The European Medicines Agency issued a negative opinion on 28 March to Eli Lilly and Co which applied to market its Alzheimer’s treatment Kisunla (donanemab) in the EU for patients with amyloid beta plaques in the brain and mild cognitive impairment. Lilly said that it will appeal against the decision.
Peter Marks, MD, PhD, and director of the US Food and Drug Administration’s Center for Biologics Evaluation and Research (CBER), announced his resignation on 28 March. His departure, which is scheduled for 5 April, coincides with a decision by the Trump Administration to downsize the Department of Health and Human Services (HHS), the FDA’s governing body, by reducing the department’s workforce by 20,000 people. The FDA is expected to lose 3,500 full-time employees. This follows the swearing in on 13 February of Robert F Kennedy Jr as the HHS secretary.
The European Commission launched a new proposal on 19 March to stimulate private investment in industry and harmonise rules for trading in securities. The plan would essentially try to encourage citizens with large cash savings in banks in the EU to invest a portion of these assets in industry, including in small and medium size companies, to stimulate innovation and boost growth. The plan, called the savings and investment union, is a new version of an earlier capital markets union proposal that was introduced in 2014 but failed to gain traction.
Francis Collins, a geneticist and director of the US National Institutes of Health for 12 years, retired from his position on 28 February, with a tribute to his colleagues and the institution that they support. “The National Institutes of Health is the world’s foremost medical research institution. It has been rightfully called the ‘crown jewel’ of the federal government for decades. It has been the greatest honour of my life to be part of this institution in various roles over the last four decades,” he said in a statement.
The US Food and Drug Administration has approved a new allogeneic encapsulated, cell-based gene therapy for a rare neurodegenerative disease that affects the macula, a portion of the eye that processes central vision. The product, Encelto (revakinagene taroretcel) is a therapy that delivers a recombinant protein to the retina to slow down the progression of the disease. Delivery is by way of a surgical intravitreal implant by an ophthalmologist.
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.
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.
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.