NEW YORK – CSL Behring Canada this week said the Drug Expert Committee of the Canadian Agency for Drugs and Technologies in Health (CADTH) has recommended reimbursement for Hemgenix (etranacogene dezaparvovec) for eligible patients with hemophilia B who require routine prophylaxis to reduce bleeding episodes. The committee's decision is based on data from the Phase III HOPE-B trial, in which the one-time gene therapy increased factor IX activity levels and significantly reduced the rate of bleeds annually for patients compared to routine prophylaxis. Hemgenix, often referred to as the most expensive drug in the world, was approved in 2022 in the US where it is priced at $3.5 million. The treatment garnered regulatory approval from Health Canada last year. CADTH estimates that the reimbursement of the hemophilia B gene therapy will be associated with a budgetary increase of C$31.5 million (US$23.1 million) the first year, C$53.5 million the second year, and C$54.8 million the third year. The agency estimates the three-year total incremental cost of providing the drug at C$139.8 million.
Donaldson Company subsidiary Univercells Technologies Business said this week that the University of Pennsylvania's Gene Therapy Program has expanded an agreement to use Univercells Technologies' bioreactors in its gene therapy product manufacturing. Under the terms of the expanded deal, UPenn's GTP will evaluate Univercells' Scale-X bioreactor with the goal of lowering the cost of viral vectors for gene therapies while increasing the production per batch.
COTA and Texas Oncology subsidiary Precision Health Informatics said this week that they have inked a deal to integrate machine learning into precision oncology across Texas Oncology's community cancer centers using COTA's curation engine, CAILIN. Through the collaboration, Precision Health Informatics will use patients' genetic information coupled with COTA's artificial intelligence platform to personalize patients' treatment.
Sanford Health said this week that it has launched a genetic screening research study called ImagineYou, conducted in partnership with Sanford Imagenetics and Helix. The population health study will provide participants with genetic screening for familial hypercholesterolemia, hereditary breast and ovarian cancer syndrome, and Lynch syndrome at no cost. Those testing positive will be referred to genetic counseling for follow-up, also at no cost. In addition, participants can learn about their ancestry and genetic traits through their Helix account.
The US Food and Drug Administration has granted regenerative medicine advanced therapy designation to Kyverna Therapeutics' KYV-101, an autologous anti-CD19 CAR T-cell therapy, in the refractory stiff-person syndrome setting, a rare autoimmune condition, the Emeryville, California-based firm said this week. For the designation, the FDA reviewed clinical outcomes of patients treated with the cell therapy in Germany under a named-patient treatment option.
Separately, Kyverna is clinically testing KYV-101 in various other rheumatological and neurological autoimmune conditions, including multiple sclerosis, myasthenia gravis, systemic sclerosis, and lupus nephritis.
Philadelphia-based Passage Bio this week said it received positive feedback from the US Food and Drug Administration in a "type C" meeting on its proposal to expand the company's Phase I/II upliFT-D clinical trial testing the investigational gene therapy, PBFT02, for frontotemporal dementia to include patients with certain mutations in the C9orf72 gene. Currently, the clinical trial is only open to patients with pathogenic mutations in the GRN gene. Passage plans to submit a revised clinical trial protocol to health authorities and expects to begin dosing in this new patient population in the second half of 2025.
Genome analysis company Genomics Plc said this week that it has extended the collaboration with Vertex Pharmaceuticals for three more years for the discovery and development of new precision medicines. As part of the extension, Genomics said it will now also explore using the company’s proprietary genetic tools to improve patient population identification and to de-risk the selection of biomarkers for measuring early readout of drug efficacy.
First announced in 2018, Genomics’ partnership with Vertex was previously extended to 2021. The collaboration will now run until 2026.
Exact Sciences said this week that it has awarded a total of $1.5 million to 28 organizations through its Funding Opportunities for CRC Screening Uptake Strategies (FOCUS) Program, which provides grants to community organizations, health foundations, public health organizations, and advocacy groups working to increase access to colorectal cancer screening and awareness. Since its launch in 2022, FOCUS has awarded more than $4.5 million to over 75 organizations throughout the United States.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared on Precision Medicine Online.