National Pharmaceutical Council’s Post

🌟 Exciting times ahead in the Cell and Gene Therapies (CGT) market for Hemophilia A and B! 🌟 In 2023, the CGT market for Hemophilia A and B soared to $12M with significant therapeutic expansions. 🚀 Our latest report dives deep into the competitive landscape, clinical trials, and future projections. 🌐 Discover insights from key opinion leaders across the 5EU, US, and China. 📊 We analyze unmet needs, regulatory challenges, and the reimbursement environment. 🔍 https://lnkd.in/gMWqaGUU Pharmaceutical-Technology #Hemophilia #GeneTherapy #CellTherapy #HealthcareInnovation #Biotech #MedicalResearch #FutureOfMedicine

VIDEO: Experts at our recent seminar discussed the high costs & access issues of multimillion-dollar gene therapies, emphasizing the need for fair pricing & policy reform. Check out the full recap here: https://lnkd.in/gH9KKjGK

𝐏𝐟𝐢𝐳𝐞𝐫 𝐬𝐞𝐜𝐮𝐫𝐞𝐬 𝐅𝐃𝐀 𝐚𝐩𝐩𝐫𝐨𝐯𝐚𝐥 𝐟𝐨𝐫 𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 ✅ Today, The U.S. FDA has approved Pfizer's BEQVEZ™, a one-time gene therapy for adults with hemophilia B. 👉 This therapy has shown significant reduction in bleeds post-treatment compared to standard care, potentially eliminating the need for regular infusions of factor IX (FIX). Hemophilia B, a rare genetic disorder, causes frequent and prolonged bleeding due to a deficiency in FIX. BEQVEZ offers a promising alternative, reducing both medical and treatment burden over the long term. With over 38,000 people worldwide living with haemophilia B. Read more... #genetherapy #cellandgenetherapy #biotech #regenerativemedicine

With the steady growth in the number of cell and gene therapies (CGTs) over the past few years, access and reimbursement for these products remains a top concern among population-based decision makers. Strategies to manage the cost of these potentially multimillion-dollar products took a large focus at this year’s AMCP, as stakeholders shared organizational perspectives and predictions for the evolving landscape. Read our most recent Access Talk POV by Zachary to learn more about key trends in CGT access and reimbursement, and what this means for market access marketers. https://lnkd.in/eFhxizjX #MarketAccess #MosaicGroup #IPGHealth #AccessReimbursement

⚕ Potentially curative gene therapies such as these will play a pivotal role in relative market positioning and overall treatment landscape compared to legacy interventions. ✅ Just announced, a new #GeneTherapy US FDA approval: Pfizer scores for their 'one-time' treatment for adults with moderate to severe Hemophilia B, BEQVEZ (fidanacogene elaparvovec-dzkt). 💉 This single-dose treatment intends to replace the need for prophylactic/ongoing administration of blood clotting factors. 🤔 For therapeutic areas seeing an uptick in forthcoming gene therapies in development (or, having already launched), how might these innovations affect the #BrandStrategy moving forward? #Innovation #RareDiseases #Strategy #CompetitiveIntelligence #MarketIntelligence #Pharma #Healthcare https://lnkd.in/eVk38c7P

Check out my latest POV on cell and gene therapies, and how the access landscape continues to evolve!

The FDA has approved fidanacogene elaparvovec-dzkt (#Beqvez™) as a one-time gene therapy to treat adult patients with moderate-to-severe hemophilia B, according to Pfizer. Eligible patients include those who: - Use factor IX (FIX) prophylaxis therapy, or - Have or have had life-threatening hemorrhage, or - Experience repeated, serious spontaneous bleeding episodes, and, - Do not have neutralizing antibodies to AAVRh74var, an adeno-associated virus (AAV), which can be screened via an FDA-approved test. “A one-time treatment with Beqvez has the potential to be transformative for appropriate patients by reducing both the medical and treatment burden over the long term,” said Adam D. Coker, M.D., MS, director of the Penn Comprehensive and Hemophilia Thrombosis Program. Read more: https://lnkd.in/eZnGhK_B #RareDisease

Developing harmonized guidelines for cell and gene therapies (CGTs) is crucial to ensure consistent and safe practices across the globe in order to promote innovation while also safeguarding patient health and well-being. Similarly, establishing robust protocols for pharmacoepidemiological studies that use real-world data (RWD) is essential for evaluating the effectiveness and safety of new medicines in real-life clinical settings, enhancing evidence-based decision-making in healthcare. Read our blog to know more on the efforts being made for the harmonization of CGTs development https://lnkd.in/gTDJG_kA Are you looking for regulatory support for your cell and gene therapy products? Reach out to DDReg on info@ddregpharma.com or submit your query here https://lnkd.in/dky_SKD7 #regulatoryservices #harmonization #realworlddata #cell #gene #therapy #patient #safety

Gene&Cell Therapy >> Sarepta faces lower revenue for Duchenne gene therapy ahead of expected sales jump with expanded treatment label: Sarepta Therapeutics recorded lower-than-expected quarterly sales of its gene therapy for Duchene muscular dystrophy, sending the company’s shares down nearly 10% in after hours trading. The biotech announced $121.7 million in Elevidys revenue for the second quarter, which was both lower than the $133.9 million generated in the first quarter and Wall Street expectations of $143 million. The company has previously said that it was expecting flat sales of Elevidys this quarter, as the therapy’s initial label included only children ages 4 and 5 diagnosed with Duchenne, while the average age of diagnosis of the muscle disease is 5 years of age. “We would have been perfectly flat, but a couple of kids that were scheduled towards the back half of the quarter actually had a viral infection, and they’d been pushed into this quarter,” said Sarepta’s CEO Doug Ingram on the company’s earnings call. In June, the FDA granted Sarepta a wide label expansion for Elevidys, making the gene therapy available to around 80% of people with Duchenne muscular dystrophy. The decision to expand the label that widely was highly controversial, and FDA documents released at the time showed that Peter Marks, the FDA official that oversees gene therapy approvals, overrode staff reviewers in granting the expanded label. In light of the lower sales, analysts repeatedly asked about potential bottlenecks in getting Elevidys to patients, whether constraints ranging from manufacturing to the capacity at active treating centers. Ingram repeatedly responded that “we don’t have bottlenecks.” He said that the company projects it takes three to five months to get patients from starting the process to gene therapy infusion, and that it expects some growth next quarter with a big jump at the end of the year, following the broad FDA label expansion. The company noted it expects the new gene therapy to begin to cannibalize its older exon-skipping Duchenne drugs, and projected 2025 sales of $2.9 to $3.1 billion. Analysts did not explicitly ask about Sarepta’s move to censor critical comments toward the company made by a parent of a Duchenne patient at a conference, which was reported by STAT last week. The annual conference is run by well-known Duchenne patient advocacy group Parent Project Muscular Dystrophy and sponsored in part by Sarepta.  Sarepta demanded that the comments were removed from the video recording and the advocacy group obliged. “You haven’t delivered any of this evidence you’re supposed to have for your $3.2 million drug,” the parent, Catherine Collins, said in a video that has since widely circulated around social media. A Sarepta spokesperson said in an email to Endpoints News that “Sarepta and PPMD… #lucidquest #genetherapy #celltherapy

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> CMS to increase reimbursement for new sickle cell gene therapies: The Centers for Medicare and Medicaid Services (CMS) said Wednesday that it will increase reimbursements for two newly approved sickle cell gene therapies that could potentially reverse the debilitating effects of the disease. CMS typically reimburses 65% of the cost of a new technology, but will increase that to 75% for Vertex Pharmaceuticals’ and CRISPR Therapeutics’ Casgevy, as well as for bluebird bio’s Lyfgenia, according to a proposed rule. The change will take effect in the US government’s 2025 fiscal year and conclude at the end of the two- to three-year so-called “newness” period. In the 1902-page rule, CMS also says it’s still looking into whether Casgevy and Lyfgenia are substantially similar and should be considered as a single application for purposes of new technology add-on payment. “If we determine that this technology is substantially similar to Lyfgenia, we believe the newness period would begin on December 8, 2023, the date both Casgevy and Lyfgenia received FDA approval for SCD,” CMS said. The switch to 75% reimbursement comes as experts have expressed concerns about how state Medicaid programs will pay for the sickle cell gene therapies, with list prices of $2.2 million for Casgevy and $3.1 million for Lyfgenia. About half of all sickle cell patients in the US are covered by Medicaid. In the drugs’ first year on the market, likely less than 1% of eligible sickle cell disease patients in the US will be able to receive gene therapy, St. Jude Children’s Research Hospital hematologist Alexis Leonard predicted during a presentation last December, just after the approvals. CMS is now inviting public comments on whether Casgevy is substantially similar to Lyfgenia and whether these technologies should be evaluated as the same technology for purposes of new technology add-on payments. #lucidquest #genetherapy #celltherapy