This week our Industry Insights: Healthcare looks at the potential for Food and Drug Administration (FDA) approval of CRISPR for sickle cell anaemia, new lung cancer screening guidelines by the American Cancer Society, and FDA approval for Phathom’s Voquezna.
A panel of external advisors to the FDA has found the safety data for Exagamglogene Autotemcel (exa-cel) for the treatment of sickle cell diseaseadministered by Vertex Pharmaceuticals and CRISPR Therapeutics support a favourable benefit-risk in adults and adolescents with sickle cell disease. Exa-cel is a therapy based on CRISPR-Cas9 genome-editing and would be the first treatment of its kind to gain approval by the FDA. This agreement on safety will allow the FDA to consider its approval by 8th December. This not only offers a potential cure for the millions of people with sickle cell disease worldwide, but also raises the prospect of further regulatory approvals of CRISPR treatment for other genetic diseases.
The American Cancer Society has released its updated guidelines for screening for lung cancer. One of the major changes is the recommendation to include adults ages 50 to 80, with at least a 20-pack-year smoking history. This is a dramatic change from previous guidelines that recommended annual screening for adults aged 55 to 74 with at least a 30-pack-year smoking history who either currently smoke or quit less than 15 years ago. The American Cancer Society estimates that this will lead to more lung cancer deaths (21%) being prevented compared to the 2013 guidelines. Screening is a key pillar to successful treatment of lung cancer, and this updated guideline will hopefully lead to earlier detection and a greater ability to offer treatment with curative intent.
Voquezna (Vonoprazan) is a novel potassium-competitive acid blocker that inhibits gastric H+/K+-ATPase. Voquezna was initially cleared to treat Helicobacter pylori in May last year, but was then revoked after trace levels of a cancer-causing agent (nitrosamine N-Nitro-vonoprazan) were identified in commercial batches of treatment. Phathom has subsequently demonstrated that Voquezna can stay below an acceptable threshold of daily intake of nitrosamine N-Nitro-vonoprazan. This has resulted in the approval of Voquezna for the treatment of both Helicobacter pylori infection and erosive gastroesophageal reflux disease (GERD). Phathom states that this is the first major innovation to the acid suppressant market in the United States (US) in over 30 years. This is an important step forward in the treatment of Helicobacter pylori,which is becoming less sensitive to clarithromycin and/or metronidazole and less likely to respond to triple therapy with proton pump inhibitors. Now that these are both approved, Phathom is hoping for a December 2023 US launch.
