CRD-TMH-001

Almost two months after the nonprofit Cure Rare Disease received FDA clearance to administer its first-in-human CRISPR therapeutic for Duchenne muscular dystrophy, the nonprofit announced that the one patient in the trial, Terry Horgan, the brother of the nonprofit’s founder and CEO, Rich Horgan, passed away. Researchers are still trying to figure out what exactly happened with the single shot CRISPR therapy, the nonprofit said in a statement, and that process may take up to four months. The therapy, known as CRD-TMH-001, treats certain mutations on the dystrophin gene, with the goal of stabilizing or potentially reversing the progression of debilitating DMD symptoms. Dosing of the drug took place at the University of Massachusetts Medical School. “The loss of any patient in any trial is immense loss for the family and for the caregiving team,” UMass Chan Medical School Dean Terence Flotte told Endpoints News via email. “This patient was very courageous, and we are all saddened by the outcome. CRD is working with a multidisciplinary, multi-institutional group of scientists to learn the most that can possibly be learned from this case, so as to inform the safe conduct of future gene therapies, whether they be for DMD or for other genetic diseases.” Cure Rare Disease said in a statement that it will share the findings from the trial with the scientific community. The trial was launched after three years of development with partners including the Lek Lab at Yale University and contract research firm Charles River Laboratories. A spokesperson with Cure Rare Disease also told the Associated Press that the nonprofit filed a report on the death with the FDA as required. But the nonprofit isn’t the only one developing a gene therapy in DMD, and isn’t the only one that has run into issues. Pfizer’s Phase Ib trial for its gene therapy for DMD was halted in 2021 after a young man in it died. One of Pfizer’s Duchenne gene therapy trials put on hold in wake of patient death as high-dose AAV concerns still cloud field In April, however, the trial hold was lifted and Pfizer opened its first US sites for a global Phase III trial assessing its gene therapy for DMD, known as fordadistrogene movaparvovec. Sarepta and Roche are also working on their own gene therapy for DMD, and have applied with the FDA for an accelerated approval. The therapy, SRP-9001, initially failed a portion of a Phase II study in early 2021, but a second part showed patients had statistically significant improvements in motor abilities and later showed that motor function improved after one year. Aiming for fourth nod, Sarepta files another DMD gene therapy to FDA; Axsome headed toward migraine resubmission

Sarah Silbiger/Getty Images Every week, the U.S. Food and Drug Administration reviews drug applications, grants various designations to drugs and medical devices and approves therapies. It’s difficult to keep up, but don’t worry, BioSpace has you covered. Daiichi Sankyo and AstraZeneca reported Friday that the FDA has approved Enhertu (fam-trastuzumab deruxtecan-nxki) for the treatment of adults with unresectable or metastatic non-small cell lung cancer whose tumors have activating HER2 mutations. This follows the August 6 approval of Enhertu for adults with unresectable or metastatic HER2-low breast cancer. The FDA nod is for patients who have received previous chemotherapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy. Guardant Health reported Friday that the FDA has approved its Guardant360 CDx liquid biopsy test as a companion diagnostic for select patients with unresectable or metastatic HER2-mutant non-small cell lung cancer whose tumors have activating HER2 mutations for treatment with Daiichi Sankyo and AstraZeneca’s Enhertu. Merck reported Tuesday that the FDA found contamination of some samples of its type 2 diabetes drug Januvia (sitagliptin). The contamination was nitrosamine Nitroso-STG-19 (NTTP). The agency will allow continued distribution, but the company is working to improve quality control mechanisms. Bayer announced Monday that the FDA has approved a supplemental New Drug Application (sNDA) for its oral androgen receptor inhibitor darolutamide in combination with docetaxel for patients with metastatic hormone-sensitive prostate cancer. The approval was based on data from the Phase III ARASENS trial. ImmVira, based in Shenzhen, China, reported that the FDA has granted its new oncolytic herpes simplex virus product MVR-C5252 Orphan Drug Designation. The therapy is targeting malignant glioma. The drug, developed on ImmVira’s OvPENS new drug development platform, is designed to achieve on-target malignant gliocyte killing while maintaining a solid safety profile. Attralus’s 124I-AT-01 (iodine (I-124) evuzamitide) received Orphan Drug Designation from the FDA as a diagnostic for the management of transthyretin amyloidosis (ATTR). The product utilizes Attralus’s pan-amyloid binding peptide as an amyloid-specific imaging agent. Relmada Therapeutics received Fast Track designation from the FDA for REL-1017, a novel NMDA receptor channel blocker being developed as a monotherapy for major depressive disorder. The drug is being assessed in a registrational Phase III trial in this indication. It is also being studied in two pivotal Phase III studies as a potential adjunctive treatment for MDD. MaaT Pharma announced Wednesday that the FDA has maintained the clinical hold on MaaT013. The drug is being developed for steroid-resistant acute graft-versus-host disease. The hold will continue while the agency awaits more data on how the drug was developed. Cure Rare Disease (CRD) received the greenlight to conduct a Phase I trial of CRD-TMH-001, a CRISPR-based therapeutic to treat muscle promoter and exon 1 mutations in the dystrophin gene for Duchenne muscular dystrophy (DMD). Bodyport received 501(k) clearance from the FDA for its noninvasive biomarker platform, the Bodyport Cardiac Scale, which can assess measures of heart function and fluid status. CereVasc announced the FDA has approved an investigational device exemption (IDE) application to initiate a pilot trial of the eShunt System in patients who develop communicating hydrocephalus post aneurysmal subarachnoid hemorrhage. Lexaria Bioscience Corp. received a positive full written response from the FDA from its pre-IND meeting regarding DehydraTECH-CBD for the treatment of hypertension. aTyr Pharma received Fast Track designation from the FDA for efzofitimod for pulmonary sarcoidosis, a type of interstitial lung disease. The drug is a first-in-class immunomodulator against neuropilin-2. The Menarini Group and Stemline Therapeutics reported that the FDA has accepted their NDA for elacestrant, a selective estrogen receptor degrader for patients with ER+/HER2- advanced or metastatic breast cancer. It has been given a PDFUA date of February 17, 2023. Aro Biotherapeutics received Orphan Drug Designation from the FDA for ABX1100, an investigational Centyrin-siRNA conjugate targeting the Gys1 gene in the muscle, for the treatment of Pompe disease.