NEW YORK – The US Food and Drug Administration has granted orphan drug designation to UniQure's AMT-191, the company said this week. Amsterdam-based UniQure is developing AMT-191 as a gene therapy treatment for Fabry disease, a rare and inherited lysosomal storage disorder that's caused by mutations in the GLA gene. The company is currently testing the gene therapy in an open-label Phase I/IIa trial.
The World Health Organization this week endorsed dual-stain cytology testing in its cervical cancer prevention guidelines. The only US Food and Drug Administration-approved and CE-marked dual-stain cytology test to help identify human papillomavirus-positive patients at risk of developing cervical cancer is Roche's CINtec Plus Cytology assay, Roche said in a statement. The test simultaneously detects two biomarkers, p16 and Ki-67, that indicate a cell may turn cancerous and can be performed using the same liquid sample used for HPV or Pap cytology testing, Roche noted.
Vertex Pharmaceuticals this week said that Health Canada has granted it marketing authorization for the sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) therapy Casgevy (exagamglogene autotemcel), marking the first CRISPR gene-editing treatment to be approved in the country. Casgevy already has been approved in the US and received conditional marketing authorization in the EU. Although the UK Medicines and Healthcare Products Regulatory Agency granted the therapy conditional marketing authorization in 2023, earlier this year the UK National Institute for Health and Care Excellence recommended the treatment for TDT, but not for SCD.
Basel, Switzerland-headquartered contract development and manufacturing organization Lonza this week said it signed a long-term commercial supply agreement with Vertex Pharmaceuticals for commercial production of Casgevy (exagamglogene autotemcel), a first-of-its-kind CRISPR gene-editing treatment for sickle cell disease and transfusion-dependent beta-thalassemia. Casgevy will be manufactured at the company's Geleen, Netherlands-based cell therapy manufacturing facility, and Lonza plans to expand production to another site in Portsmouth, New Hampshire.
The US Food and Drug Administration has granted rare pediatric disease designation to EXG-34217, an investigational treatment that Elixirgen Therapeutics is developing for dyskeratosis congenita and related telomere biology disorders, the Baltimore-based company said this week. EXG-34217 is comprised of autologous CD34-positive hematopoietic stem cells that have been modified ex vivo with EXG-001, an RNA-based Sendai virus vector encoding ZSCAN4 to extend telomeres in patients. Elixirgen is testing EXG-34217 within a Phase I/II trial at Cincinnati Children's Hospital Medical Center.
Eli Lilly this week said that the Ministry of Health, Labour, and Welfare in Japan has approved its beta-amyloid-targeting Alzheimer's disease drug Kisunla (donanemab-azbt) as a treatment designed to slow disease progression for patients with early Alzheimer's and confirmed beta-amyloid pathology. Japan is the second major market in which Indianapolis-based Lilly has gained approval for Kisunla, following initial approval in the US in July.
Cancer detection company ClearNote Health announced this week that its Avantect Ovarian Cancer Test has received a Proprietary Laboratory Analyses (PLA) code. The code, 0507U, is effective Oct. 1, 2024. The Centers for Medicare and Medicaid Services also proposed a preliminary reimbursement rate for the code of $1,160.00, a rate that was established last year for ClearNote's Avantect Pancreatic Cancer Test. CMS will issue a final determination on the reimbursement rate for 0507U later this year that will go into effect on Jan. 1. The ovarian cancer test is intended for patients at high risk for ovarian cancer and uses epigenomic and genomic methods to measure levels of the 5-hydroxymethylcytosine biomarker, copy number variants, and fragment sizes in cell-free DNA.
Penn State College of Medicine this week announced that Sinisa Dovat, a pediatric oncology researcher in the Department of Pediatrics, received a $4 million grant from the Pennsylvania Department of Health. Dovat will put the funding toward researching high-risk pediatric leukemia subtypes, which are prevalent in Hispanic and Latino populations, and developing personalized therapies that target their unique genetic pathways.
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.