A Record That Keeps Getting Broken: Hemophilia Treatment Poised To Become Most Expensive Drug In World
The gene therapy, which isn't officially priced yet, was extremely successful in trials. Its maker says that insurers seem on board with paying somewhere between $2 million and $3 million for the drug, which would break the previous record held by Novartis' spinal muscular atrophy drug. Experts warned when Novartis' drug was approved at its $2.1 million price that it was setting a bad precedent. In other pharmaceutical news: more updates from the JP Morgan conference, the science behind the Ebola vaccine, a diabetes pill, and more.
The Wall Street Journal:
BioMarin Explores Pricing Experimental Gene Therapy At $2 Million To $3 Million
BioMarin Pharmaceutical Inc. BMRN 0.29% is exploring pricing its experimental gene therapy for hemophilia patients between $2 million and $3 million if it is approved, which could make it the most expensive drug in the world. The company hasn’t yet announced a price for the therapy, but Chief Executive Jean-Jacques Bienaimé said in an interview at the J.P. Morgan Healthcare Conference that the drugmaker has spoken to insurers, who have indicated they are comfortable with the range. (Hopkins, 1/16)
Reuters:
Eli Lilly Targets Quarterly Deals Of $1 Billion-$5 Billion In 2020: CFO
Eli Lilly and Co aims to announce roughly one $1 billion to $5 billion deal every quarter in 2020, its chief financial officer told Reuters, as the U.S. drugmaker looks to build up its pipeline of future products. It will focus largely on earlier stage opportunities across key therapeutic areas including oncology, pain, immunology, and neurology, CFO John Smiley told Reuters in an interview at the JP Morgan Healthcare conference in San Francisco earlier this week. (1/16)
Stat:
Lilly Exec Says ‘There Are No Villains’ In The High-Priced Insulin Market
Several months ago, Eli Lilly launched LisPro, an authorized generic version of its Humalog insulin at half price, or $137.35 a vial. The move was designed to defuse anger over the rising cost of insulin because LisPro would be more affordable to the uninsured or those with high-deductible insurance plans, whose copays are, typically, tied to list prices. But last month, a pair of U.S. senators issued a report saying the vast majority of pharmacies failed to offer LisPro and the effort was a bust. Lilly chief executive David Ricks called the findings “nonsense,” and argued the real access problem has been with middlemen — the pharmacy benefit managers — that prefer products with higher list prices because these offer higher rebates. (Silverman, 1/16)
Reuters:
Novo Nordisk's Diabetes Pill Rybelsus To Be Covered By Express Scripts
Novo Nordisk's new diabetes pill, Rybelsus, will be covered by Express Scripts Holding Co, one of the largest U.S. pharmacy benefit managers, the Danish drugmaker said on Friday. Pharmacy benefit managers act as middlemen in the drug supply chain, and negotiate discounts on drugs on behalf of health insurers. (1/17)
Stat:
The Public Science Behind The 'Merck' Ebola Vaccine
The U.S. Food and Drug Administration recently approved an important vaccine against Ebola, five years after an epidemic in West Africa killed 11,310 people and after more than 2,200 have died of it in the Democratic Republic of Congo in the last 18 months. Alex Azar, who heads the U.S. Department of Health and Human Services, quickly congratulated his department’s funding and “American global health leadership” for the vaccine, which is called Ervebo. (Herder, Graham and Gold, 1/16)
The Wall Street Journal:
Health Executives Gear Up For Change At J.P. Morgan Conference
The annual J.P. Morgan health care conference is taking place this week in San Francisco. Here are some of the hot topics under discussion at the four-day event, which wraps up Thursday. (Wilde Mathews and Loftus, 1/16)
Stat:
‘A Tragedy Of The Commons’: How A VC Wants To Lower Drug Prices
Aventure capitalist made some waves early in the J.P. Morgan Healthcare Conference when he announced his new company’s mission is to make medicines less expensive. After all, two of his previous investments have fueled drug makers using genetics to treat cancer — the kind of approach that has produced remarkable outcomes but also driven up the price of drugs. Alexis Borisy, known for co-founding Foundation Medicine and Blueprint Medicines and wearing felt fedoras, seeks to bring down prices at his new company, called EQRx. (Feuerstein and Herper, 1/17)
Stat:
A Venture Firm Warms To Funding Neuroscience
Longtime venture capital star Beth Seidenberg and former Amgen R&D chief Sean Harper say they have funded 10 companies from their new fund, Westlake Village BioPartners. But only three — the cell therapy companies Kyverna, Tmunity, and Arsenal Biosciences — are publicly known. Any hints? Neuroscience, Seidenberg said in an interview with STAT during the J.P Morgan Healthcare Conference in San Francisco. (Herper, 1/16)
Boston Globe:
How Swallowing A Star Might Keep You From Missing A Dose Of Medicine
Patricia Hurter makes her pitch by pulling a white capsule apart and withdrawing a plastic star-shaped structure. Hurter is the new chief executive of Lyndra Therapeutics, based in Watertown. She spent much of the J.P. Morgan Healthcare Conference in restaurants and hotel lobbies showing prototypes of a new drug delivery system designed to help patients stay on their medications. After a patient swallows the ordinary-looking capsule, the star’s six arms unfold inside the stomach and steadily release ingredients for a week; then the arms break off and leave the body like undigested food. (Saltzman, 1/16)
The Associated Press:
Three African Nations Roll Out First Malaria Vaccine In Closely Watched Trial
A pinch in the leg, a squeal, and a trickle of tears. One baby after another in Malawi is getting the first and only vaccine against malaria, one of history’s deadliest and most stubborn of diseases. The southern African nation is rolling out the shots in an unusual pilot program along with Kenya and Ghana. Unlike established vaccines that offer near-complete protection, this new one is only about 40% effective. But experts say it’s worth a try as progress against malaria stalls: Resistance to treatment is growing and the global drop in cases has leveled off. (1/16)