Moderna’s ambitions in messenger RNA technology include therapies that do their editing work inside the patient. To that end, the biotech company is starting an R&D collaboration with a division of ElevateBio that brings…
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What You Should Know:– BrightInsight announced that ixlayer has joined the BrightInsight Ecosystem, a network of more than 20 of the world’s leading healthcare and technology companies driving growth and adoption of digital health solutions.– The BrightInsight Ecosystem of…
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The medical industry is far from achieving racial health equity, according to a new report from Accenture. But it could be. The report, which outlines some of the stark racial health disparities still plaguing…
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Travere Therapeutics has won FDA approval for a drug that treats a rare autoimmune disorder that is typically first noticed as blood in the urine and can progress over time to end-stage kidney failure.
The…
Clotting disorders can be treated by infusing patients with clotting proteins they lack, or with drugs that replace the function of these proteins. Gene therapies may soon become another option. Hemophilia A and B…
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Geographic atrophy, an eye disorder that starts as a loss of central vision and progressively worsens to total blindness, now has its first treatment. The FDA on Friday approved an Apellis Pharmaceuticals drug that slows the progression of this disease, which affects more than 1 million people in the U.S.
Approval of the Apellis drug, pegcetacoplan, covers all patients with geographic atrophy, a broad label that reflects the representative patient population tested in pivotal studies, Chief Medical Officer Caroline Baumal said during a Friday evening conference call. The Waltham, Massachusetts-based company plans to launch the drug in March, marketing the product under…
A Chiesi Farmaceutici drug for a rare enzyme deficiency is now approved by the FDA, making it the first U.S. treatment for a disorder that leads to a range of cognitive and muscle problems.
The regulatory decision announced late Thursday covers the treatment of children and adults who have alpha-mannosidosis (AM), a disease caused by a genetic mutation that leads to defective or inactive forms of an enzyme called alpha-D-mannosidase. Chiesi’s drug, velmanase alfa, is an engineered version of that enzyme. The privately held Italian company will market its new FDA-approved drug as Lamzede, the same name for the product in Europe,…
The current Adderall shortage is of serious concern to both patients who have attention deficit hyperactivity disorder (ADHD) and clinicians. This shortage cannot be quickly alleviated by a simple increase in the amount of medication manufactured, unless the upper limit to the amount of Adderall’s active ingredient that can be manufactured in a given year is raised by the Drug Enforcement Agency (DEA). The limit has been imposed because the active ingredient can be abused as a recreational drug. It is necessary to control the supply of any medication with abuse liability, but this must be achieved without compromising the legitimate…
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A Moderna messenger RNA vaccine for influenza now has preliminary data from a pivotal study showing the shot prompted a strong immune response to the more common strains of the virus but fell short against less common strains.
The results reported Thursday are from one of two pivotal studies for the Moderna vaccine, mRNA-1010, which is assessing the shot for safety and immune response. A separate Phase 3 study is evaluating the shot for efficacy. Results from that study could come as early as next month. Moderna’s vaccine encodes hemagglutinin, a protein on the surface of the influenza virus that is also…
Talaris Therapeutics aimed to transform solid organ transplants with a cell therapy that replaces anti-rejection drugs patients need for the rest of their lives. But after clinical trial setbacks, the biotech has decided to end two kidney transplant studies. Although a separate test of its experimental therapy is continuing in a rare autoimmune condition, Talaris has commenced a restructuring plan that it said could culminate in a merger or sale of the company.
The restructuring announced Thursday will lead to the layoff of about one-third of Talaris’s staff. As of the end of the third quarter of 2022, the Louisville, Kentucky-based biotech…
