News

An Israeli medical device company has been sold to Boston Scientific Corp of the US concurrent with the start of a pivotal trial intended to confirm the effectiveness of its lead product for hypertension. SoniVie Ltd has developed an intravascular device for renal denervation procedures. These procedures reduce activity in the renal nerves in the kidneys in order to help lower blood pressure. France-based Andera Partners has been a venture investor in the company since 2023. Separately, Boston Scientific has been a minority shareholder.

A new gene therapy has been given a positive opinion by the European Medicines Agency for the treatment of dystrophic epidermolysis bullosa (DEB), a rare skin disease. The therapy, Vyjuvek (beremagene geperpavec), has been cleared for the treatment of the disease in patients of all ages.

A new small molecule combination treatment for cystic fibrosis has been given a positive opinion by the European Medicines Agency – expanding the repertoire of protein modulators for treating this disease. The combination, Kaftrio (ivacaftor/tezacaftor/elexacaftor) and Kalydeco (ivacaftor), was developed by Vertex Pharmaceuticals Inc. It modulates the cystic fibrosis transmembrane conductance regulator (CFTR) protein which is produced by a gene of the same name.

AMYRA Biotech AG, a Basel, Switzerland-based company working in digestive health innovation, has published a peer-reviewed literature review in Alimentary Pharmacology & Therapeutics supporting a novel approach to enzyme therapy for coeliac disease. The study underscores the critical role of the intestinal brush border membrane (BBM) in protein digestion and explores the potential benefits of supplementing exopeptidase activity in affected patients.

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.