The AMS Blog

On 24 March, Novartis announced new anticipated long-term positive data for its one-time infusion gene therapy, Zolgensma (onasemnogene abeparvovec-xioi), that showed significant therapeutic benefit in patients with spinal muscular atrophy (SMA) over a range of extended studies, covering patients treated presymptomatically, and sustained durability in patients up to five years post-dosing. Also, interim results of a novel intrathecal formulation testing patients with SMA type 2, ages two to five years, were reported. The robust overall trial results are expected to…

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Novo Nordisk has halted one midstage and two late-stage tests of its bleeding disease drug concizumab after seeing blood clots in some patients.  The Danish pharma, more known for its diabetes franchise, has been branching out more deeply into hemophilia in the past few years, following a growing market for the bleeding disease. This includes the recent approval for its hemophilia A drug Esperoct and an R&D tie-up with Bluebird Bio for its gene therapy approach to the disease. Concizumab…

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It would never be good timing for a pandemic. But with COVID-19 beginning its spread in the first months of the year, it could be especially unfortunate for patients with high deductibles who catch the virus, according to a new analysis from the Peterson-KFF Health System Tracker, a partnership between the Peterson Center on Healthcare and the Kaiser Family Foundation. Most people have not accrued much health spending to satisfy their deductibles this early in the year. And with the cost of hospital admissions…

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Dive Brief: Novartis needs to sharply cut the price of its new sickle cell drug Adakveo and Global Blood Therapeutics needs to steeply discount its therapy Oxbryta in order to meet cost-effectiveness thresholds, the Institute for Clinical and Economic Review said in a review of new drugs for the blood disorder.  Adakveo significantly reduced episodes of pain stemming from the blockage of blood vessels, leading to a conclusion that it delivers a net health benefit, ICER said. Oxbryta was only able…

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Biosimilar sales are expected to nearly triple in the coming years, from about $2.5 billion last year to more than $6.5 billion in 2024, according to an S&P Global Ratings analysis cited by STAT.  Biosimilars are lower-cost versions of biologic drugs created to bring down overall drug spending and designed to have the same health outcomes. The first biosimilar was approved by the FDA in 2015, and since then, 26 biosimilars have received approval.  Biosimilars were created to combat higher drug…

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For the first time, doctors have used a CRISPR gene editing therapy in an attempt to fix broken genes within the body, marking another step forward for a technology that promises to change how some inherited diseases are treated. Clinicians at Oregon Health and Science University recently injected the therapy, developed by biotech Editas Medicine and partner Allergan, into the eye of a patient with a type of severe blindness, the companies confirmed Wednesday. A study last year tested another…

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In an increasingly crowded multiple sclerosis market, Merck KGaA’s Mavenclad has scrapped for market share against behemoths such as Roche’s Ocrevus. But a year into its U.S. launch, Mavenclad is posting exponential growth. In 2019, Mavenclad raked in €321 million ($358 million) in global sales, a more than 250% increase from the €90 million it posted the previous year. That significant leap has more than offset the sales loss from Merck KGaA’s older MS med Rebif and represents a more than 5% share of…

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According to a new report from the Leonard D. Schaeffer Center for Health Policy and Economics, a $1 increase in drug rebates was associated with a $1.17 rise in list prices, hurting uninsured patients who pay list prices and insured patients who pay coinsurance and deductibles based on list prices. The authors noted that there are competing theories about the drivers behind growing list prices. Some argue that manufacturers cause the increases because they unilaterally set list prices. Others point…

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A regulatory pathway that aims to ensure drug safety is inflating healthcare spending by billions of dollars, according to a new report.  Four widely used drugs funneled through the Unapproved Drug Initiative will increase spending by more than $20.25 billion over a five-year span as manufacturers hiked prices between 525% to 1,644%, a new Vizient analysis found. Vizient identified another 19 unapproved drugs that could increase costs by $8.75 billion.  “Our members remember these drugs costing dollars, now they are seeing up…

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Direct-to-consumer drug ads tend to strike a dissonant chord. However, fan and foe can count on one thing: Prescription DTC advertising is here to stay. The evidence? Surviving more than 20 years of numerous legal, regulatory and perception challenges on its way to becoming a $6 billion-plus annual industry. That’s mostly because DTC works. Study after study finds that patients follow the well-worn DTC advice and actually “ask your doctor” about a condition or a specific medicine after they’ve seen ads…

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