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.
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.
The US Food and Drug Administration has approved a new non-opioid treatment for pain that targets a signalling pathway in the peripheral nervous system before pain signals reach the brain. The drug, Journavx (suzetrigine), is the first drug in a new class of medicines for the treatment of moderate to severe acute pain arising from trauma or surgery. Typically, these cases may be treated with prescription opioids.
“The world is not waiting for us,” Ursula von der Leyen, the European Commission president told journalists on 29 January as she introduced new proposals to improve the competitive position of European industry. The Commission president outlined three goals for the coming year. They include providing incentives for new companies, especially in artificial intelligence; advancing clean energy; and expanding commercial partnerships with companies and countries across the globe. The measures for companies include incentives for generating risk capital.
The Alzheimer’s disease drug, Leqembi (lecanemab), has been approved in the US for a new dosing schedule enabling patients to continue taking it after 18 months of treatment. The new maintenance indication will enable dosing once every four weeks using an intravenous administration. It will take effect after the 18-month initiation phase of bi-weekly dosing finishes. Leqembi has been approved for patients with mild cognitive impairment or mild dementia, also known as early Alzheimer’s disease.