Pharmaceutical Industry Roundup: Gene Therapies, Drug Approvals, and Clinical Trial Updates

Sarepta's Elevidys Trials to Resume After Brief Halt

Independent trial monitors have recommended the continuation of dosing in three studies testing Sarepta Therapeutics' Duchenne muscular dystrophy gene therapy, Elevidys. This decision comes after a brief halt requested by the European Medicines Agency (EMA) following the death of a Duchenne patient treated with the therapy. The monitors concluded that Elevidys' "risk-benefit profile remains favorable," and dosing should resume without changes to study protocols.

Sarepta and Roche, which owns European rights to Elevidys, plan to share these findings with regulators within a week. The EMA will then evaluate the information. One of the affected trials, Envision, aims to confirm Elevidys' benefits in Duchenne patients who can no longer walk.

Novartis Secures Approval for Kidney Disease Drug Vanrafia

Novartis has received accelerated approval from the U.S. Food and Drug Administration (FDA) for Vanrafia, a drug targeting IgA nephropathy. This chronic kidney condition has become an increasingly competitive area of pharmaceutical research. Novartis acquired the drug through a $3.2 billion buyout two years ago and is positioning it to compete with Travere Therapeutics' Filspari.

Vanrafia's potential commercial advantage lies in the absence of required periodic liver function tests for patients. Priced at approximately $163,000 per year, Vanrafia comes at a slight premium compared to Filspari's list price of just over $151,000.

Industry Consolidation: Allakos Agrees to Acquisition by Concentra Biosciences

Allakos, a developer of immune disease drugs, has agreed to be acquired by Concentra Biosciences for $0.33 per share in cash. This deal follows two strategic resets and major restructurings for Allakos over the past two years. Concentra Biosciences, controlled by Tang Capital Partners, has been actively pursuing distressed biotech companies, having previously acquired Theseus Pharmaceuticals and Jounce Therapeutics.

Advancements in Hemophilia and Multiple Sclerosis Treatments

CSL Behring has reached an agreement with German health insurance funds to provide access to Hemgenix, a gene therapy for hemophilia B. This marks the first time Hemgenix will be available under a national, success-based reimbursement model in Germany. The therapy has already secured access in several European countries, including Denmark, Switzerland, Spain, Austria, and the UK.

In other news, Roche's late-stage study testing a high dose of its multiple sclerosis drug Ocrevus failed to show benefits over the currently approved dose. The company's Chief Medical Officer, Levi Garraway, stated that the data confirmed Ocrevus is "optimally dosed" at its current level. Roche plans to present full data from the study at an upcoming meeting and has recently launched a subcutaneous form of the blockbuster drug.