June 25, 2023: Notable pediatric drug approvals; 2 new treatments for men; important cancer drug research; stimulant effects on task performance; aspirin and anemia; cancer drug shortage
Plus, a rare success story from a collaboration that dramatically lowered the price of a life-saving generic drug.
New Drug & Treatment Approvals
The U.S. Food & Drug Administration (FDA) approved Sarepta Therapeutics' Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy for treating Duchenne muscular dystrophy (DMD). The therapy, a one-time intravenous infusion treatment, produces a miniature version of a protein called dystrophin, a necessary protein that those afflicted (primarily boys) with DMD are lacking. The treatment is intended to slow the progression of the disease.
Elevidys was approved with some conditions: it is only for patients four and five years old with the confirmed genetic mutation and continued approval is contingent on the results of an ongoing Phase 3 clinical trial. The cost for a treatment is estimated to be $3.2 million, making it one of the most expensive treatments in the world. Sarepta has two other therapies approved to treat DMD: VYONDYS 53 and Exondys 51, both RNA-based treatments. DMD affects approximately one in every 3,500 newborn boys.
https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-certain-patients-duchenne-muscular-dystrophy
Individuals with Duchenne produce little or no dystrophin in their muscle. Without dystrophin, normal activity causes excessive damage to muscle cells, and over time is replaced with fat and fibrotic tissue. Click to view image of muscle tissue example from Sarepta Therapeutics
The FDA approved Pfizer's Talzenna (talazoparib), to be taken with enzalutamide, as a once-daily oral treatment for homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (mCRPC). Talzenna was originally approved in 2018 to treat advanced BRCA-mutated, HER-2 negative breast cancer.
https://www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-talazoparib-enzalutamide-hrr-gene-mutated-metastatic-castration-resistant-prostate
The U.S. FDA approved Ironwood and Abbvie's Linzess (linaclotide) once-daily capsules to treat functional constipation (constipation with no known cause) in pediatric patients age 6 and up. Linzess is already approved for irritable bowel syndrome and idiopathic or functional constipation in adults.
https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-treatment-
Related to the approval, Linzess had a safety label change to warn of the risk of diarrhea and dehydration that may affect pediatric patients.
https://www.accessdata.fda.gov/scripts/cder/safetylabelingchanges/index.cfm?event=searchdetail.page&DrugNameID=382
Read Patient Reviews for Linzess on AskaPatient.com
The FDA approved Boehringer Ingelheim's Jardiance (empagliflozin) and Synjardy (empagliflozin and metformin hydrochloride) as additions to diet and exercise to improve blood sugar control in children 10 years and older with type 2 diabetes. Metformin, the only other oral therapy available for the treatment of children with type 2 diabetes, was first approved for pediatric use in 2000. Empagliflozin works by increasing the excretion of glucose in the urine.
https://www.fda.gov/news-events/press-announcements/fda-approves-new-class-medicines-treat-pediatric-type-2-diabetes
While type 2 diabetes among children is uncommon, the number of gestational and pediatric cases is growing, according to new research presented at ENDO 2023, the Endocrine Society's annual meeting in Chicago.
https://www.cidrap.umn.edu/covid-19/gestational-diabetes-pediatric-type-2-diabetes-increased-during-pandemic
Read Patient Reviews for Jardiance on AskaPatient.com
Read Patient Reviews for Synjardy on AskaPatient.com
The FDA approved U.K. company Futura Medical's Exeron, a drug-free topical gel for treating erectile dysfunction. The gel was approved as a "de novo device" because it does not use a drug in its formulation. Standard prescription treatments such as Viagra pills (sildenafil citrate) can take up to an hour to kick in. Exeron can act within fifteen minutes and is already available as single-dose tubes in the UK and Belgium, priced at about $30 for a four-tube box. Exeron is expected to be available in U.S. stores by 2025.
https://www.upi.com/Health_News/2023/06/13/topical-gel-erectile-dysfunction-FDA/7371686661560/
Drug Research
A decade-long study led by Yale University has shown that once-daily pill osimertinib reduces the risk of dying from lung cancer by over half. The study involved 682 non-small cell lung cancer patients between age 30 and 86 with a mutated EGFR gene, a mutation present in a quarter of lung cancer cases worldwide. 339 randomly received the AstraZeneca-made osimertinib pill, while 343 were given a placebo. After five years, the non-placebo group observed a survival rate of 88 percent compared to the placebo group, which saw a survival rate of 78 percent.
"It is hard to convey how important this finding is and how long it's taken to get here," Nathan Pennell, an expert at the American Society of Clinical Oncology who was not involved with the study. Because it's not yet standard practice for lung cancer patients to be tested for EGFR mutations, the study highlights the need to identify patients with the biomarkers at the time of diagnosis and before treatment begins. The study was published in The New England Journal of Medicine.
https://futurism.com/neoscope/scientists-pill-lung-cancer-deaths
Researchers from University of Melbourne studied the use of stimulants by people without a diagnosis of attention deficit hyperactivity disorder (ADHD). Study participants were young adults ages 18 to 35 without a mental health condition. Surprisingly, after a dose of one of three so-called "smart drugs," participants had small decreases in accuracy and efficiency on a cognitive task, along with large increases in time and effort, compared with task performance without taking the drugs.
The three "smart drugs" used in the study were modafinil (brand name Provigil), methylphenidate (sold under various brand names including Ritalin, Concerta, and others), and dextroamphetamine (ingredient is in various brand names including Adderall). The study was published in the journal Science Advances.
https://www.unimelb.edu.au/newsroom/news/2023/june/smart-drugs-can-decrease-productivity-in-people-without-adhd,-new-study-shows
A Cleveland Clinic study suggests that testosterone replacement therapy is safe for men with “low T” who have heart disease or are at high risk for it. More than 5,000 men ages 45-80 at 316 trial sites throughout the U.S. were randomly assigned to get the testosterone gel or the placebo, which they used daily for about 22 months. “Major cardiac events” occurred in 182 patients in the testosterone group and 190 patients in the placebo group. However, the testosterone group had a higher incidence of less severe problems, such as atrial fibrillation, acute kidney injury and issues from blood clots in veins. Doctors warn the popular treatment is no “anti-aging tonic." The study was published in The New England Journal of Medicine.
https://apnews.com/article/testosterone-replacement-therapy-low-t-safety-study-702598905c3f650576247a5d7322fcdf
Scientists from the joint U.S. and Australian study called Aspirin in Reducing Events in the Elderly (ASPREE) found that regular low-dose aspirin use led to increased incidence of anemia in otherwise healthy older adults. A follow-up analysis of data suggests that daily low-dose aspirin increases the risk of anemia in people age 65 years and older by approximately 20%. Given these findings, older adults on low-dose aspirin and their care providers may want to consider periodic monitoring of red blood cells or hemoglobin. Anemia in older adults is associated with functional decline, increased fatigue, disabilities, depressive symptoms, and cognition problems. The study was published in the Annals of Internal Medicine.
https://www.nih.gov/news-events/news-releases/low-dose-aspirin-may-increase-risk-anemia-older-adults
Drug Shortages
As part of its investigation into the shortage of cancer drugs, KFF News interviewed ten cancer clinicians, who said that shortages force them to prioritize patients who can be cured over later-stage patients, in whom the drugs generally can only slow the disease, and for whom alternatives — though sometimes less effective and often with more side effects — are available. A recent survey of academic cancer centers revealed that 93% couldn’t find enough carboplatin and 70% had cisplatin shortages. Prices for chemotherapy drugs carboplatin, cisplatin, and others have skyrocketed on the so-called gray market, where speculators sell medicines they snapped up in anticipation of shortages. A 600-milligram bottle of carboplatin, normally available for $30, was going for $185 in early May and $345 a week later, said clinic pharmacist Richard Scanlon.
Why the shortages?
While post-covid supply chain issues and limited FDA oversight of manufacturing practices overseas (where most generics are produced) are part of the problem, experts say the main cause is the underlying weakness of the generic drug industry. Old but crucial drugs are often sold at a loss or for little profit. Domestic manufacturers have little interest in making them, setting their sights instead on lucrative high-priced drugs that tend to target small patient populations. The FDA listed 138 drugs in shortage as of June 23, including many essential medicines.
https://kffhealthnews.org/news/article/drugmakers-are-abandoning-cheap-generics-and-now-us-cancer-patients-cant-get-meds/
In the U.S., stimulant and ADHD drug Adderall has been in short supply since August 2022, after a labor shortage at Teva Pharmaceutical, the largest manufacturer of brand name and generic Adderall. Since then, other ADHD drugs, like Takeda's Vyvanse, have seen surges in demand and experienced shortages of their own.
https://fortune.com/well/2023/06/20/adderall-alternative-vyvanse-adhd-drug-shortages/
Novo Nordisk is suing compounding pharmacies, medical spas, and weight loss clinics for allegedly selling unauthorized versions of its popular weight loss and diabetes drugs Wegovy and Ozempic, which contain the patented ingredient semaglutide. Novo Nordisk is the sole patent owner and does not sell the ingredient to outside entities, raising questions about what is actually being sold to consumers. The company said it is suing clinics and pharmacies for "false advertising, trademark infringement and/or unlawful sales of non-FDA approved compounded products claiming to contain semaglutide."
Although making compounded versions of commercially available drugs is permissible under special circumstances, like a shortage, state regulators are concerned with medications that are made using semaglutide sodium salt — a cheaper and modified version of the compound that is used for scientific research but not intended for use in humans. Ozempic and Wegovy are both in short supply in the U.S.
https://www.nbcnews.com/health/health-news/novo-nordisk-sues-clinics-allegedly-selling-copycat-versions-ozempic-w-rcna90187
The FDA has warned not to use off-brand versions of Ozempic and Wegovy, citing safety concerns.
https://www.fda.gov/drugs/postmarket-drug-safety-information-patients-and-providers/medications-containing-semaglutide-marketed-type-2-diabetes-or-weight-loss
Drug Pricing Success Story
For decades, Philadelphia business executive Drew Katz paid about $1 a pill to treat his case of Wilson's disease, a dangerous condition that causes copper to accumulate in the body. The medication, called trientene, helps his body rid itself of the copper. Without the drug, the condition would be fatal. He noticed the cost of the pill rising, slowly at first and then steeply, to more than $200 a dose in 2015. Even when generic versions came along, the price was still high. Katz worked with entrepreneur Mark Cuban and generic drug maker Dr. Reddy's on a lower-cost version of a drug for the disease. Mark Cuban's Cost Plus Drug Co. now sells trientine for $3.55 a dose, which is well below the $55 per dose that Medicare paid for a name-brand equivalent and other generic versions in 2021.
https://www.inquirer.com/health/mark-cuban-drug-costs-drew-katz-rare-disease-20230623.html
PharmedOut Conference Highlights
The 9th conference of Georgetown University's PharmedOut project brought together more than 150 medical doctors, public health experts, scholars, and students to look at the problem of "invented diseases and overtreatment." Normal human conditions and stages of life are increasingly "medicalized" so that they can be "monetized" by selling drugs or treatments. Some presenters also spoke about puzzling high drug prices. For example, why is a vial of insulin, once produced for five dollars, now $300? Here is a round-up of program highlights.
https://www.counterpunch.org/2023/06/23/georgetown-university-conference-addresses-invented-diseases-overtreatment/
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