October 29, 2023: Pediatric drug approvals and probiotics warning; safety label updates for Enbrel and Adderall; beating cancer with bots; epilepsy research; statins medication tips
New Drug Approvals
The U.S. Food & Drug Administration (FDA) approved Santhera Pharmaceuticals' Agamree (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in patients age 2 years and older. DMD is a rare disorder in boys that causes progressive muscle weakness due to a lack of muscle protein called dystrophin. While it doesn't slow down the progression of the disease more than current treatments, Agamree, an oral corticosteroid, causes less side effects than current corticosteroids used by DMD patients. In June 2023, the first gene therapy for DMD was approved but only is for use by four and five-year-olds. (check our June 2023 news on this.)
https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-approves-santheras-duschenne-drug-2023-10-26/
The FDA approved the expanded use of Voxzoco (vosoritide) for use by all pediatric patients with achondroplasia, instead of just pediatric patients age five and up. Voxzogo is the only approved therapy for children with achondroplasia, a rare and genetic disease that causes the most common form of dwarfism. This change will make the drug available to younger children and allows patients and physicians to initiate treatment earlier, potentially leading to a much greater growth effect. People with achondroplasia have a genetic mutation that causes a certain growth regulation gene called fibroblast growth factor receptor 3 to be overly active, which prevents normal bone growth. The drug inhibits the gene, stimulating bone growth in children.
https://www.biospace.com/article/fda-approves-biomarin-s-voxzogo-for-young-children-with-dwarfism/
The FDA approved the expanded use of Merck's immunotherapy cancer drug Keytruda in early-stage patients with non-small cell lung cancer who can get their tumors removed surgically. This extends Keytruda's use in combination with chemotherapy as a treatment given before surgery to shrink the size of the tumor in patients and also allows use of the drug as a follow-up treatment after surgery. This is Keytruda's sixth indication for resectable non–small cell lung cancer (NSCLC). NSCLC is the most common type of lung cancer, which is the leading cause of cancer death worldwide.
https://www.biopharmadive.com/news/keytruda-fda-approval-perioperative-adjuvant-neoadjuvant-lung-cancer/696790/
Drug Safety Label Updates
Rheumatoid arthritis (R.A.) drug Enbrel (etanercept) had drug safety label updates, related to warnings about not taking it if you have sepsis, tuberculosis, bacterial, fungal, or other infections. Also, patients should not take any live vaccines concurrently with Enbrel. The revised label also adds new sections on safety and effectiveness for pediatric use. It is now approved for active juvenile psoriatic arthritis in children 2 years of age and older.
https://www.accessdata.fda.gov/scripts/cder/safetylabelingchanges/index.cfm?event=searchdetail.page&DrugNameID=540
Patient reviews of Enbrel on Ask a Patient website
Stimulant/ADHD Drugs Label Updates
All strengths of Adderall, Adderall XR, Adzenys Xr-ODT, Dyanavel XR, Evekeo ODT, Mydayis, Xelstrym stimulant drugs had safety label updates. Extensive edits of the label included emphasis in the boxed warning section on the high potential for abuse and misuse and addiction. Also, more detail is provided on the risk of motor and verbal tics and worsening of Tourette's syndrome side effects.
DEXTROAMPHETAMINE Safety Label updates
Adderall Safety Label updates
The label updates content is the same for all of the drugs in this category. Look up each drug's safety label change at this FDA website link: https://www.accessdata.fda.gov/scripts/cder/safetylabelingchanges/index.cfm
Check out Ask a Patient’s list of stimulant drugs with links to patient reviews.
16 patients report tics, Tourette's, or tremors as a side effect of Adderall.
Here are similar patient reports for Dexedrine (Dextroamphetamine sulfate).
Warning about Premature Infants and Probiotics Use
After an infant developed sepsis and later died after being given a probiotic at a hospital, the FDA issued a warning letter to medical professionals advising not to give probiotics to hospitalized premature infants. The FDA warns that preterm infants given probiotics are at risk of potentially fatal infection because of the bacteria and fungi that may be contained in those supplements. The infant who died weighed less than 2.2 pounds and was given the probiotic Evivo with MCT Oil as part of in-hospital care. The FDA also said that it has not approved use of probiotics in infants of any age.
https://medicalxpress.com/news/2023-10-death-fda-hospitals-probiotics-preemies.html
Beating Cancer with Bots
The year is 2033. You’re sitting in the doctor’s office when your worst fears are confirmed. Your doctor shows you an X-ray with a small but suspicious mass. You're mentally preparing for surgery, and possibly months of chemotherapy, so you’re surprised when your doctor pulls out a syringe. “This should take care of it,” she says. “You’ll feel a slight pinch, and then we’ll check on it next week to see that it’s gone down.”
What's this treatment? Read Ask a Patient's featured post to learn how the emerging technology of nanorobots -- designed from human molecules -- may soon be used to treat cancer and more. A recent design of a hybrid molecule nanorobot is said to be a "breakthrough" for its treatment potential. Check out our article and infographic that explains the high-tech treatment.
https://askapatientnews.substack.com/p/beating-cancer-with-bots
Medication Tips: Statins
Are you taking a statin drug to treat your high cholesterol? Check out MedShadow Foundation's special report on statins. It includes an explanation of why statins are prescribed, how they affect people differently, side effects, possible drug interactions, and alternatives.
https://medshadow.org/statins-need-to-know/
Check out Ask a Patient’s list of cholesterol-lowering drugs (most are statins) with links to ratings and reviews:
Hyperlipidemia (High Cholesterol) Drug List and Patient reviews
Commonly taken statin medications include rosuvastatin, atorvastatin calcium, and simvastatin. Check these out and add your own review:
Rosuvastatin Reviews
Atorvastatin Calcium Reviews
Simvastatin (brand name Zocor) Reviews
Thank you for reading this issue. Next time we’ll present a special report on drug quality and a world map of active pharmaceutical ingredients (APIs) manufacturer locations.
Visit us as AskaPatient.com for drug ratings and reviews provided by patients and contribute your own experience with a medication or vaccine!
Note to readers: Ask a Patient® Health News (general edition, which covers non-drug health news like disease outbreaks, major food recalls, diagnosing disease, health care costs, patient privacy, medical ethics, medical mysteries, and more) will be sent on Tuesday, October 31. If you are not already subscribed, sign up today on our home page.