InsightRX’s Post

Excited to share our recent publication on,” Expanding Role of Endogenous Biomarkers for Assessment of Transporter Activity in Drug Development: Current Applications and Future Horizon”. The article was published in the Special Issue (Guest Editors Dr. Sook Wah Yee and Dr. Hong Shen) of Pharmaceutics on “New Insights into Transporters in Drug development”. Evaluation of transporter-mediated drug–drug interactions (DDIs) during drug development and post-approval contributes to benefit–risk assessment and helps formulate clinical management strategies. The use of endogenous biomarkers, which are substrates of clinically relevant uptake and efflux transporters, to assess the transporter inhibitory potential of a drug has received widespread attention. The article highlights how the incorporation of endogenous biomarker assessments as part of the overall DDI assessment strategy can provide mechanistic insights into observed DDIs and enable robust quantitative DDI predictions. Further, the article summarizes how endogenous biomarkers have shed light on alterations in transporter activity due to organ impairment or various disease states.  Terrific collaboration with Joe Ma and Kine Kvitne https://lnkd.in/erJrSi5H

Drug repurposing through label expansions cannot be emphasized enough in its potential to address patient unmet needs for newer and better treatments. This is also true for a drug's off-label use, but it is allowed for and reimbursed only in very few countries. Developed markets such as US and EU4 do not reimburse it for majority of drugs. In a recent example, Pfizer is currently seeking a broad FDA approval for prostate cancer patients for its PARP inhibitor drug, Talzenna after it demonstrated to increase life expectancy of patients with metastatic-castration-resistant prostate cancer (mCPRC) regardless of the tumor’s mutation status in phase 3 TALAPRO-2 trial. It might be the broadest FDA approval in this drug class. Why this can be an effective strategy for maximize reach and penetration of a drug? 1. It is most time and cost-effective mechanism to provide new medicines for patients for whom newer treatments didn't exist. 2. Manufacturers can probably adopt a dual-branding approach for a drug targeting either a broad indication or more than one indication. Pharma manufacturers can also charge a premium price for indications with a very high unmet need and disease burden. 3. Easy to gain positive reimbursement decision for a broader patient population, if the drug is already approved for a specific subtype of patients. 4. Ease of integration into treatment pathways 5. Physicians find it easier to manage treatment with an existing drug class on a newer group of patient population than a newer drug or drug class. 6. Increased market share and access coverage for the drug across various markets. Thus, planning ahead during pre-clinical or early clinical development phase can be an important strategic consideration for pharma / biotech developers. It can help them develop a financial, clinical and regulatory plan down the line, intellectual property considerations, provide ethical justifications for randomized controlled trials and formulate a forward-looking RWE strategy. It arms pharma manufacturers better for long-term sustained success of a clinically effective drug. Sources: Fierce Pharma; Rapid label expansion based on public private partnership Meeting abstract 2024 ASCO annual meeting; Label extensions: Benefits and challenges for drug repurposing, Donald Lo 3 October 2023, RExPO23 Conference; Drug repurposing: Misconceptions, challenges, and opportunities for academic researchers, C. Glenn Begley et al. Science Translational Medicine, 2021 P.S. - Strictly Human Generated #Labelexpansion #FDAapproval #drugdevelopment #strategy #drugapprovals

A Phase 3 trial wasn’t technically a condition for approval of Amylyx Pharmaceuticals' #Relyvrio, an ALS drug that was greenlit in the US and Canada in 2022. But there was still pressure, both from patient advocacy groups and from regulators. Billy Dunn, then the director of the FDA’s neuroscience office, went so far as to ask the company’s cofounders during a public meeting in 2022 if they’d be willing to pull their drug from the market should it fail in the Phase 3 study. The cofounders—Justin Klee and Joshua Cohen, who are also Amylyx’s co-CEOs—agreed. “If the drug’s not helping patients, then why would we want to be giving it to them?” Klee said at the time. But that’s now the situation Amylyx finds itself in. After being approved in the US and Canada based on a successful Phase 2 study, the drug failed to meet its primary goal in a Phase 3 study, the company announced last week. It didn't achieve statistical significance on its secondary goals, either. Amylyx has already stopped advertising the drug and may pull it from the market altogether in the next eight weeks. If Relyvrio is pulled from the market, patients with ALS will have just a few treatment options. Managing the disease comes down largely to supportive therapies, including speech therapy, dietary support, and physical and occupational therapies. There are two pharmaceutical ALS interventions: riluzole, an oral medication that can increase life expectancy by about 25%, and edaravone, an intravenous drug that slows the rate of functional decline. The average survival time for people with ALS is just 2–5 years after diagnosis. It’s possible that Relyvrio might be helpful in subgroups of patients, but while Amylyx researchers plan to examine that possibility, the company isn’t in the business of cherry-picking data for commercial reasons, Cohen says. “As a company, we’ve always tried to be extremely rigorous in the science,” Cohen says. “Our intention is to evaluate the rigorous data, the prespecified data, not to create some sort of post hoc narrative. The point here is to evaluate the real science, as it stands, and make the decision on that.” More on the future of Relyvrio here: https://lnkd.in/enc9TceV

⚡️⚡️FDA approves safety labeling changes for fluorouracil injection products. On March 21, 2024, the Food and Drug Administration approved safety labeling changes for fluorouracil injection products. This effort was a collaboration between FDA’s Office of Generic Drugs and the Oncology Center of Excellence (OCE). In addition, a new subsection 12.5 (Pharmacogenomics) has been added to section 12 (Clinical Pharmacology). The labeling changes align with those approved for another fluoropyrimidine drug, Xeloda (capecitabine) tablets, on December 14, 2022. #Pharmacogenomics #Cancer #oncology

It seems that lack of transparency and data sharing in the pharmaceutical industry is a real issue when it comes to developing potential or improved anti-cancer treatments. This is causing concern among cancer clinicians, researchers, and consumers. Despite commitments made by regulatory bodies in 2013, it appears that critical areas for improvement are still required in data sharing and collaboration within the entire industry to ensure that clinical trials are easily accessible to those who need it. If you're a practice managing a large chronic condition patient population, contact me to learn more about a new, unique, AI driven solution to enhance your patient engagement, and improve your long-term clinical results. #DragonMedicalOne #DAXCoPilot #Clinicaldocumentation #AIDrivenPatientEngagement  

Rznomics’ HCC Treatment Receives Orphan Drug Designation by FDA Rznomics' drug RZ-001 has been designated as an orphan drug by the FDA. This designation brings benefits such as marketing exclusivity, exemption from user fees, tax credits, and regulatory assistance from the OOPD. RZ-001 suppresses hTERT expression in cancer cells and demonstrated effectiveness in preclinical models. Rznomics also received IND clearance for Phase I/IIa studies in HCC. They have partnered with Roche and received approval for another drug, RZ-004, for a clinical trial in Australia. Overall, Rznomics is committed to advancing medical solutions for various diseases. For more details please click the link! https://lnkd.in/d8njKrEi #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical

A Drug Fails post Approval due to lack of Clinical Benefit and Concerns of Toxicity: Aduhelm abandoned For Alzheimer's Aducanumab (Aduhelm), a drug recently approved for the treatment of Alzheimer's Disease, was withdrawn from selling in the US. Biogen stopped an ongoing confirmatory clinical trial and abandoned its rights to the drug. Aducanumab (Aduhelm) was approved by the USFDA for the treatment of AD. The main questions about its approval was about lack of clear evidence of clinical benefit and concerns of toxicity. Learning: Only drug candidates which show meaningful clinical response with an acceptable safety profile (as opposed to objective responses whether CNS or oncology) in registration studies should be considered for full approval. Drugs granted accelerated approval (based on surrogate biomarkers or objective responses) should be evaluated for clinical benefit in confirmatory trials. https://lnkd.in/garPWSK8

START Program: FDA's Promise of Accelerating Rare Therapeutics Salma Saiger from SMS Clinical Research LLC provides an in-depth look at the FDA's START Pilot Program in "Understanding FDA's Rare Disease Therapeutics Pilot Program". A key highlight of the article is the enhanced communication between selected Sponsors and FDA staff, fostering a collaborative environment to address the unique challenges of rare disease drug development. Reflecting on the article, it's important to recognise the START Program represents a significant stride toward streamlining the process of rare disease research to patient access. The initiative's objective include accelerating the development of much-needed treatments, and emphasising the importance of collaboration between regulatory bodies and researchers. In drug development where efficiency and quality are paramount, programs like this are critical to bridge the gap between innovation and impact. Check out the full article: https://lnkd.in/e6u6dpub #clinicaltrials #clinicaldevelopment #regulatorycompliance #quality #risk #pharma #biotech

🌟 Exciting News from the FDA! 🌟 🔍 The FDA’s Center for Drug Evaluation and Research (CDER) has just released its "New Drug Therapy Approvals 2023" report. This comprehensive document, released on Jan 16, 2024, shines a light on the groundbreaking achievements in drug approvals over the past year. 💡 Key Highlights: • CDER approved a remarkable total of 55 novel drugs in 2023. • These drugs target a wide array of conditions, ranging from COVID-19 and RSV to neurological conditions, anemia, and type 2 diabetes. • 🚀 A significant milestone: Over half of these drugs were approved under the orphan drug designation program, focusing on rare diseases. • 🎗️ This includes treatments for conditions like Friedreich’s ataxia, rare cancers such as mantle cell lymphoma, and more. • 🆕 2023 also saw the approval of five biosimilars, bringing new options to the market. 📈 Efficiency and Impact: • CDER’s report also highlights their efficient review process, with 89% of drug approvals meeting the Prescription Drug User Fee Act goal dates. • 🥇 84% of these approvals were achieved on the “first cycle,” and 35 of the 55 novel drugs were approved ahead of other countries. • 🌍 36 drugs benefited from the FDA’s expedited programs. 🔬 A Collaborative Effort: • CDER Director Patrizia Cavazzoni emphasizes the collaborative nature of the approval process, involving expertise from various sectors. • She lauds the dedication of the teams involved, whose efforts significantly impact patient health and well-being nationwide. 👏 As we celebrate these advancements, let's acknowledge the hard work and dedication of everyone at the FDA and the broader medical community. These approvals are not just numbers; they represent hope, innovation, and a brighter future for patients around the world. #FDA #DrugApproval #HealthcareInnovation #Pharmaceuticals #MedicalResearch #PublicHealth