ITM Isotope Technologies Munich SE's August 28, 2026 PDUFA target action date for 177Lu-edotreotide (ITM-11, a synthetic somatostatin-analog peptide-radioconjugate for gastroenteropancreatic neuroendocrine tumors) passed without an FDA approval decision, following the August 7, 2026 Complete Response Letter that cited Chemistry, Manufacturing, and Controls items and unresolved conditions at a third-party commercial manufacturing facility as the sole basis for the refusal. The FDA explicitly did not raise clinical or nonclinical safety or efficacy concerns, and did not request additional clinical data. ITM has stated it plans to resubmit the NDA once the manufacturing and facility items are addressed. The Phase 3 COMPETE trial (309 patients, first- or second-line inoperable progressive Grade 1 or Grade 2 GEP-NETs) had met its primary endpoint with a significant progression-free survival benefit over everolimus and a significantly higher objective response rate. The CRL is the second on the ITM-11 program and delays direct commercial competition with Novartis Lutathera (177Lu-DOTATATE, the incumbent peptide-radioconjugate for GEP-NETs).
Capricor Therapeutics (NASDAQ: CAPR) faced its Prescription Drug User Fee Act (PDUFA) target action date Saturday August 22, 2026 for deramiocel (a cardiosphere-derived allogeneic cell therapy, not a peptide) in Duchenne muscular dystrophy (DMD) cardiomyopathy. Background: the FDA issued a Complete Response Letter (CRL) in July 2025 citing that the Phase 2 data supporting the cell therapy fell short of the statutory requirement for substantial evidence of effectiveness. Capricor reported positive Phase 3 HOPE-3 trial results in December 2025 meeting both primary and secondary endpoints, and the FDA accepted the resubmission in March 2026 with a Class 2 resubmission classification and August 22 target action date. The July 29, 2026 FDA Cellular, Tissue, and Gene Therapies Advisory Committee voted 9-3 that available evidence does not provide substantial evidence of effectiveness to recommend approval. Panel members cited concerns about the stability of the statistical results, with left ventricular ejection fraction (LVEF) endpoint outcomes appearing highly sensitive to how missing data were handled and which analytic assumptions were applied. A second Complete Response Letter is the expected outcome, and CAPR stock slipped in advance of the decision on elevated volume as investors positioned for the anticipated regulatory rejection. Duchenne muscular dystrophy affects approximately 20,000 US patients (predominantly boys with X-linked inheritance) and progressive cardiomyopathy is the leading cause of death in the DMD population. The DMD cardiomyopathy indication remains without an FDA-approved therapy. Capricor holds Rare Pediatric Disease Designation for deramiocel, which may qualify the company for a Priority Review Voucher upon eventual approval if it comes.
Capricor Therapeutics (NASDAQ: CAPR) faced its PDUFA target action date Saturday August 22, 2026 for deramiocel (a cardiosphere-derived allogeneic cell therapy, not a peptide) in the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy. Background: the FDA issued a Complete Response Letter (CRL) in July 2025 citing that the Phase 2 data supporting the cell therapy fell short of the statutory requirement for substantial evidence of effectiveness. Capricor reported positive Phase 3 HOPE-3 trial results in December 2025 meeting both primary and secondary endpoints, and the FDA accepted the resubmission in March 2026 with a Class 2 resubmission classification and August 22 target action date. The July 29, 2026 FDA Cellular, Tissue, and Gene Therapies Advisory Committee voted 9-3 that available evidence does not provide substantial evidence of effectiveness to recommend approval. Panel members cited concerns about the stability of the statistical results, with left ventricular ejection fraction (LVEF) endpoint outcomes appearing highly sensitive to how missing data were handled and which analytic assumptions were applied. A second Complete Response Letter is the expected outcome. Duchenne muscular dystrophy affects approximately 20,000 US patients (predominantly boys with X-linked inheritance) and progressive cardiomyopathy is the leading cause of death in the DMD population. The DMD cardiomyopathy indication remains without an FDA-approved therapy, which continues the substantial unmet need in the space. Capricor holds Rare Pediatric Disease Designation for deramiocel, which may qualify the company for a Priority Review Voucher upon eventual approval if it comes.
Capricor Therapeutics (NASDAQ: CAPR) faces its Prescription Drug User Fee Act (PDUFA) target action date Saturday August 22, 2026 for deramiocel (a cardiosphere-derived cell therapy, not a peptide) in the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy. Background: the FDA issued a Complete Response Letter (CRL) in July 2025 citing that the Phase 2 data supporting the cell therapy fell short of the statutory requirement for substantial evidence of effectiveness. Capricor reported positive Phase 3 HOPE-3 trial results in December 2025 meeting both primary and secondary endpoints, and the FDA accepted the resubmission in March 2026 with a Class 2 resubmission classification and August 22 target action date. The July 29, 2026 FDA Cellular, Tissue, and Gene Therapies Advisory Committee voted 9-3 that available evidence does not provide substantial evidence of effectiveness to recommend approval. Panel members cited concerns about the stability of the statistical results, saying that left ventricular ejection fraction (LVEF) endpoint outcomes appeared highly sensitive to how missing data were handled and which analytic assumptions were applied. A second Complete Response Letter is the expected outcome. Duchenne muscular dystrophy affects approximately 20,000 US patients (predominantly boys with X-linked inheritance) and progressive cardiomyopathy is the leading cause of death in the population. The DMD cardiomyopathy indication remains without an FDA-approved therapy.
Camurus said on June 10 that the FDA had issued a Complete Response Letter on the Oclaiz (CAM2029) NDA, the second the program has received and the second based solely on unresolved observations from a September 2024 cGMP inspection at a third-party contract manufacturer. The CRL did not cite clinical efficacy or safety. Camurus expects to resubmit in Q4 2026 with a second validated manufacturer; an approval pushed to the first half of 2027 leaves the US launch about a year behind the EU and UK rollouts (under the Oczyesa brand) that landed in 2025.