing
In a pioneering study published in *Science*, a team of researchers led by Peter H. Yoon and Jennifer A. Doudna from the University of California, Berkeley, has made a remarkable discovery in the realm of CRISPR technology. The team has identified an ancestral clade...
KBI Biopharma Expands Manufacturing Contract with Global Pharmaceutical Company
KBI Biopharma Inc., a JSR Life Sciences company and global cGMP contract development and manufacturing organization (CDMO), has extended and expanded its manufacturing contract with a leading global pharmaceutical company. Originally initiated in 2020, the renewed...
Related Services