NEW YORK – The European Commission has provided a positive opinion from the European Medicines Agency's Committee for Advanced Therapies for Ocugen's retinitis pigmentosa gene therapy, OCU400, to receive advanced therapy medicinal product classification, the Malvern, Pennsylvania-based company said this week. Ocugen said it expects to submit regulatory applications in Europe and the US after it completes its Phase III trial in 2026 and potentially be able to launch the gene therapy commercially in Europe and the US by 2027.
The US Food and Drug Administration has granted breakthrough device designation for Acrivon Therapeutics' multiplex immunofluorescence assay to identify endometrial cancer patients who may benefit from treatment with the company's investigational CHK1 and CHK2 inhibitor, ACR-368.
The assay, dubbed ACR-368 OncoSignature, was developed using the Acrivon Predictive Precision Proteomics (AP3) platform, a generative AI-based tool, and relies on protein biomarkers to identify best responders. Clinical data presented at the 2024 annual European Society for Medical Oncology meeting found that patients who were OncoSignature-positive were more likely than those who were OncoSignature-negative to respond to ACR-368 treatment, the company said.
Acrivon is currently enrolling patients with endometrial and other cancers into a registrational-intent, multicenter Phase IIb trial of ACR-368 in which it is using the ACR-368 OncoSignature assay to predict which patients are likely to respond to treatment.
Myriad Genetics announced this week that it partnered with Interlink Care Management to make its MyRisk with RiskScore hereditary cancer test available to qualifying patients in Interlink’s CancerCARE network. Patients in that network will have access to Myriad’s guideline-based MyGeneHistory online patient screening tool, and if criteria are met, they will be referred to a clinician who can order MyRisk on their behalf. Those patients will also be able to review their results with one of Myriad’s genetic counselors.
Ariceum Therapeutics this week said the US Food and Drug Administration granted orphan drug designation to its radiopharmaceutical 225-Ac-SSO110 (satoreotide) as a treatment for patients with small cell lung cancer (SCLC). In preclinical studies, satoreotide showed encouraging anti-tumor activity against SSTR2-positive xenografts of SCLC in mice. The firm is planning to begin a Phase I/II trial of the product in the first quarter of 2025. The FDA grants orphan designation to drugs for rare diseases, which provides sponsors tax credits for clinical trials, exemption from user fees, and seven years of market exclusivity for the drug after regulatory approval.
The US Food and Drug Administration has granted orphan drug designation and rare pediatric disease designation to ABO-101, a gene-editing therapy that Arbor Biotechnologies is developing as a treatment for primary hyperoxaluria type 1, the Cambridge, Massachusetts-based biotech said this week. Arbor anticipates launching a Phase I/II trial, dubbed redePHine, to investigate ABO-101 in adult and pediatric patients with the rare genetic kidney disease in the first half of this year.
The FDA grants orphan designation to drugs for rare diseases, which provides sponsors tax credits for clinical trials, exemption from user fees, and seven years of market exclusivity for the drug after regulatory approval. A sponsor with an approved drug with a rare pediatric disease designation may qualify for a voucher that it can redeem to receive priority review for a different product, or transfer or sell the voucher to another sponsor.
The Australian government this week awarded nearly A$20 million (US$12,567,597) in research grants to support research in pediatric brain tumors, including diffuse intrinsic pontine glioma. The government dedicated A$14 million to the establishment of a national childhood brain cancer clinical trial consortium, led by Monash University in Melbourne, and A$6 million to support early-stage clinical research. Monash University received an additional A$2 million to fund the development of new biomarker-targeted therapies for high-grade gliomas. The University of Newcastle received A$2 million to use artificial intelligence to develop an improved treatment regimen for children with diffuse midline glioma. The University of Western Australia received A$1.1 million to develop treatments for infants with rare brain cancers.
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