March 5, 2023: New treatment for leading cause of blindness; new generics; insulin price drop; FDA loophole that allows unsafe devices to be approved; rehab after heart attack; antidepressant tapering
Drug News
The U.S. Food and Drug Administration (FDA) approved Apellis Pharmaceuticals' Syfovre (pegcetacoplan) as a treatment to slow the progression of geographic atrophy (GA), an advanced form of dry age-related macular degeneration (AMD). Syfovre is taken as an injection in the eye every one or two months. GA is a leading cause of blindness and affects over 5 million people globally, including 1 million in the U.S. The disease typically causes patients to quit driving within two years of diagnosis. Apellis' pegcetacoplan (Empaveli brand name) was originally approved in 2021 to treat adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare disease that kills red blood cells. Empaveli is taken by subcutaneous infusion at a much higher concentration than Syfovre.
Pictured below is an illustrative example of what someone might see when they have Geographic Atrophy (left) and how it can worsen after six years (right).
CBS reporter Chloe Rafferty interviewed Bob Normandin, a Durham, NC patient who was diagnosed with GA 7 years ago and participated in the Syfovre clinical trial. Pleased with the results, Normandin plans to get the medication injection in his untreated eye.
https://www.cbs17.com/news/local-news/durham-county-news/durham-man-was-trial-patient-for-recently-fda-approved-treatment-for-leading-cause-of-blindness/
The FDA approved Travere Therapeutics' Filspari (sparsentan) a treatment to reduce proteinuria (high levels of protein in the urine) for a rare kidney disease, immunoglobulin A vasculitis( IgAN), in adults with a high risk of kidney failure. The drug has a boxed warning for liver and fetal toxicity and requires that patients register under a Risk Evaluation and Mitigation Strategy (REMS) program. It has not been established whether sparsentan slows kidney function decline in patients with IgAN.
https://www.medpagetoday.com/nephrology/generalnephrology/103204
The FDA released its 2022 annual report on generic drugs. In 2022, the FDA gave full or tentative approvals for 914 drugs, including 106 first-time generics. Notable brands made available as generics for the first time in 2022 include Restasis (for treating dry eye), Sovaldi (hepatitis C), Farxiga (type 2 diabetes), Jardiance (type 2 diabetes), Apokyn (Parkinson's), and Vimpat (seizures). The 42 page booklet is available as a PDF download.
https://www.fda.gov/drugs/generic-drugs/office-generic-drugs-2022-annual-report
The FDA issued an import alert for the veterinary tranquilizer xylazine. Under the alert, suspicious shipments of xylazine and the ingredients used to make it may be detained. Xylazine continues to be found in the nation's illicit drug supply, further complicating the fentanyl addiction problem.
https://www.cbsnews.com/news/fda-acts-to-restrict-illicit-import-of-xylazine-veterinary-drug-found-in-multiple-overdoses/
A safety label change was posted for the bipolar and anticonvulsant drug Depakote (divalproex sodium) to warn of drug interactions with methotrexate (potential increased symptoms of bipolar or frequncy of seizures) and cannabidiol (potential increased risk of ALT and/or AST liver enzyme levels). Other extensive changes were made to the medication guide (Click February 24 tab on link below).
https://www.accessdata.fda.gov/scripts/cder/safetylabelingchanges/index.cfm?event=searchdetail.page&DrugNameID=54
Patient reviews of Depakote and Depakote Extended Release
On the heels of a $35 a month insulin price cap that began in January for Medicare patients, Eli Lilly is now making its insulin (Humalog and Humalin) available in the U.S. to everyone for a $35 a month out-of-pocket cost. In this Kaiser Health News report, industry experts explain possible reasons for Eli Lilly's announcement and whether other suppliers may follow suit with price reductions.
https://khn.org/news/article/eli-lilly-slashed-insulin-prices-this-starts-a-race-to-the-bottom
Medical Device News & Research
Kaiser Health News and CBS News looked into what's behind the increasing number of lawsuits related to jaw and teeth injuries caused by the Anterior Growth Guidance Appliance (AGGA). Affixed by a dentist, the device is used to make the jaw "grow" and as a result cure ailments like sleep apnea and TMJ without surgery. In reality, say experts, it only shifts teeth dramatically over a period of months, causing some patients pain and tooth loss. The investigation reveals that AGGA was never reviewed by the Food and Drug Administration, the agency responsible for regulating medical and dental devices in the United States. According to court documents, over 10,000 patients have used the appliances. Includes a two-part news report running about 8 minutes each.
https://khn.org/news/article/watch-dental-device-at-center-of-lawsuits-was-used-on-patients-without-fda-review/
A research team from Yale School of Medicine and Harvard Medical School studied the extent to which a loophole in the 510K FDA regulation has allowed manufacturers to obtain approval for many unsafe medical devices.
Explanation of the 510K device approval pathway:
Under the 510K approval pathway, which is used for more than 90% of new medical devices, a product that has enough similarity to a previously approved device, called a "predicate," can bypass the clinical and lab study requirements that demonstrate its safety and effectiveness. If the predicate is recalled by the manufacturer, subsequent new devices can still be approved, for many "generations" of products, based on the potentially unsafe predicate device. This is the loophole, and patient advocacy groups and government watchdogs say the pathway lets far too many dangerous devices on the market, causing injury and even death to patients.
In the study, researchers analyzed the FDA paper trail of 156 devices approved under the 510K pathway that had Class 1 recalls (the most serious kind) between 2017 and 2021, to find out how many had a predicate that also had a history of recalls. They found that 44.1% of those products had used predicates with Class I recalls. In addition, 48.1% of these devices were subsequently used as predicates to authorize descendant devices later subject to Class I recalls. The risk of a Class I recall was 6.40 times higher for descendants that used predicates with Class I recalls than for devices using Class I recall–free predicates. The study was published in JAMA.
https://news.yale.edu/2023/01/19/loophole-enables-fda-approval-unsafe-medical-devices-yale-study-finds?page=1
Until recently, Melanie Carson, a 41-year old mother with Parkinson's disease, found leaving her house was exhausting, both physically and emotionally. Her medication caused dyskinesia, involuntary muscle jerks and spasms that made it hard for her to walk, drive, or even hold her young daughter. But after receiving a new treatment called focused ultrasound, her symptoms have improved and she can safely take her daughter for long outings. Carson was one of the first Parkinson’s patients to undergo the recently approved minimally-invasive procedure at the University of Maryland Medical Center that uses focused ultrasound to relieve symptoms of advanced disease and the side-effects of the medicine used to treat it. The $28,000 treatment is not covered by insurance, and Carson's parents sold a car to pay for her procedure.
Recently, a University of Maryland study showed that nearly 70% of treated patients had improvements in symptoms, compared to 32% of patients in the placebo ("sham treatment") group. Two-thirds of the patients who responded to the treatment continued to benefit from it a year later. The study was published in New England Journal of Medicine. This Baltimore Sun article features an interview with Carson.
https://www.msn.com/en-us/health/medical/night-and-day-parkinson-s-symptoms-eased-by-non-surgical-procedure-study-shows/ar-AA18aCQU
Drugs Research
A study led by University of Alabama researchers and funded by NIH's National Institute of Child Health and Human Development found that a single dose of the antibiotic azithromycin given vaginally to mothers during labor lowered their risk of sepsis by 35 percent. Azithromycin did not reduce the risk of stillbirth, newborn sepsis or newborn death. Results from the study, which enrolled more than 29,000 women in seven low- and middle-income countries, were published the New England Journal of Medicine.
https://www.scientificamerican.com/article/a-common-antibiotic-could-prevent-deaths-from-childbirth-complications/
Researchers from Samaritan Mental Health found that when antidepressants are discontinued, dose tapering is not routine but suggest that it should be. Using 7 years of medical records from Samaritan Mental Health, researchers determined that 8.9% of patients received a prescription for the lowest available dose of their antidepressant before it was discontinued, an indicator of a deliberate taper. They also found that antidepressant venlafaxine (brand name Effexor) tended to be tapered significantly more often than fluoxetine (Prozac) and paroxetine (Paxil). The authors say that since it is difficult to identify which patients will struggle when discontinuing antidepressants without a careful taper, a "one-size-fits-all" taper approach is recommended. The study was published in the Journal of the American Board of Family Medicine.
https://www.madinamerica.com/2023/01/researchers-seek-standardized-and-safe-antidepressant-tapering-protocol/
University of Cincinnati researchers reviewed both early and late-emerging side effects of antidepressant SSRI medications (such as Lexapro and Zoloft), and SNRIs (such as Cymbalta and Effexor) in children and youth age 7 to 17. Using publicly available data from the Child/Adolescent Anxiety Multimodal Study (CAMS), the largest trial of an SSRI in pediatric patients with anxiety disorders, they examined the course of specific physical symptoms over the 12-week study period. They found that some side effects emerge early and tend to resolve over the first weeks of treatment. These include headache, insomnia, and abdominal pain. Known late-emerging effects, which weren't included as part of the CAMs analysis, include weight gain and "persistent" effects like sexual dysfunction. The authors suggest that pharmacokinetically-informed approaches may address some adverse effects in pediatric patients. The study was published in Pharmacotherapy.
https://accpjournals.onlinelibrary.wiley.com/doi/10.1002/phar.2767
Research: Rehabilitation after Heart Attack
U.S. veterans who use home-based cardiac rehabilitation after a heart attack may lower their risk of death, according a study by the San Francisco Veterans Affairs Medical Center. Researchers reviewed medical data for 1,120 veterans treated at the medical center between August 2013 and December 2018 for heart attacks, angioplasty, coronary bypass grafting and other heart conditions, and found that a year after hospitalization, those who did not participate (more than half of those offered the program) had double the death rate compared to those who did.
The veterans, mostly men with an average age of 68, were given the option of enrolling in a 12-week, home-based cardiac rehabilitation program following their hospitalizations. The program included coaching calls, motivational interviews, a workbook, personal health journal, a blood pressure monitor, a scale and a stationary bike. Patients worked one-on-one with a nurse or exercise physiologist and follow-up calls were made to participants at three and six months. Participants were followed for an average of 4.2 years. The study was published in the Journal of the American Heart Association.
https://www.heart.org/en/news/2023/03/01/home-based-cardiac-rehab-may-lower-death-risk-for-us-veterans
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