RAS LSS Consulting’s Post

As part of the Module 1 from the "Pharmacokinetics" course from Novartis, today I studied the Routes of Administration of a drug. It caught my attention particularly, the difference between INTRACAVERNOUS and INTRACAVITARY administration. ✅ The first one, INTRACAVERNOUS administration, refers to the administration within a PATHOLOGIC cavity, such as the ones that could occur in the lung from a patient with tuberculosis. ✅ On the other hand, an INTRACAVITARY administration is the one that is done within a NON-PATHOLOGIC cavity, such as that of the cervix, uterus, or penis, or such as that which is formed as the result of a wound. These, and other routes of administration, such us EPIDURAL (from which the procedure to provide pain relief or a lack of feeling for labor and childbirth gets its name, as it's the administration upon or over the dura mater) or the INTRACORPORUS CAVERNOSUM administration, one that I personally hope not to experience in my life, you may find in the link below. I hope you find it useful and insightful. NOTE: This information is the one linked in the curse and presented by the FDA. You may also find interesting to review the ISO IDMP standard for this topic and others (pharmaceutical dose forms, units of presentation and packaging, the ISO 11239), available here: https://lnkd.in/eZAheFGu #Novartis #Swissmedic #EuropeanMedicinesAgency #FDA #Pharmacokinetics #DrugDiscovery #Learning #ContentCreation

Exciting news! 🎉 The U.S. FDA has accepted Sentynl's New Drug Application for CUTX-101, a potential breakthrough treatment for #MenkesDisease, with a PDUFA target action date of June 30, 2025 Matt Heck, President & CEO of Sentynl, shared: "Supported by clinical trial data spanning over decades to evaluate the safety and efficacy of CUTX-101, we are eager for the FDA to review the application for our product, which has the potential to be the first FDA-approved therapy for this devastating condition." This milestone brings hope to patients and families affected by Menkes disease. CUTX-101 has shown promising results, with a nearly 80% reduction in the risk of death compared to untreated patients[1][3]. Let's keep our fingers crossed for a positive outcome that could transform lives! #RareDiseaseResearch #FDA #Biotech #MedicalBreakthrough

Nipocalimab's potential to redefine treatment paradigms for generalized myasthenia gravis is a noteworthy development in the healthcare landscape. With promising Phase 3 results and ambitions for broader applications, J&J positions itself in a competitive arena of FcRn blockers. This evolution emphasizes the importance of creativity and strategy in pharmaceutical marketing. As we navigate these changes, let’s explore innovative ways to convey patient benefits and drive engagement.

Our regulatory consultants are often asked by clients, ‘When should I apply for orphan drug designation?’ If you’re in the early phases of developing a drug product with strong nonclinical proof of concept or mechanism of action data in in vitro cell lines or in vivo data in an animal model or with preliminary but promising readout from an early phase trial, and believe your candidate will improve an orphan disease, you may have the same question.  Read Hema Balasubramanian and Mamta Puri-Lechner's strategic considerations – the how, why, and when to apply for an orphan drug application: https://hubs.la/Q02JTLyt0 #ClinicalDevelopment #OrphanDisease #RareDisease #Regulatory #StrategicAdvising 

Noxopharm Limited (ASX:NOX, OTC:NOXOF) is preparing to launch a first-in-human Phase 1 trial assessing SOF-SKN™ drug candidate and Sofra™ platform for #AutoimmuneDiseases in its HERACLES or ‘Harnessing Endogenous Regulators Against CLE Study’. NOX sees the Australia-based trial as the first step in leveraging the Sofra™ platform’s potential to tackle the autoimmune disease market in areas such as #RheumatoidArthritis. Holding the trials within Australia will offer access to R&D tax incentives from the Federal Government and Australian expertise in #lupus research and early-phase clinical trials. The trial is planned to start in early 2025 and is designed to provide proof of concept for the skin disease that is caused by cutaneous lupus erythematosus (CLE). More at #Proactive #ProactiveInvestors #Biotech #Biopharmaceutcials #ASX #NOX http://ow.ly/4ENI105FPZa

It is important to have a proactive approach when it comes to minimizing obstacles that are associated with drug development for rare diseases. This is to ensure access to more therapies that address rare diseases. In support of these efforts while also encouraging innovation the US FDA’s CDER collaborated to review and evaluate educational opportunities across various topics in rare disease drug development. This blog covers the methodology and key findings that came out from the Learning and Education to Advance and Empower Rare Disease Drug Developers, or LEADER 3D https://lnkd.in/gxXkmaEc Meet us at the BIO International Convention in San Diego to discuss your regulatory requirements for rare disease drug approval. Schedule a meeting with us https://lnkd.in/gJt7Vxwv Write to DDReg on info@ddregpharma.com or submit your query here https://lnkd.in/gxXkmaEc #raredisease #pharma #pharmaceuticalmanufacturing #innovation #drugdevelopment #empowering #USFDA #BIO2024

We have completed an update report for Acurx following the release of the March quarterly report on 14 May 2024 and the business update provided on 15 May 2024. The Company reached a significant milestone with the end of Phase 2 meeting with the FDA in late April. At the meeting, the Company and the FDA reached an agreement on the protocol for the Phase 3 clinical trial for ibezapolstat in the treatment of CDI and the regulatory pathway for a new drug application (NDA) filing for marketing approval in the US. The agreed upon trial design is largely the same as anticipated, with the Company to run an international trial comprising two arms to be completed sequentially. The trials are required to comprise 450 patients each for a total of 900 patients. The primary endpoint for the trial will be non-inferior CC to vancomycin with secondary endpoints including non-inferior SCC and reduced recurrence rates. The ability to show non-inferior CC and reduced recurrence on the larger patient population will set ibezapolstat up to become a first-line therapy for the treatment of CDI, with the expectation that it would replace the current standard of care. We have reduced our price target for Acurx from $12.35 per share to $10.64 per share ($7.40 per share on a fully diluted basis and $8.13 per share using the treasury stock method). The reduction in the price target is due to the increased number of shares on issue after the issue of new shares under the ATM program in the March quarter and the increased costs associated with the Phase 3 trial due to the larger than expected patient population required by the FDA. Despite the reduction in the price target, IIR continues to view there to be material upside for Acurx with our price target representing a 373% premium to the share price as at 17 May 2024. The finalisation of the Phase 3 protocol was a significant milestone achieved by the Company with commencement of the first of the two Phase 3 trials expected in 4Q’2024 set to be the next major milestone for the Company. Ibezapolstat has shown strong Clinical Cure (CC), Sustained Clinical Care (SCC) and Extended Clinical Cure (ECC) for the treatment of CDI. Results from the Phase 2 trial show ibezapolstat to be noninferior to vancomycin with ibezapolstat having a more favourable impact than vancomycin on the microbiome, which is believed to a key reason for reduced recurrence rates. IIR views ibezapolstat to be an attractive target for big pharma with the drug candidate providing a novel target to extend the pipeline of antibiotics for companies and if the Phase 3 trial validates the reduction in recurrence rates the Company may prove to be an attractive target for those who have invested in drugs focused solely on rCDI, a market which may be impacted if ibezapolstat is approved.

The need for a dedicated FDA center for rare diseases is more critical than ever. With over 7,000 rare diseases affecting millions of people, a specialized FDA division could fast-track drug approvals, focus on tailored regulatory pathways, and drive innovation in rare disease treatments. This could be a game-changer for patients and the pharma industry alike, accelerating the development of life-saving therapies. #RareDiseases #FDA #DrugDevelopment #PharmaInnovation #LifeSciences #Healthcare #RegulatoryAffairs

We sadly, have another example highlighting concerns with the accelerated approval pathway. Oxbryta first received accelerated approval in 2019 for sickle cell disease age 12 and older and then received additional accelerated approval in 2021 for age 4 and up. Pfizer, the manufacture, is pulling it from all markets and stopping all trials involving the agent as of last week. Why? "Pfizer’s decision came a day before European regulators met to review Oxbryta’ safety. The European Medicines Agency started its review because of data from two trials, one that had a higher-than-anticipated number of deaths and another that showed more deaths among patients taking Oxbryta compared with those on placebo." Sickle Cell Disease is an indication where we need more treatment options, more focus, and more funding. The accelerated approval pathway was designed to bring these treatments forward quicker. But how do we reconcile approvals too soon leading to harm when additional studies and full approval may have caught this? #Pharmacist #Pharmacybenefits #Sicklecelldisease #accerlatedpathway https://lnkd.in/gDGxcGBs