So here is a question, is it better to have the drug but only help a few or not have it at all? Novartis is looking into whether the death of one severely ill baby treated with Zolgensma was related to the therapy. The FDA recommends the gene therapy, used to treat spinal muscular atrophy, remain on the market as the agency continues to review data, according to Peter Marks, director of the agency's Center for Biologics Evaluation and Research. On what grounds does the pharmaceutical industry have to charge that much? Insurance companies will be covering the Zolgensma. “But we can’t do anything to speed it up.”, By John Miller, Caroline Humer It points out, too, the expense of developing such innovative drugs. Once the genes reach their destination, the vectors are broken down and excreted from the body and do not become part of the child’s DNA. It's pretty depressing that in the future people will be more often told that they can't afford treatment instead of "there isn't any treatment". is able to offer advertisers an unmatched, high-quality audience worldwide. Photo: Novartis. If the entire world ran on socialist medicine, these drugs wouldn't exist. If that isn’t enough of a reason for why we shouldn’t use cost-effectiveness analysis as a basis for FDA approval, then I’m not sure what is. Pretty much zero. On May 24, the FDA approved Novartis’ new drug, Zolgensma, which now holds the title of the most expensive drug in the world at $2.125 million. Zolgensma is a gene therapy. Novartis is expecting European and Japanese approval later this year. Except aren't a lot of these drugs funded through public research? The problem is the business case for this drug is fundamentally flawed. All rights reserved. Thus, the big reason why Novartis set the price for Zolgensma so high: Its addressable market is minuscule. The FDA is requiring Zolgensma’s label to include a warning that acute serious liver injury can occur. "However, the integrity of the product testing data used in the development of the product’s manufacturing process is still a matter that we are continuing to evaluate and take very seriously.". 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The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. Can you keep a drug arrest secret from your employer and family? Subscribe to newsletters for the latest medication news, alerts, new drug approvals and more. Does Evrysdi cure spinal muscular atrophy (SMA)? “This is potentially a new standard of care for babies with the most serious form of SMA,” said Dr. Emmanuelle Tiongson, a pediatric neurologist at Children’s Hospital Los Angeles who has provided Zolgensma to patients under an expanded access program. The acting FDA Commissioner Ned Sharpless said that the approval marks “another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases.”, Even the Institute for Clinical and Economic Review, known for being an unofficial force for assessing the economic benefits of new medicines in the U.S, has endorsed Novartis’ pricing strategy. Simply put, I am a strong proponent of rewarding drug companies that have developed novel and efficacious drugs with a clear benefit in the lives of its patients. The FDA said the disclosure does not affect the agency's "positive assessment" of data from the company's human clinical trials. Parents of children with SMA have battled with insurance companies to gain coverage of the medication. Novartis has defended its position. The disease often leads to paralysis, breathing difficulty and death within months for babies born with the most serious Type I form. But the reality is that Zolgensma is literally the difference between life and death. That’s the thing. The Food and Drug Administration approved Zolgensma for children under the age of two with SMA, including those not yet showing symptoms. The orphan drug designation comes with a whole menu of incentives including a 50% of tax credit, fast track designation, accelerated approval, extended exclusivity, and priority review programs. Zolgensma (onasemnogene abeparvovec-xioi), Zolgensma (onasemnogene abeparvovec-xioi), How can a drug cost $2.1 million? Ekurd Daily, 22 years online, reaches over 6 million anually visitors worldwide. ©2020 BioProcess International All Rights reserved, BioProcess International US Conference & Exhibition, Bluebird: CMC and COVID delays push sickle cell gene therapy BLA back to 2022, Novartis continues Kymriah capacity expansion with Japanese CDMO, Bayer adds AAV-based gene therapy tech in $2bn AskBio buy, Thermo Fisher: ‘$700m COVID CapEx easily repurposed post-pandemic’, Single-Use Technologies: Innovation and Performance, Pall and Cytiva reaping $1bn coronavirus bonus for Danaher, J&J trial pause does not bode well for viral-vector COVID vaccines, analyst. So when they designed this drug their target market was millionaires children with this disease? Better than socialism, I'm told. The drug addresses the underlying causes of SMA according to Novartis spokesman Eric Althoff. To better understand ICER’s cost-effectiveness analysis, I also looked at the economic burden data for early onset SMA provided in a 2012 study conducted by the Lewin Group on behalf of the Muscular Dystrophy Association. Of course, it cannot be denied that some drug companies have also been able to reap the benefits of orphan drug designation including a high price, despite not actually developing something groundbreaking. ZURICH,— Swiss drugmaker Novartis on Friday won U.S. approval for its gene therapy Zolgensma for spinal muscular atrophy (SMA), the leading genetic cause of death in infants, and priced the one-time treatment at a record $2.125 million. Probably in most countries. Zolgensma From Novartis Is The Most Expensive Drug Ever Approved : Shots - Health News The Food and Drug Administration approved a new gene therapy for … Only 1 out of every 12.5 potential drugs ever reaches patients, the average drug takes 11–14 years to develop, and the costs of bringing a drug to market range from $1 to $2.6 billion. Zolgensma can be a one-time, life-saving treatment that allows … We comply with the HONcode standard for trustworthy health information -, We want to create an inspiring environment for our members and have defined a set of guidelines. Results from an open-label, single-arm clinical trial that enrolled 21 children (range of ages 0.5 to 5.9 months) who all received 1.1 x 1014 VG/kg Zolgensma reported: The easiest way to lookup drug information, identify pills, check interactions and set up your own personal medication records. This type of virus does not make you sick but can quickly travel through the body to the motor neuron cells and deliver the new gene. 13 of the 19 patients continuing in the trial reached at least 14 months of age without permanent ventilation, at the March 2019 data cut off, 10 of the 21 patients (47.6%) achieved the ability to sit without support for ≥ 30 seconds between 9.2 and 16.9 months of age (mean age was 12.1 months). Dr. Laurent Servais, a child neurologist in Liege, Belgium, called any delay in implementing newborn SMA screening “completely unethical.”. But ultimately, it is clear that for most parents, $2.125 million is a small price to pay to save their child’s life. This study is referenced in ICER’s final review for Zolgensma. Product can be stored for 12 months.”, Categories: BioProcess Insider, Therapeutic Class, Upstream & Downstream Processing. It is delivered by infusion. And just the profits are privatized? No insurance company will cover this drug for anyone saying insurance is the market. But this price is not without controversy. The vector in this case is a virus called AAV9 that has had its DNA removed and replaced with the SMN1 gene. In Zolgensma’s clinical trial and ongoing trial, infants and young children, who normally would have died before age two, are approaching their 5th birthdays, are able to breathe unassisted, and have shown improvements in motor milestones like being able to sit unassisted. Nothing could put a cost on saving my child’s life.” Now, it is important to note, that no patient in U.S. will actually have to pay even close to $2.125 million. Drug products combine active pharmaceutical ingredients with excipients in a final formulation for delivery to patients in liquid or lyophilized (freeze-dried) packaged forms — with the latter requiring reconstitution in the clinical setting. Novartis $2 million gene therapy for rare disorder is world’s most expensive drug, May 2019. Drug development is a long and challenging process from drug discovery to approval and it’s a highly regulated industry. Many technologies are used to characterize biological products, manufacturing processes, and raw materials. Insurers will be able to pay for the treatment over 5 years and Novartis will also provide a discount if a patient on the drug dies or needs permanent ventilator support. A review in April by an independent U.S. group, the Institute for Clinical and Economic Review (ICER), concluded Novartis’ value estimate for Zolgensma was excessive. Novartis said the drug is priced at half of the estimated $4 million-plus cost of managing the disease with therapy for one decade. It does this by using a vector, which is a “carrier” that can get the new, working SMN1 gene into the body. Private insurance. How many American's insurance do you think covers a drug like this? The Food and Drug Administration on Friday approved the first gene therapy for a type of spinal muscular atrophy, a lifesaving treatment for infants that will also be the most expensive drug in the world. They are lobbying to make SMA screening standard for newborns in every market. However, most media coverage focused on the drug’s $2.1m price tag, setting it in the context of the wider debate about pharmaceutical prices and rare disease therapies. But unlike Zolgensma, which is administered just once, Spinraza must be taken for life. Partnerships of many kinds — from outsourcing to licensing agreements to consultancies — help companies navigate this increasingly global business environment. Zolgensma is also the first gene therapy for spinal muscular atrophy, a disease that robs children of their physical strength. The US government currently is heavily involved in assisting with funding. That's how the system works. I think the target was families with a baby that had spinal muscle atrophy.