Rosemarie Sellati

AbbVie today announced the U.S. Food and Drug Administration (FDA) has approved SKYRIZI® (risankizumab-rzaa) for adults with moderately to severely active ulcerative colitis, making it the first IL-23 specific inhibitor approved for both moderate to severe ulcerative colitis and moderate to severe Crohn's disease. SKYRIZI is now approved for four indications across immune-mediated inflammatory diseases. "When treating patients with ulcerative colitis, it's important to prioritize both early and sustained clinical remission as well as endoscopic improvement," said Edward Loftus, Jr., M.D., Maxine and Jack Zarrow Family Professor of Gastroenterology in the division of gastroenterology and hepatology at Mayo Clinic in Rochester, Minnesota. "This approval for SKYRIZI is an important step toward addressing these treatment goals." With over 1 million people living with ulcerative colitis, the United States has one of the largest populations affected by this disease and the numbers are continuing to rise. Ulcerative colitis is a form of inflammatory bowel disease (IBD) that causes inflammation in the digestive tract and can result in damage to the colon lining. Patients often experience a range of unpredictable symptoms that impact their daily lives, such as abdominal pain, bloody stool and urgency to use the bathroom. The disease course of ulcerative colitis varies between patients, and in some cases can lead to surgery or complications, including cancer or death. "Today's approval of SKYRIZI for ulcerative colitis expands our IBD portfolio and demonstrates our commitment to helping address ongoing needs of patients," said Roopal Thakkar, M.D., senior vice president, chief medical officer, global therapeutics, AbbVie. "We will continue to invest in transforming the treatment landscape and the lives of people suffering from lBD." Dosing of SKYRIZI for this indication includes a 12-week induction period with three 1200 mg doses delivered every four weeks, followed by maintenance therapy of either 180 mg or 360 mg delivered every eight weeks. Following the induction period, SKYRIZI treatment can be maintained at home using an on-body injector (OBI). The OBI is a hands-free device designed with patients in mind that adheres to the body and takes about five minutes to deliver the medication following preparation steps. #gastroenterology #immunology #ulcerativecolitis #CrohnsDisease  #IBD #SKYRIZI #AbbVie

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In many ways, #prescriptiondrug #affordabilityboards or #PDABs are the new frontier in the national #drugpricing debate. A growing number of states have recently established #PDABs, with widely varying organizational structures, compositions, and mandates. The field is evolving quickly, with little standardization of definitions, methodology or approaches, making it hard to make progress or even keep track. On the other hand, drug pricing is highly complex and a drug's 'price' (think for instance WAC, ASP or AMP) is often poorly correlated with what the drug actually costs #patients and #families and how affordable it is for them - something which may, in fact, fluctuate widely, including across different times of the plan year (e.g., based on progress towards made towards deductibles and copay limits). Unfortunately, this affordability of out-of-pocket costs for #patients and families is often not at the center of the PDAB debate. Great article in Bloomberg Law today summarizing the current state of play.

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Join FDA’s OCE and the American Cancer Society for what will be a stimulating symposium discussing Diversity in Cancer Clinical Trials. Topics of discussion are approaches to enhance diversity in oncology clinical trials (statistical, epidemiological and global considerations) and patient-centric approaches (e.g., use of decentralized trials and rural health considerations).

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⚔ Battle of the anti-#FcRNs in myasthenia gravis... 📑 Johnson & Johnson touts its recent nipocalimab PIII data showcased at the #EAN: European Academy of Neurology Conference, possibly competitively positioned well vs. argenx's #Vyvgart. ⏩ Forward looking: 👥 To leverage efficacy as a differentiator; across a range of antibody-positive gMG patients (i.e., MuSK+, anti-LRP4+, and anti-AChR+) 🗓 Regulatory filing est. by EOY #gMG #GeneralizedMyasteniaGravis #Clinical #MedicalAffairs #Nipocalimab #ActivitiesOfDailyLiving #Autoimmune #Neurodegenerative #Pharma #Healthcare

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Three weeks ahead of schedule, the FDA has approved GSK's dostarlimab-gxly (Jemperli) in combination with carboplatin and paclitaxel, followed by dostarlimab on its own, for treating adults with primary advanced or recurrent endometrial cancer. This approval moves dostarlimab beyond the mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H) space for endometrial cancer. It was based on results from part 1 of the phase 3 RUBY trial, which showed that patients receiving this combination lived significantly longer compared to those on standard chemotherapy. The median overall survival was 44.6 months for those on dostarlimab compared with 28.2 months for the placebo group. This equates to a 31% reduction in the risk of death.

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Congrats to Madrigal Pharmaceuticals on a great initial Rezdiffra launch! I listened to their Q2 earnings call and noted a question from one investment firm about MRE/PDFF availability, and its role in providing access to Rezdiffra for patients. Let me provide some numbers: 🔵 Installation numbers: there are 1.5x as many MRE locations (1,250) as Fibroscan (~800) in the US, which is generally regarded as widely available. So MRE access is certainly not a barrier to Rezdiffra. It is readily available to help tackle this important patient population. 🔵 Prior Authorizations: we're seeing commercial payers who rightly identify MRE as the only MRI tool capable of staging F2/F3 for Rezdiffra qualification. We're also seeing most payer policies identify PDFF for fat quant and monitoring. All of this is consistent with the MAESTRO-NASH trial. 👍 🔵 Cut-offs: PDFF >8% and MRE 3.3kPa to 4.9kPa = Treat with Rezdiffra For combo scores, go to coulditbenash.org 🔵 BMI dependent? No. Unlike ultrasound, elevated BMI does not affect MRE assessment or the cut-offs to achieve 90% specificity 🔵 Monitoring: critically, MRE was the only elastography tool significant for monitoring fibrosis regression in the MAESTRO-NASH trial. A 15%+ response in MRE equates to fibrosis regression. Again, not BMI-dependent. 🔵 Cost: an exam consisting of just MRE & PDFF costs just $205 through Medicare (CPT code 76391). It's very inexpensive compared to a traditional MRI exam. Clinicians should order it as MRE/PDFF only so that the Radiology practice does the rapid (10 minute), low-cost exam. 🔵 In sum, MRE/PDFF are widely available, low-cost, non-invasive & highly accurate. They will be key to delivering this breakthrough new therapy to patients who need it. We're proud to be a part of this new paradigm in liver care.

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Why Summits is excited for our upcoming 23rd Pharma & BioTech PPM Conference being held in Boston, Ma (Oct. 16, 17, 18). Please contact me or Simona Marčeková to discuss attending or speaking at this event. #PPM #Pharma #Conference #ProjectManagement

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Interesting articles in BioSpace earlier this month about FDA's #regulatoryflexibility and the varied ways in which it can support #raredisease #drugdevelopment. From #biomarkers and #accleratedapproval to #single-arm trials, #innovative #clinicaltrialdesigns, #decentralizedclinicaltrials, pilots like #FDA's START and RDEA pilots, and much more, regulatory flexibility can streamline the path to approval, shaving precious months or years of the drug development pipeline. https://lnkd.in/eTDpUgVK

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Pulmonary Arterial Hypertension Pipeline Update 2024: FDA Approvals, Therapeutic Advancements, Therapies and Key Companies involved by DelveInsight | Ribomic, Gmax Biopharm, Novartis, Cereno: (Las Vegas, Nevada, United States) As per DelveInsight’s assessment, globally, Pulmonary Arterial Hypertension pipeline constitutes 55+ key companies continuously working towards developing 55+ Pulmonary Arterial Hypertension treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and … Continue reading → #Business #HealthMedicine #MarketingSales #PharmaceuticalsBiotech

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Glad to see more commitment from Bristol Myers Squibb Bristol Myers Squibb Announces 10-Year Strategy to Reach More Patients in Low- and Middle-Income Countries PRINCETON, NJ / ACCESSWIRE / May 22, 2024 / Bristol Myers Squibb (NYSE:BMY) today announced ASPIRE (Accessibility, Sustainability, Patient-centric, Impact, Responsibility and Equity), a 10-year strategy to advance access to our innovative treatments and help patients in low- and middle-income countries (LMICs) gain access to potentially life-saving medicines. This strategy supports the company's commitment to reach more than 200,000 patients in LMICs by 2033 with its innovative treatments. "Access to potentially life-saving innovative medicines is very limited in some parts of the world, leaving patients with few treatment options for serious diseases like cancer," said Christopher Boerner, Ph.D., board chair and chief executive officer, Bristol Myers Squibb. "Bristol Myers Squibb believes patients who can benefit from our medicines should have access to them. Our ASPIRE strategy is an important step toward accelerating and expanding patient access to these much-needed treatments." As part of ASPIRE, Bristol Myers Squibb (BMS) is developing tailored strategies to increase affordability and availability of its medicines in LMICs. Our work to help ensure patients who need our potentially life-saving medicines can access them begins by ensuring that 100 percent of our marketed products are supported by access plans. Today we have defined access pathways for every country in which our medicines may be available to patients. In low- and middle-income countries, to help improve the affordability of our medicines, we have introduced local brands of many of our innovative medicines to address affordability issues, expand access, and help reduce the time lag between the availability of our medicines in higher-income countries and lower-income countries. In 2023, BMS filed a total of 11 local brands of which five received regulatory approvals. BMS local brands are making an impact in countries like Thailand, which has the highest prevalence of beta thalassemia, by providing treatment options like Rojusna™ to help combat this widespread disease. BMS is also making its medicines available where we don't have a commercial presence through the Direct Import and the Direct-to-Institution pathways. Through the Direct Import pathway, BMS is providing access to 12 medicines in more than 80 LMICs, using tiered pricing commensurate with affordability. The Direct-to-Institution (DTI) pathway is a scalable and sustainable channel that provides safe, broad, timelier access to BMS' innovative medicines to multiple patients through collaboration with local institutions. The company's initial DTI efforts are in five LMICs in East Africa and Pakistan and West Africa https://lnkd.in/gR42JzGj

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GSK, ViiV Tout 99% Effectiveness for Long-Acting HIV PrEP as Gilead Gains Ground Real-world evidence show that GSK and ViiV Healthcare’s long-acting pre-exposure prophylaxis Apretude can prevent nearly all HIV infections, while rival Gilead Sciences is moving quickly toward an approval for a twice-yearly option. GSK and ViiV Healthcare’s pre-exposure prophylaxis drug Apretude is more than 99% effective at preventing HIV infections, according to real-world results being presented at this week’s IDWeek 2024 conference. The results came from the real-world OPERA and Trio Health cohorts, which together enrolled almost 1,300 individuals, and “support the strong and sustained effectiveness of Apretude … outside the controlled environment of a clinical trial,” ViiV CMO Harmony Garges said in a statement. These data “add to the growing body of evidence generated over the last three years, showing [Apretude] for PrEP is a highly effective option for HIV prevention.” In Trio Health, which involved both cis- and transgender male and female participants, none of the patients taking Apretude PrEP were diagnosed with HIV during follow-up. https://lnkd.in/e25smmAE

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Harris vs. Trump: What the Presidential Election Could Mean for the Biopharma Industry As the 2024 election approaches, the close race between Vice President Kamala Harris and former President Donald Trump is sparking significant discussion about potential impacts on the biopharma sector. Analysts and industry stakeholders are weighing in on key issues like drug pricing reforms and regulatory policies that could shape biopharma's future under either administration. 🔴Potential Impacts Under a Trump Administration 🔴 Some believe a second Trump term could favor biopharma. According to BMO Capital Markets analysts Evan Seigerman and Kostas Biliouris, PhD, the industry might benefit from: 🔴Inflation Reduction Act Stability: The IRA may not expand and could be repealed. 🔴Less FTC Scrutiny: Potential decrease in Federal Trade Commission oversight on biopharma. 🔴Drug Pricing Reforms Targeting PBMs: Reforms may focus on pharmacy benefit managers (PBMs) over manufacturers. However, Trump's stance on drug pricing has been unpredictable. He has supported measures like price controls, not typically aligned with Republican positions, adding uncertainty for the industry. 🔵 Potential Impacts Under a Harris Administration 🔵 Vice President Harris's plan focuses on tackling high drug prices by addressing both PBMs and pharmaceutical companies. Key aspects include: 🔵 Cracking Down on Anti-Competitive Practices: Targeting practices that inflate drug prices. 🔵 Accelerating Medicare Negotiations: Speeding up talks to reduce medication costs faster. 🔵 Potential Use of "March-In Rights": Considering invoking Bayh-Dole Act provisions for reasonable pricing on federally funded drugs. While these measures aim to lower costs, some industry experts express concerns about unintended consequences. Invoking march-in rights could affect pharma-academic partnerships, potentially impacting innovation. The Industry at a Crossroads As election day nears, the biopharma industry is closely monitoring developments. The outcome could significantly influence regulatory policies, drug pricing reforms, and the business environment for pharmaceutical companies. Looking Ahead Regardless of the election outcome, biopharma will continue to face challenges in drug pricing, innovation, and regulation. Stakeholders are encouraged to engage in dialogue to find balanced solutions that promote both public health and industry growth. Join the Conversation What are your thoughts on how the election might impact biopharma? How do you see policies shaping the future of drug pricing and innovation? Share your perspectives below. Here a great article from BioSpace on this topic: https://lnkd.in/dv7dyphx

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Great session on the past, preset and future of #acceleratedapproval, in particular for #raredisease #drugdevelopment at #DIA2024 today. Great discussion expertly moderated by Danielle Friend, Ph.D. featuring Peter Marks, Alexis Reisin Miller and #NORD‘s very own Edward Neilan, MD, PhD. We at the National Organization for Rare Disorders have been strong proponents of the accelerated approval pathway since its inception (and we are supportive of the relatively recent #FDORA reforms). It was really great to see such strong support for this FDA program, and so much agreement that: - accelerated approval is really about bringing life-altering therapies to patients who desperately need them in a timely manner - on average years earlier than traditional approval - which is huge for #raredisease patients and families who in many cases essentially have no other treatment options; - accelerated approval meets #FDA’s statutory ‘substantial evidence of effectiveness’ standard; the uncertainty comes in because the surrogate or intermediate clinical #endpoint has not been fully validated to predict #clinical #efficacy; that’s where the ‘reasonable likely’ comes in - not in whether there is a measurable and significant treatment effect. - any assessment of the program must be data-driven and consider the overall benefits, risks and uncertainties surrounding the program. - the data clearly show, accelerated approval derisks rare disease drug development and shaves years of the approval process; at the same time, for the vast majority of #acceleratedapproval #drugs, clinical benefit *is* confirmed in a timely manner. A few ‘bad apples’ should not spoil the whole bunch (or sour public opinion about the pathway as a whole; but we know they unfortunately do) - enrolling patients in confirmatory trials after approval can be a challenge, in large part because patients may lack the incentive to participate. The new FDORA authorities may help, and there are a number of ways to help ensure timely trial enrollment and completion, including #realworldevidence #rwe and for some products long-term surveillance/post-market requirements of study subjects originally enrolled in the pivotal study. - every #raredisease #drugdevelopment program is different (loved the ‘snowflake’ analogy!) -and if and how accelerated approval may be used will be as well - and having this appropriate level of flexibility is really important for rare disease drug development. - leaning into #acceleratedapproval means every once in a while a product will turn out not to be effective. Having the new #FDORA withdrawal authorities is helpful (although most sponsors voluntarily withdraw their products). - continued questions and challenges to the #acceleratedapproval pathway by the Centers for Medicare & Medicaid Services and other public and private payers threatens to undermine this vital important pathway.

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💥 SUPER MSL: The Quest to Become the TOP MSL on Your Team💥 Don't miss this weeks MSL Talk podcast with Daniel Snyder, Ph.D., Senior Medical Affairs & HEOR Leader who discusses the differentiators for MSLs that will make you stand out and become the top MSL on your team. Learn more about….   👉 The qualities and differentiators of a “Super MSL” 👉 The skills and attributes that make MSLs stand out relative to their peers 👉 What common mistakes do MSLs need to avoid to 👉 What MSL Team leader should do to encourage and entice ideal MSL skills The MSL Talk Podcast is available on all podcast platforms and the MSL Talk YouTube Channel.     MSL Talk is a podcast that features helpful information for current Medical Science Liaisons, Medical Affairs leaders, job seekers looking to break into the pharmaceutical industry in their first MSL role as well as anyone interested in hearing good industry conversations and medical affairs discussions.   #msl #medicalaffairs #medicalscienceliaison #MSLtalk #careers  

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💥 SUPER MSL: The Quest to Become the TOP MSL on Your Team💥 Don't miss this weeks MSL Talk podcast with Daniel Snyder, Ph.D., Senior Medical Affairs & HEOR Leader who discusses the differentiators for MSLs that will make you stand out and become the top MSL on your team. Learn more about…. 👉 The qualities and differentiators of a “Super MSL” 👉 The skills and attributes that make MSLs stand out relative to their peers 👉 What common mistakes do MSLs need to avoid to 👉 What MSL Team leader should do to encourage and entice ideal MSL skills The MSL Talk Podcast is available on all podcast platforms and the MSL Talk YouTube Channel. MSL Talk is a podcast that features helpful information for current Medical Science Liaisons, Medical Affairs leaders, job seekers looking to break into the pharmaceutical industry in their first MSL role as well as anyone interested in hearing good industry conversations and medical affairs discussions. #msl #medicalaffairs #medicalscienceliaison #MSLtalk #careers

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COTA and the FDA will advance #cancerresearch using #realworlddata, focusing on #regulatory decision-making and improving treatment evaluation and availability. Read at The Evidence Base® #heor #healtheconomics #outcomesresearch #rwe #rwd #realworldevidence #pharma #biopharma #medicalaffairs #regulation #regulatoryaffairs #healthdata

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Natural history studies play a pivotal role at every step of the #raredisease #drugdevelopment pathway, from pre-clinical research to post-approval surveillance. Thank you Jen Farmer and Friedreich's Ataxia Research Alliance (FARA) for sharing this amazing story! The path to the first FDA approved treatment for Friedreich's Ataxis demonstrates the different ways in which natural history studies support rare disease drug development. As we also heard, it took 15 years, and involved many types of regulatory engagement between #patientadvocates and #FDA, including clinical conferences, confidential sponsor meetings, and Critical Path Institute (C-Path) partnerships in addition to Patient-focused drug development #PFDD meetings. What a great reminder that patient-focused drug development spans the whole #medicalproduct #lifecycle and is not limited to one single engagement between patients and regulators! And thank you Reagan-Udall Foundation for the FDA and FDA for hosting such an insightful public workshop today.

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Giovanni Caforio, formerly CEO of Bristol Myers Squibb, is set to become the next board chairman of Novartis, replacing Jörg Reinhardt upon his retirement in 2025. Known for his robust leadership during significant acquisitions at Bristol Myers—including the $74 billion purchase of Celgene—Caforio brings a wealth of strategic leadership skills and experience to Novartis. His appointment, announced alongside Novartis’ first-quarter earnings which exceeded Wall Street forecasts, is seen as pivotal for driving innovation and growth at Novartis. The company has been focusing on high-margin prescription pharmaceuticals and streamlining operations, a strategy that Caforio is well-equipped to advance given his track record in managing growth and navigating complex regulatory environments. Reinhardt, who has been instrumental in major transformations at Novartis since 2013, including strategic divestitures and focusing the company's core on lucrative areas, leaves a legacy of significant change. Caforio’s leadership is anticipated to maintain this momentum, tackling future challenges and seizing opportunities in the competitive pharmaceutical market. #Pharmaceuticals #LeadershipChange #StrategicGrowth #Novartis #Innovation #HealthcareIndustry #ExecutiveLeadership #PharmaNews

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