A new small molecule drug has won approval from the US Food and Drug Administration to treat patients with metastatic pancreatic ductal adenocarcinoma – an aggressive cancer with a poor prognosis for survival. The drug, Rasonque (daraxonrasib), has been authorised for adults who have received at least one prior systemic therapy for their cancer, or who are not candidates for multiagent systemic therapy.
US-based ReCode Therapeutics Inc has strengthened its ties with the Cystic Fibrosis Foundation as it progresses an experimental messenger RNA (mRNA) therapy for the disease through the clinic. Simultaneously, it promoted Heather Clark on 1 July to the role of chief executive from vice president and head of the company’s cystic fibrosis franchise. Ms Clark joined ReCode in 2022. She has nearly 30 years of experience in rare disease drug development of which more than half were spent at Vertex Pharmaceuticals Inc, developer of five marketed cystic fibrosis products.
The US Food and Drug Administration granted an accelerated approval on 19 August for a new gene therapy for patients with an inherited disorder caused by the shortage of an enzyme needed to maintain stable blood sugar levels in the body. The therapy, Genglycos (pariglasgene brecaparvovec), is the first treatment to be authorised for the disorder – glycogen storage disease type la. Also called von Gierke disease, the disorder results in the build-up of glycogen, a complex sugar, in the body’s cells.
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