News

Zolgensma (onasemnogene abeparvovec), the gene therapy developed by Novartis for spinal muscular atrophy (SMA), has shown efficacy in a Phase 3 study of patients aged two to less than 18 years who are able to sit but have never walked independently. SMA is a genetic neuromuscular disease caused by the lack of a functional SMN1 gene that affects muscle functions and basic movement. Zolgensma was first approved in the US in 2019, and one year later in the EU, as a paediatric drug for children less than two years old and administered as a single, one-time infusion. 

A kinase inhibitor for the treatment of lung diseases has been recommended for use in children and adolescents from the age of 6 years by the European Medicines Agency. The treatment, Ofev (nintedanib), was authorised for adults in 2014 and subject to approval by the European Commission, will be available for children in 2025. The indication is progressive fibrosing interstitial lung disease which includes a group of rare respiratory disorders characterised by the build-up of scarring in the lungs which can lead to breathing difficulties.

A small molecule drug, Welireg (belzutifan), has been given conditional authorisation by the European Medicines Agency for tumours associated with von Hippel-Lindau disease and previously treated advanced kidney cancer. The opinion, announced on 13 December, expands the availability of the drug to Europe from the US where it was approved in 2021 by the Food and Drug Administration. The developer is Merck & Co Inc which submitted data to the EMA from two clinical studies showing efficacy.

A new cell therapy has been approved by the US Food and Drug Administration to treat paediatric patients from two months of age who experience complications from a stem cell transplant for blood cancer or an immune system disorder. The treatment, Ryoncil (remestemcell-L rknd), is an allogeneic therapy made up from mesenchymal stromal cells and isolated from the bone marrow of healthy adult donors. It is indicated for the treatment of acute graft-versus-host disease in patients undergoing transplants who do not respond to corticosteroids.

A treatment for classic congenital adrenal hyperplasia (CAH) has been approved by the US Food and Drug Administration – the first new drug in decades for a genetic disorder involving the adrenal glands. The drug, Crenessity (crinecerfont), is a small molecule to be used as an adjunct to glucocorticoids. In two randomised, placebo-controlled trials in adults and children Crenessity was able to reduce an overproduction of androgen in adults and children relative to the production of cortisol, an essential hormone. This was achieved without the need for high levels of glucocorticoids.

A second candidate drug for Parkinson’s disease has failed to meet the primary endpoint in a clinical study of patients with early-stage disease. The Roche Group announced the results of its Phase 2b PADOVA study on 19 December, only days after UCB SA said its medication for early Parkinson’s disease had failed to show efficacy. Roche said it will continue to evaluate data from the main trial and an ongoing extension study in deciding what to do. The candidate drug, prasinezumab, is a monoclonal antibody designed to bind to alpha-synuclein protein and reduce neuronal toxicity.

Merck Inc has entered into a licensing deal with the Chinese pharmaceutical group Hansoh Pharma to gain access to a preclinical obesity asset, bringing it into a field currently dominated by Eli Lilly and Co and Novo Nordisk A/S. The asset, HS-10535, is an oral small molecule glucagon-like peptide-1 receptor agonist.

A candidate drug for early-stage Parkinson’s disease has failed to show efficacy at Phase 2a despite having demonstrated promise in earlier studies, the co-developer UCB SA announced on 16 December. The drug, minzasolmin, is a small molecule compound designed to inhibit alpha-synuclein misfolding in the brain. Alpha-synuclein is a normal neuronal protein that regulates synaptic vesicle trafficking. However in Parkinson’s disease it misfolds and aggregates into toxic fibrils.

New York Life Investments, a US money manager and its European affiliate Candriam, have entered a partnership under which New York Life, through its affiliate network, will take a 40% minority stake in Andera Partners, the Paris-based investment group. The goal is to jointly expand their pool of clients. Financial terms of the transaction were not disclosed.  Andera manages €4.3 billion in assets for institutional and private investors. New York Life  has a much deeper portfolio with assets under management of $750 billion.

The Swiss neuroscience company Noema Pharma AG has received fresh financing from the private equity group EQT Life Sciences in order to advance its lead drug candidate for two rare diseases and its pipeline of other central nervous system disorders. The Series B extension brings total capital raised in the round up to CHF 130 million ($145.7 million). This figure includes earlier contributions from Forbion, Jeito Capital, and Sofinnova Partners, among the venture capital groups.