June 30, 2024: Treatments for COPD, excessive sweating, UC, colorectal cancer, pediatric narcolepsy, Duchenne's, depression, hot flashes from cancer drugs, lower back pain, heartburn
Also: childhood obesity recommendations, cancer drug shortages, opioid lawsuit, supplements poll.
New Drug Approvals
COPD Treatment
The U.S. Food and Drug Administration (FDA) approved Verona Pharma's Ohtuvayre (ensifentrine) as a maintenance treatment for COPD (Chronic Obstructive Pulmonary Disease). COPD is an ongoing lung disease that includes chronic bronchitis and emphysema. Ohtuvayre is the first inhaled therapy that achieves anti-inflammatory effects in the lungs without the same risks as inhaled corticosteroids (like Symbicort), which can increase risk for pneumonia. The drug is to be taken twice daily by oral inhalation using a standard jet nebulizer with a mouthpiece. (Note: the label and/or announcement is not yet published on the FDA website)
https://www.yahoo.com/news/fda-approves-copd-drug-developed-151819599.html
Hyperhidrosis (excessive sweating)
The U.S. FDA approved Botanix’s Sofdra (sofpironium) to treat hyperhidrosis, or excessive sweating for patients age 9 and older. It’s the first topical gel treatment specifically approved for this condition. The gel is applied under the arms once a day at bedtime. The most common side effects are dry mouth, blurred vison, mydriasis (pupils enlarged or dilated).
Currently, treatment options for hyperhidrosis include Botox injections and glycopyrrolate products like Robinul (pill) or Qbrexza (cloth application).
FDA approval document for Sofdra
Botanix press release.
Ulcerative Colitis
Abbvie’s plaque psoriasis and Chron's drug Skyrizi (isankizumab-rzaa injection) received expanded approval for treating moderately to severely active ulcerative colitis in adults.
FDA approval document for Skyrizi
https://www.healio.com/news/gastroenterology/20240619/skyrizi-snags-fda-approval-for-moderate-to-severe-uc-first-il23-approved-for-uc-cd
Tuberculosis
After almost 12 years, FDA granted full approval for tuberculosis (TB ) drug Sirturo (bedaquiline). Since 2012, it had been approved under the FDA "accelerated pathway," with full approval contingent on the results of clinical trials. Sirturo is indicated for pulmonary tuberculosis (TB) due to Mycobacterium tuberculosis resistant to at least rifampin and isoniazid, also known as multi-drug resistant tuberculosis (MDR-TB), as part of a combination therapy, for adults and pediatric patients (5 years and older, weighing at least 15 kg).
https://www.fda.gov/news-events/press-announcements/fda-roundup-june-25-2024
https://www.empr.com/home/news/fda-grants-traditional-approval-to-tb-treatment-sirturo/
Colorectal Cancer
The FDA granted accelerated expanded approval to Mirati Therapeutics' Krazati (adagrasib) pill plus cetuximab for adults with KRAS G12C-mutated locally advanced or metastatic colorectal cancer (CRC). Previously it had only been approved to treat NSCLC (non-small-cell-lung-cancer).
https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-adagrasib-cetuximab-kras-g12c-mutated-colorectal-cancer
New Pediatric Drug Approval: Narcolepsy Treatment
The FDA expanded the approval of Harmony Bioscience’s narcolepsy drug Wakix (pitolisant) to treat excessive daytime sleepiness (EDS) in patients aged 6 years and older with narcolepsy. The label says that the use of WAKIX in this age group was supported by one study of 110 pediatric patients with narcolepsy ages 6 to less than 18 years of age.
Revised Label with Pediatric Approval of Wakix
Unlike the narcolepsy drugs Provigil and Nuvigil, which are classified as DEA schedule IV (other Schedule IV drugs are valium and Lyrica), Wakix is not on a controlled drug list. For more, check out MedShadow Foundation’s explanation of the DEA’s schedule of drugs:
https://medshadow.org/drug-classifications-schedule-ii-iii-iv-v/
New Pediatric Drug Approval: Infantile Spasms Treatment
The FDA approved Pryos Pharmaceuticals' Vigavyde (vigabatrin) to treat infantile spasms in babies 1 month old to 2 years old for whom the potential benefits outweigh the potential risk of vision loss. Vigavyde has boxed warning about the risk of permanent vision loss, and comes with an associated Risk Evaluation and Mitigation Strategy (REMS) program that caretakers must adhere to in order to monitor vision and seizures. This is the first ready-to-use vigabatrin formulation for infantile spasms; other formulations are powders that must be first mixed with water.
https://www.morningstar.com/news/business-wire/20240617159224/pyros-pharmaceuticals-announces-fda-approval-of-vigafyde-vigabatrin-as-the-first-and-only-ready-to-use-vigabatrin-oral-solution
New Pediatric Drug Approval: Duchenne’s Muscular Dystrophy
The FDA expanded the use of Sarepta's gene therapy for Duchenne muscular dystrophy (DMD), Elevidys, for all patients 4 years and older. Previously, it had only been approved for ambulatory patients 4 and 5 years of age with DMD. Elevidys was also granted traditional approval, converted from the accelerated approval pathway.
https://www.fda.gov/news-events/press-announcements/fda-expands-approval-gene-therapy-patients-duchenne-muscular-dystrophy
This expanded approval is controversial because it was made by a top FDA official, against the advice of FDA staff reviewers.
https://medcitynews.com/2024/06/sarepta-gene-therapy-duchenne-muscular-dystrophy-fda-approval-peter-marks-elevidys-srpt/
Cancer Drug Shortages
The FDA announced an end to the national shortage of cancer chemotherapy drug cisplatin. Since announcing the cisplastin shortage in February 2023, the FDA has taken specific steps, including not enforcing importation requirements for cisplatin to help meet patient needs during the shortage. The FDA says the total number of approved manufacturers of cisplatin is now seven.
https://www.fda.gov/news-events/press-announcements/fda-roundup-june-28-2024
Nevertheless, cancer drug centers report that other key cancer drug treatments remain in short supply.
https://www.kcci.com/article/cancer-centers-warn-drug-shortages-continue/61420938
Purdue Pharma’s Sackler Family Loses Financial Shield
The U.S. Supreme Court ruled (5-4) that the Purdue Pharma opioid settlement deal violated bankruptcy law by shielding Sackler family members from future litigation without the consent of potential plaintiffs. As part of the original settlement, the family, which controlled the company, agreed to pay $6 billion that could be used to settle opioid-related claims, but only in return for a complete release from any liability in future cases. The government said the family shielded $11 billion from lawsuits by moving it to personal offshore accounts, trust funds, and other tax havens. The ruling means that victims’ and communities’ settlement funds payouts are on hold until a new deal is reached, but perhaps the new settlement amounts could be larger.
https://www.nbcnews.com/politics/supreme-court/supreme-court-blocks-purdue-pharma-opioid-settlement-rcna139066
Weight loss recommendations for Kids: behavior and diet interventions, not drugs
The U.S. Preventative Services Task Force (USPSTF) recommended against endorsing the prescribing of weight loss drugs (including Wegovy and others) to children and adolescents because of the lack of “totality of evidence,” long-term studies and the harms of gastrointestinal symptoms. Instead, the USPSTF recommends providing children and adolescents 6 years or older with a high BMI to comprehensive, intensive behavioral interventions. The recommendation, which was an update to its 2017 USPSTF recommendation statement on screening for obesity in children and adolescents 6 years or older, was published in a letter in JAMA.
The American Academy of Pediatrics supports use of drug treatments and surgical interventions for children age 12 and up with obesity, in addition to lifestyle interventions. Some editorials express disappointment and disagreement with the USPSTF recommendation.
The letter in JAMA from USPSTF:
https://jamanetwork.com/journals/jama/fullarticle/2820244
Stat News Coverage:
https://www.statnews.com/2024/06/18/children-obesity-behavioral-therapy-wegovy-recommendations/
This article from “Your Local Epidemiologist” says that more than 30,000 children between 12 and 17 used the Wegovy-type medications in 2023:
https://yourlocalepidemiologist.substack.com/i/145722437/what-about-kids
Drugs & Treatments Research
Acupuncture and Hot Flashes During Breast Cancer Treatment
Researchers at the Dana Farber Cancer Institute conducted three parallel, randomized trials in the United States, China, and South Korea to study the use of acupuncture in women undergoing endocrine therapy (like Tamoxifen or Tamoxifen Citrate) for hormone receptor (HR)-positive breast cancer. They found that acupuncture can provide significant improvements in hot flashes and other endocrine symptoms. The study was published in Cancer.
https://johnwiley2020news.q4web.com/press-releases/press-release-details/2024/Can-acupuncture-lessen-hot-flashes-and-other-side-effects-of-anti-hormonal-breast-cancer-therapy/default.aspx
Want to learn more about acupuncture? Check out our interview with an acupuncture practitioner:
Walking for Preventing Low Back Pain
Macquarie University’s Spinal Pain Research Group conducted the “Walkback” trial examine whether a program of walking combined with education could be effective in preventing recurrences of low back pain. They followed over 700 adults for three years who had recently had low back pain. Half of the adults were assigned to a walking program and given six educational sessions from a physiotherapist over six months; the other half weren’t given any intervention. They found that people in the walking group had fewer issues with back pain recurring and more time between back pain flares than those in the control group. The study was published in The Lancet.
https://lighthouse.mq.edu.au/article/june-2024/walking-away-from-pain-world-first-study-shows-dramatic-improvement-in-lower-back-trouble
Ketamine in a Pill for Depression Treatment?
A slow-release oral formulation of ketamine may be more effective and less dangerous than injectable or nasal spray versions, which must be administered in a clinic because of side effects like blood pressure spikes and psychiatric effects like dissociation. Researchers at Otue University developed a ketamine-containing pill called R-107 and gave it to 231 study participants, all of whom had a major depressive disorder that had not improved despite trying at least two antidepressants. They found that the tablet containing ketamine had antidepressant effects in more than 150 people who had not responded to other drugs. The study was published in Nature Medicine.
The People’s Pharmacy’s Joe Graedon discusses ketamine therapy and explains why it is unlikely that an official version of oral ketamine will become available in the US anytime soon.
https://www.peoplespharmacy.com/articles/oral-ketamine-vs-depression-what-took-so-long
Medication Tips: Famotidine
Do you take famotidine (Pepcid brand name) to relieve stomach acid, GERD, or heartburn? Find out when you might want to take this H2-blocker, depending on whether you’re taking it for occasional heartburn or a chronic condition. Also learn what drugs to avoid while you’re taking famotidine.
https://www.singlecare.com/blog/why-take-famotidine-at-night/
Pepcid reviews on Ask a Patient website
Multivitamin Study
A recent study by the NIH’s National Cancer Institute, published in Jama Network Open, suggests that multivitamins overpromise and under-deliver. “Multivitamin Use and Mortality Risk in 3 Prospective US Cohorts”
https://www.forbes.com/sites/ariannajohnson/2024/06/26/daily-multivitamin-use-in-healthy-adults-doesnt-decrease-risk-of-death-study-suggests-what-to-know-about-pros-and-cons-of-multivitamins/
Do you take multivitamins or other supplements that you feel help you stay healthy? Answer Ask a Patient’s quick poll:
Which of the following dietary supplements is the MOST important for YOUR health?
- Multivitamin
- Single Vitamins
- Probiotics
- Fish Oil
- CBD Oil
- Other
- I don’t take supplements
Thank you for reading this edition of the Drugs & Treatments newsletter. Visit us at AskaPatient.com for patient ratings and reviews and our health supplements poll!