Trial monitors recommend Elevidys studies continue; Novartis kidney drug approved
Today, a brief rundown of news involving Sarepta Therapeutics, Novartis and Allakos, as well as updates from CSL Behring and Roche that you may have missed.
A group of independent trial monitors on Friday recommended dosing continue in a trio of studies testing Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy, Elevidys, after a brief halt. Dosing in the trials was paused earlier this week at the request of the European Medicines Agency, which wanted researchers to conduct an analysis into the recent death of a Duchenne patient treated with Elevidys. On Thursday, they met and concluded Elevidys’ “risk-benefit profile remains favorable” and dosing should resume without any changes to the study protocols, Sarepta said in a statement. Sarepta and Roche, which owns European rights to Elevidys, will share the findings with regulators within a week, after which the EMA will evaluate the findings. The three trials affected include one called Envision that’s meant to confirm Elevidys’ benefits in Duchenne patients who can no longer walk. — Ben Fidler
Novartis on Thursday won accelerated approval of a kidney disease drug it acquired in a $3.2 billion buyout two years ago. Called Vanrafia, the drug was cleared by the Food and Drug Administration for use treating IgA nephropathy, a chronic condition that can lead to kidney failure as well as an increasingly competitive area of pharmaceutical research. Novartis aims to grab market share from Travere Therapeutics’ Filspari. One advantage it may have commercially, according to some Wall Street analysts, is that patients on Vanrafia won’t need to be periodically checked for liver problems. At about $163,000 per year, Vanrafia is priced at a slight premium to Filspari, which has a list price of just over $151,000. — Ben Fidler
Allakos has agreed to be acquired by Concentra Biosciences, a firm controlled by Tang Capital Partners, for $0.33 per share in cash, the company said Wednesday. The deal comes after two strategic resets and a pair of major restructurings for Allakos, a developer of immune disease drugs, in the last two years. It’s also the latest bid for a distressed biotech by Concentra, which since 2023 has acquired Theseus Pharmaceuticals and Jounce Therapeutics and amassed stakes in other struggling drugmakers with the intent of buying and liquidating them. — Ben Fidler
People in Germany living with hemophilia B now have access to a gene therapy for their condition after maker CSL Behring and a national association of health insurance funds in that country reached an agreement on a reimbursement price. The therapy, which CSL sells as Hemgenix, was conditionally authorized in the European Union in 2023. CSL has already secured access decisions in Denmark, Switzerland, Spain, Austria and the U.K, but said the agreement in Germany is the first time Hemgenix is being made available under a national, success-based reimbursement model. Last summer, two people in France became the first to be commercially treated with Hemgenix in Europe. — Ned Pagliarulo
A late-stage study testing a high dose of Roche’s multiple sclerosis drug Ocrevus failed to show a benefit over the the currently approved dose, the company disclosed Tuesday. Roche ran the trial to test whether a dose two or three times higher, depending on a patient’s weight, could further slow disability progression in people with the relapsing form of the disease. Instead, the data confirmed that Ocrevus is “optimally dosed,” said Roche Chief Medical Officer Levi Garraway. Roche plans to present full data from the study at an upcoming meeting. The company recently launched a subcutaneous form of the blockbuster drug. — Ned Pagliarulo
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