News

The US Food and Drug Administration has approved a new gene therapy for a rare neurological disorder that manifests at an early age – Sanfilippo syndrome type A. Until the approval, treatment was limited to managing symptoms. The therapy, Fayuvi (rebisufligene etisparvovec), is an adeno-associated serotype 9 virus therapy (AAV9) that delivers a working copy of a gene enabling the body to produce sulfamidase, an enzyme which is missing in patients with the disease. Children with deficient supplies of the enzyme cannot beak down a sugar chain known as heparan sulfate.

A $123 million financing has been raised for US-based Sling Therapeutics Inc, equipping it to carry out a Phase 3 trial of a candidate therapy for thyroid eye disease – an autoimmune disorder that can affect vision. Announced on 16 September, the Series C round was led by Forbion Capital Partners of the Netherlands with participation from new investor Sectoral Asset Management of Canada and existing investor TPG Life Sciences Innovations of the US.

GSK Plc is to pay up to $750 million to acquire a new asset for multiple myeloma – its latest move to expand its portfolio for blood cancers. The asset is a trispecific T cell engager, an engineered antibody linking the body’s immune cells directly to antigens on cancer cells. There are currently no approved trispecific T cell engagers, but authorisations have been granted to bispecific T cell engagers, a simpler version of the same therapy. Announced on 15 September, the deal includes an undisclosed upfront fee and milestone payments.

The following article is a summary of the 2026 financial forecasts for several publicly listed European biopharmaceutical companies.

France-based ophthalmology company Nicox SA announced an extension of its cash runway on 2 September ahead of regulatory reviews in both the US and China for its lead product NCX 470 (bimatoprost grenod) for two ocular conditions. As of 31 August, the company’s estimated cash and cash equivalents was €8.4 million compared with €4.1 million on 31 December 2025. The company said the cash position will carry it through the regulatory decision period which, if successful, would generate new revenue.

Patients with polycythaemia vera, a rare blood disorder that causes the body to make too many red blood cells, will be eligible for treatment with a new peptide therapeutic approved by the US Food and Drug Administration on 28 August. Mimrylo (rusfertide) is a hepcidin mimetic that works by mimicking the action of hepcidin, a hormone that regulates the body’s use of iron. Too little iron can lead to iron-deficiency anaemia while too much can be toxic.

The US Food and Drug Administration approved a new treatment for dermatomyositis on 27 August – the first oral drug for a disease that can cause progressive damage to the muscles, skin and lungs and an impaired quality of life. Existing treatments include combinations of chronic steroids and antirheumatic drugs which have not been effective in controlling the disorder. The new treatment, Lisraya (brepocitinib), works as a Janus kinase inhibitor, blocking pathways that play a key role in the body’s immune responses.

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.