Capricor Therapeutics’ investigational cell therapy, deramiocel, for cardiomyopathy associated with Duchenne muscular dystrophy (DMD-CM), has been turned down by the FDA. The stock plunged 40% on the news.
In its Complete Response Letter (CRL), the FDA cited a lack of “substantial evidence of effectiveness” and requested additional clinical data. The CRL also noted Chemistry, Manufacturing, and Controls (CMC) deficiencies, which Capricor claims it had previously addressed but were not reviewed due to the CRL’s early issuance. The PDUFA date for deramiocel was set for August 30, making this an early rejection.
This decision comes amidst an internal FDA clash surrounding deramiocel. Reports from STAT News indicated that the former head of CBER’s Office of Therapeutic Products, Nicole Verdun, had scheduled an advisory committee meeting for deramiocel, which was then unilaterally canceled by CBER head Vinay Prasad, leading to the departure of Verdun and her deputy.
Capricor CEO Linda Marbán expressed surprise at the rejection, stating the company had followed FDA guidance throughout the process. Capricor plans to resubmit its application, incorporating upcoming data from the Phase III HOPE-3 trial, expected in Q3.
Deramiocel is composed of allogeneic cardiosphere-derived cells (CDCs), which are believed to reduce fibrosis, modulate immune response, and preserve cardiac and skeletal muscle. The initial application was supported by Phase II HOPE-2 data, which showed improvements in upper-limb strength and sustained heart function in DMD patients over three years.
Source:
https://www.biospace.com/fda/capricor-plunges-on-fda-rejection-of-dmd-cell-therapy
About PackGene
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.
