The FDA has approved crovalimab-akkz (brand name, #PiaSky®) for the treatment of paroxysmal nocturnal hemoglobinuria in adults and pediatric patients aged 13 years and older weighing at least 40 kg. Read more: https://brnw.ch/21wL9gX #FDAApproval #RareDisease
Rare Disease Advisor’s Post
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Many (if not MOST) drugs on the market lack specific pediatric safety and dosage guidelines, meaning that health care providers must use these medications “off-label,” and guess (based on adult studies) how to use them to treat infants and children. The Best Pharmaceuticals for Children Act (BPCA) program (https://lnkd.in/eNFNPDKz) at NICHD supports research to find the safe and effective doses for infants and children.
Item of Interest: NIH-funded research leads to fluconazole labeling updates for use in infants
nichd.nih.gov
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TV Health expert. Pharmacist. Healthcare Lawyer (Brabners LLP), Former Chair- Royal Pharmaceutical Society
Legal update Amendments have been made to the Human Medicines Regs 2012- Pharmacists must either round up /down so patients receive their supply in the manufacturer’s original full pack when it comes to valproate-containing medicines. There will be exceptional circumstances in which the manufacturers full pack does not need to be supplied. Please carefully note the importance of patient leaflets and risk assessments in the below link. More guidance available from MHRA. https://lnkd.in/eyDVM5Es #PharmacistThorrun #HealthcareLawyer #MHRA
Full pack dispensing of valproate-containing medicines
gov.uk
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The FDA has approved #Rezenopy (naloxone hydrochloride) nasal spray for the emergency treatment of known or suspected opioid overdose as manifested by respiratory and/or central nervous system depression in adult and pediatric patients. Rezenopy is the 4th naloxone nasal spray to be approved joining Rivive, Narcan Nasal Spray, and Kloxxado. This approval follows the White House’s recent nationwide launch of the initiative ‘Challenge to Save Lives from Overdose” which calls for more training and better access to the lifesaving opioid overdose drug naloxone, in the wake of ever-increasing overdose deaths. https://lnkd.in/eKQN3gYm https://lnkd.in/ew-bJhaC
High-Dose Naloxone Nasal Spray Rezenopy Gets FDA Approval
https://www.empr.com
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Pragmatic Privacy & Security | AI & Digital Governance | Board Member
North America: Supreme Court Unanimously Rejects Challenge to FDA’s Approval of Mifepristone: On June 13, 2024, the Supreme Court of the United States (SCOTUS) unanimously held in FDA v. Alliance for Hippocratic Medicine that plaintiff physicians and pro-life medical associations lacked Article III standing to challenge the U.S. Food and Drug Administration's (FDA) regulation of mifepristone, a prescription drug primarily used in terminating pregnancy. Following the ruling, mifepristone remains available and approved. Also importantly, SCOTUS reasoned that a desire to make a drug less available for others does not establish standing to sue FDA. Since SCOTUS focused on standing as a threshold issue, the broader question of whether the FDA acted within its authority and jurisdiction to approve mifepristone remains unanswered. The post North America: Supreme Court Unanimously Rejects Challenge to FDA’s Approval of Mifepristone appeared first on Global Compliance News. -via @bakermckenzie
North America: Supreme Court Unanimously Rejects Challenge to FDA’s Approval of Mifepristone
globalcompliancenews.com
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Valproate: The Medicines and Healthcare products Regulatory Agency has issued new guidance (which came into force yesterday) relating to dispensing of medication containing valproate. Previously, patients that were prescribed an amount different to that contained in a full pack were given their medication in separate packaging resulting in the patient not always receiving all of the information about the risks of valproate-containing medicines. The new guidelines mean that pharmacists must now dispense valproate-containing medicines in the manufacturer’s original full pack (which includes specific warnings and pictograms together with a Patient Information Leaflet), with the amount dispensed being as close as possible to the amount on the prescription. The guidance applies to both NHS and private prescriptions in England, Scotland and Wales and should be regarded as good practice by pharmacists in NI. A copy of the dispensing guidance can be found here:- https://lnkd.in/d3YhBKkj #healthcarelaw #pharmacists #valproate #MHRA #dwf
Full pack dispensing of valproate-containing medicines
gov.uk
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Dupixent® Phase 3 Results Demonstrate Sustained Efficacy for One Year in Children with Eosinophilic Esophagitis (EoE) Positive Phase 3 trial results indicate that Dupixent (dupilumab) is effective and safe for up to one year in children aged 1 to 11 with EoE, a chronic condition causing eating difficulties and abdominal pain. The trial showed sustained efficacy, improvements in outcomes, positive caregiver reports, and weight gain. The medication's safety profile was consistent with known information, and adverse events were rare. The FDA is reviewing the application to approve Dupixent for children with EoE. The study highlights the potential of Dupixent to alleviate symptoms and improve the quality of life for these young patients. For more details please click the link! https://lnkd.in/djdfyvq6 #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical
Dupixent® Phase 3 Results Demonstrate Sustained Efficacy for One Year in Children with Eosinophilic Esophagitis (EoE)
https://marketaccesstoday.com
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Equating an infection to sepsis seems like bad faith on behalf of #pfizer Pfizer: "[T]heir claims can be broken down into two distinct contentions: (1) that #pfizer Pfizer should have included additional warnings (#xeljanz) in its initial proposed label during the FDA approval process and (2) that Defendant should have sought the FDA's approval to add new warnings after the initial label was approved." #21cfr #cfr #fda #fdaapproval #fdaapproved #pharmaceutical #pharma #law #litigation #litigation #litigationsupport #regulatoryaffairs #regulatorycompliance #cmc
She's now a quadruple amputee. Are you paying attention to 21 CFR 314.50 and your label?: Stube v. Pfizer Inc., 446 F. Supp. 3d 424 (2020) Contact Us at B.A. to make sure your label complies and to Learn More! "...On March 14, 2013, Mrs. Stube was prescribed #Xeljanz and she ingested it until the time of her injuries. On March 24, 2017, Mrs. Stube presented at the CHI St. Vincent Hospital in Hot Springs, Arkansas, complaining of shoulder pain after moving a kayak. She was discharged with an arm sling and, four days later, returned to the hospital with complaints of chronic pain, fever, nausea, vomiting, and shortness of breath. After admitting her, hospital staff instructed her to stop taking Xeljanz. The next day, she experienced septic shock due to Streptococcus Group A infection and ultimately experienced multi-organ failure, gangrene, and amputation of all four of her limbs. For Plaintiffs' post-approval claims to not be preempted, they must allege that newly acquired information existed such that Defendant could have unilaterally changed the Xeljanz label in accordance with the CBE regulation. Defendant (#pfizer Pfizer) became aware of several medical studies, trials, and analyses reporting a higher incidence rate of patients developing herpes zoster after taking Xeljanz, which was much higher than previously known. Further, Plaintiffs allege that, post-approval, Defendant became aware of 47,287 adverse events related to ingestion of Xeljanz—257 of which involved sepsis, with 55 of those resulting in death; 34 of which involved septic shock, with 16 of those resulting in death; 1,214 of which involved herpes zoster, with 7 of those resulting in death; 14 to 16 of which involved amputations; and 5 of which involved gangrene, with 1 of those resulting in death. Plaintiffs alleged that none of the above information had been previously disclosed to the FDA. Thus, they argue that the above information constitutes newly acquired information, thereby giving Defendant the ability to utilize the CBE regulation to change the Xeljanz label ..." #21cfr #cfr #fda #fdaapproval #fdaapproved #pharmaceutical #pharma #law #litigation #litigation #litigationsupport #regulatoryaffairs #regulatorycompliance #cmc
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Aspiring RA Professional I Graduate Student (MSRA) NEU I Student Member @RAPS I Biopharmaceuticals I Biologics I Medical Devices
🎉 New approval by the FDA 🎉 👍 The U.S. Food and Drug Administration (FDA) has given the green light to Aurlumyn (iloprost) injection, marking a significant milestone as the first-ever medication approved specifically for treating severe frostbite in adults. This groundbreaking approval opens new doors in healthcare, offering hope and relief to those facing the daunting prospect of amputation due to severe frostbite. 👣 Aurlumyn's key component, iloprost, acts as a vasodilator, working to widen blood vessels and prevent blood clotting. This mechanism is crucial in restoring blood flow to frostbitten tissues, a critical step in reducing the risk of amputation and promoting healing. 💡 Clinical trials have demonstrated the remarkable efficacy of Aurlumyn in treating severe frostbite. In a rigorous study involving 47 adults with severe frostbite, those treated with iloprost showed significantly lower rates of the bone scan abnormality predictive of amputation compared to those receiving other medications or no treatment alongside iloprost. This compelling evidence underscores the potential of Aurlumyn to make a tangible difference in the lives of frostbite patients. 📊 Moreover, the safety profile of Aurlumyn is well-established, with common side effects including manageable symptoms such as headache, flushing, nausea, and dizziness. However, it's important to note that Aurlumyn carries a warning regarding the risk of symptomatic hypotension, emphasizing the importance of patient monitoring and healthcare provider guidance. 🏆 This milestone achievement wouldn't have been possible without the dedication and innovation of Eicos Sciences Inc., the company behind Aurlumyn. Their commitment to advancing medical science and improving patient outcomes is truly commendable, and we're excited to see the positive impact Aurlumyn will have on the lives of frostbite patients. #FDAApproval #MedicalInnovation #FrostbiteTreatment #Aurlumyn #HealthcareAdvancement #EicosSciences #PatientCare #HopeForFrostbitePatients
FDA Approves First Medication to Treat Severe Frostbite
fda.gov
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Inpatient Pharmacist at Aga khan University Hospital | Uptodate Certified Trainer.
⭐️Medication Error Related to Look-Alike, Sound-Alike Drug. A physician wrote a prescription of Navidoxine (Meclizine, B6) for nausea vomiting for pregnant women with the instructions to every Q8 hourly. The patient presented the written prescription to a pharmacy assistant at their local community pharmacy for processing. Upon reading the prescription, the pharmacy assistant incorrectly assumed that Navidoxine was Novidate. So, He dispensed Novidate (ciprofloxacin) over Navidoxine which is NOT SAFE IN PREGNANCY and held in category C And pregnant lady intake Novidate continuously every Q8hourly for 3 months. ‼️The medication error (confusion of Navidoxine with Novidate) 🌟So please whenever purchase any medicines from the medical store/local pharmacies, please cross checked from the pharmacist or Doctor. ⛔️DEFINITION OF PREGNANCY CATEGORIES (FDA) Category C: animal studies have related adverse effects and there are no controlled studies in women OR studies in women and animals are not available. Benefit must exceed risk: 🌟The presented cases demonstrate that LASA medication errors may have various causes. In this case, the medication error (confusion of Navidoxine with Novidate) 🌟HOW TO MINIMIZE THE ERROR? ➡️In order to minimize the risk of medication errors due to LASA drug names, the concept of Tall Man lettering, that is, capitalizing parts of a drug name, has been proposed. which might have been useful to prevent the medication error in this case. ➡️Another strategy to avoid LASA medication errors may be the specification of the indication for which a certain drug is prescribed ➡️Also, Physicians should be aware of look-alike/sound-alike (LASA) drug names. Clearly legible drug prescriptions, preferably via electronic drug prescription systems. #clinicalpharmacy #AKUH #residentpharmacist #pharmacist #hospitalpharmacist
