A new gene therapy for cystic fibrosis has entered clinical development in the UK with the aim of improving outcomes for people with cystic fibrosis regardless of their gene mutation. The trial specifically focuses on adults with cystic fibrosis who cannot benefit from the current modulators of the membrane protein – cystic fibrosis transmembrane conductance regulator (CFTR).
According to Reuters news agency, some 300 former members of staff at the US Food and Drug Administration have been asked to return to work following a mass dismissal of more than 1,000 ordered by the Trump Administration as part of its government reorganisation plan. The White House confirmed to Reuters that more than 1,000 had been asked to leave their jobs. The subsequent attempt to bring back 300 of these individuals could not be directly confirmed with the administration.
The US Food and Drug Administration has approved a small molecule drug to treat a rare genetic disorder that can cause progressive damage to the brain and other areas of the body. The drug, Ctexli (chenodiol), is a bile acid replacement therapy developed to treat cerebrotendinous xanthomatosis (CTX), a lipid storage disorder. Patients with CTX are deficient in an enzyme that helps the body break down fats.The disorder is caused by a reduced production of bile acid in the liver, resulting in the deposit of atypical cholesterol metabolites in the brain, liver and other parts of the body.
bluebird bio Inc is to be acquired by two private equity groups in a move to provide more capital to the gene therapy developer which was founded by two academics in 1992 and now has three approved products on the market. The transaction, which was announced on 21 February, will transform bluebird into a private enterprise from its current status as a publicly listed concern on the Nasdaq market. The buyers are Carlyle Group Inc and SK Capital Partners LP.
The European Commission has proposed to shorten the settlement period for securities such as shares and bonds from two days to one in a move to increase liquidity on its capital markets. If approved, the measure would take effect on 11 October 2027. Member states of the union currently manage securities on national markets in their own jurisdictions. But there is growing pressure from the private sector to consolidate securities trading on a single European market.
A new EU regulation for the conduct of clinical trials in the EU took effect on 31 January after a long period of discussion. A key feature of the new legislation is the introduction of a single authorisation procedure for all trials instead of the earlier multiple procedures. Regulation 536/2014 of the European Parliament and of the Council replaces Directive 2001/20/EC. As a Regulation it will apply directly to all member states of the union.
Novartis is to pay up to $3.1 billion to take full control of Anthos Therapeutics Inc, a Boston, US based company that it helped launch with Blackstone Life Sciences in 2019 to develop an asset for a cardiovascular disease. The company has a product in Phase 3 that was in-licensed from Novartis and is now being developed to prevent stroke and systemic embolism in patients with atrial fibrillation. Novartis had a minority stake in Anthos but will now take full control.
Novartis reported a double digit increase in operating profit in 2024 and through higher sales and lower impairments, achieved a profit margin of 28.9% for the year. The goal is to continue this margin expansion during the next 12 months, in addition to raising sales by a mid-to high-single digit percentage, Vas Narasimhan, the company’s chief executive, said in a statement issued on 31 January. Novartis’ 2024 results were the first full-year figures for the company since the spin-out of Sandoz, its generics and biosimilar business in October 2023.
With a focus on a 2030 revenue target, AstraZeneca Plc presented 2024 financial results on 6 February which exceeded expectations but also threw up some risks. Group revenue for 2024 was $54.1 billion, up by 18% at actual exchange rates and by 21% at constant rates, buoyed by double-digit increases across the portfolio. This included sales of drugs for cancer, cardiovascular, renal, metabolic, and respiratory diseases as well as for rare conditions.
GSK Plc has upgraded its forecast for long-term growth following a year in which specialty medicines for oncology and HIV delivered double-digit increases in sales. At the end of 2024 the company had 19 new products in Phase 3 or registration. Emma Walmsley, the chief executive, told journalists on 5 February that the company now expects to achieve more than £40 billion in sales by 2031, up from the company’s previous forecast of £38 billion. At the same time, GSK announced plans to buy back £2 billion of its shares over the next 18 months.