Ming-Hei Tai, PharmD, BCOP’s Post

FDA grants accelerated approval to asciminib for newly diagnosed chronic myeloid leukemia FDA has approved asciminib (Scemblix—Novartis) for the treatment of adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP). The drug's efficacy was evaluated in the ASC4FIRST trial, which randomized 405 patients to receive either asciminib or investigator-selected tyrosine kinase inhibitors (IS-TKIs). At 48 weeks, the major molecular response (MMR) rate was 68% in the asciminib group and 49% in the IS-TKI arm. The most common adverse reactions in the pooled safety population of patients with newly diagnosed and previously treated Ph+ CML in CP were musculoskeletal pain, rash, fatigue, upper respiratory tract infection, headache, abdominal pain, and diarrhea. The most common laboratory abnormalities in patients with newly diagnosed Ph+ CML in CP were decreases in lymphocyte count, leukocyte count, platelet count, neutrophil count, and calcium corrected. The recommended dosage of asciminib is 80 mg taken orally once daily at about the same time of day, or 40 mg taken orally twice daily at approximately 12-hour intervals. Asciminib received accelerated approval. https://lnkd.in/gMyJE6Hx

📢 FDA approves revumenib for relapsed or refractory acute leukemia with a KMT2A translocation (Syndax Pharmaceuticals, Inc.) ➤ Efficacy was evaluated in a single-arm cohort of an open-label, multicenter trial (NCT04065399) in 104 adult and pediatric patients (at least 30 days old) with R/R acute leukemia with a KMT2A translocation ➤ CR+CRh rate was 21.2% (95% CI: 13.8, 30.3), and the median CR+CRh duration was 6.4 months (95% CI: 2.7, not estimable) ➤ Among the 83 patients dependent on RBC and/or platelet transfusions at baseline, 12 (14%) became independent of RBC and platelet transfusions during any 56-day post-baseline period. Of the 21 patients independent of both RBC and platelet transfusions at baseline, 10 (48%) remained transfusion independent during any 56-day post-baseline period ➤ Due to an anticipated delay in commercial availability of the lowest dose strength of revumenib, which may be used to treat patients who weigh < 40 kg, revumenib will be available through an expanded access program to allow for dosing of patients who weigh < 40 kg (information available here: NCT05918913 -- https://lnkd.in/eqEA4ge8 ) ➤ This review used RTOR pilot program, which streamlined data submission prior to the filing of the entire clinical application, and the Assessment Aid. FDA approved this application 6 weeks early (https://lnkd.in/e2FdYf8a) #oncology #biotech #biotechnology #regulatoryaffairs #pharma #drugdevelopment #oncologytrials #oncologyresearch #cancer #oncology #regulatoryintelligence #regulatoryprecedent https://lnkd.in/e8_cz_xV

FDA grants accelerated approval to asciminib for newly diagnosed chronic myeloid leukemia On October 29, 2024, the Food and Drug Administration granted accelerated approval to asciminib (Scemblix, Novartis AG) for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP). Full prescribing information for Scemblix will be posted on Drugs@FDA.  Efficacy and Safety The efficacy of asciminib for newly diagnosed Ph+ CML in CP was evaluated in ASC4FIRST (NCT04971226), a multicenter, randomized, active-controlled, open-label trial. A total of 405 patients were randomized (1:1) to receive either asciminib or investigator-selected tyrosine kinase inhibitors (IS-TKIs) (imatinib, nilotinib, dasatinib, or bosutinib). The main efficacy outcome measure was major molecular response (MMR) rate at 48 weeks. The MMR rate at 48 weeks was 68% (95% CI: 61, 74) in the asciminib arm and 49% (95% CI: 42, 56) in the IS-TKIs arm (difference 19% [95% CI: 10, 28], p-value <0.001). Within the imatinib stratum, the MMR rate was 69% (95% CI: 59, 78) in the asciminib arm and 40% (95% CI: 31, 50) in the IS-TKIs arm (difference 30% [95% CI: 17, 42], p-value <0.001). In the pooled safety population in patients with newly diagnosed and previously treated Ph+ CML in CP, the most common adverse reactions (≥20%) were musculoskeletal pain, rash, fatigue, upper respiratory tract infection, headache, abdominal pain, and diarrhea. The most common laboratory abnormalities (≥40%) in patients with newly diagnosed Ph+ CML in CP were decreased lymphocyte count, decreased leukocyte count, decreased platelet count, decreased neutrophil count, and decreased calcium corrected. #fda #oncology #leukemia #drugapproval #asciminib https://lnkd.in/g9CrBaC3

📢 FDA approves amivantamab in first-line EGFR exon 20 insertion-mutated non-small cell lung cancer indications; converts to traditional approval (The Janssen Pharmaceutical Companies of Johnson & Johnson) ➤ Amivantamab + carboplatin +pemetrexed showed a statistically significant improvement in PFS vs. carboplatin + pemetrexed with a HR of 0.40 (95% CI: 0.30, 0.53; p-value<0.0001). Median PFS was 11.4 months (95% CI: 9.8, 13.7) and 6.7 months (95% CI: 5.6, 7.3) in the respective arms ➤ FDA action converts the May 2021 2nd line accelerated approval to a full approval based on the confirmatory Phase 3 PAPILLON study #oncology #biotech #biotechnology #regulatoryaffairs #pharma #drugdevelopment #oncologytrials #oncologyresearch #cancer #oncology #regulatoryintelligence #regulatoryprecedent #nsclc https://lnkd.in/estqwicD

#Astellas has achieved #approval in #Japan for its #gastriccancer drug #Vyloy (zolbetuximab), targeting #Claudin18.2 (#CLDN18.2) on the surface of gastric cancer cells, despite facing rejection from the FDA due to manufacturing issues. This marks the first worldwide approval for a therapy targeting CLDN18.2-positive gastric cancer, presenting a novel treatment option for this patient group. The drug had previously been granted #priorityreview in the US for CLDN18.2-positive patients with specific types of gastric cancer, but faced delays due to concerns from the FDA regarding a third-party manufacturing facility. Astellas is actively working with the FDA and the involved manufacturer to resolve these issues, although no specific timeline for resolution or reapplication for approval in the US has been provided. Key Highlights: Innovative Treatment Option: Vyloy is the first drug worldwide to offer a targeted therapy for CLDN18.2-positive gastric cancer, representing a significant advancement in the treatment landscape for this disease Regulatory Hurdles: Despite its innovative approach, Vyloy faced regulatory challenges in the US due to manufacturing issues, highlighting the importance of quality control and regulatory compliance in drug development Global Regulatory Dynamics: The approval in Japan demonstrates the variability in regulatory outcomes across different geographies, underlining the strategic importance of navigating global regulatory landscapes Commitment to Resolution: Astellas's engagement with the FDA and its commitment to addressing the manufacturing concerns signify the company's dedication to making this treatment available to a broader patient population https://lnkd.in/gphbNMTn

Exciting progress in multiple myeloma treatment! 🌟 The GMMG-HD7 phase 3 study has shown that adding isatuximab (Sarclisa; Sanofi) to the standard-of-care regimen of lenalidomide (Revlimid; Bristol Myers Squibb), bortezomib (Velcade; Millennium Pharmaceuticals), and dexamethasone (Decadron; Pfizer) (RVd) significantly improves progression-free survival (PFS) for patients with newly diagnosed multiple myeloma (MM). This promising combination not only enhances the effectiveness of treatment but also marks a significant advancement in the evolving landscape of MM therapies. With MM being the second most common hematological malignancy, these results highlight the need for continuous innovation in treatment approaches. The integration of isatuximab could provide a new standard in frontline care, offering hope for better outcomes and extended survival for patients. #MultipleMyeloma #Isatuximab #CancerResearch #ClinicalTrials #Oncology #MedicalAdvances https://lnkd.in/ecRNwJPV

Astellas Pharma receives NMPA approval in China for #PADCEV to Treat #AdvancedUrothelialCancer. #PADCEV (enfortumab vedotin) gains regulatory #Approval in #China, providing new hope for patients with locally advanced or #MetastaticUrothelialCancer who have previously undergone #Chemotherapy and PD-1/PD-L1 inhibitor treatments. Astellas Pharma, led by President and CEO Naoki Okamura, announced that China's NMPA-National Medical Products Administration (NMPA) through its #CenterforDrugEvaluation (CDE), has approved PADCEV (enfortumab vedotin) for the treatment of adult patients with locally advanced or metastatic urothelial cancer (la/mUC) who have previously received platinum-based chemotherapy and PD-1 or PD-L1 inhibitors. Urothelial cancer is a severe and frequently aggressive disease impacting both the lower and the upper urinary tract. In 2022, over 92,000 individuals were diagnosed with #BladderCancer in China, with approximately 41,000 deaths attributed to the condition. Survival rates are notably low for those with locally advanced or metastatic urothelial cancer, underscoring the critical need for new therapies that can prolong patients' lives. This approval, based on data from a global Phase 3 registration study and a bridging study in Chinese patients, marks a significant milestone, enabling access to this new #AntibodyDrugConjugate (ADC) treatment in China. #Astellas #Oncology #Cancer #UrothelialCancer #ADC #PD1 #PDL1 #Padcev #EnfortumabVedotin #China #NMPA #CDE #Approval #RASLifeScienceSolutions #MarketIntelligence #MarketResearch #CompetitiveIntelligence #StrategySupport #PortfolioOptimization #MarketExpansion #PartnerIdentification For detailed news: https://lnkd.in/d7bqJ_bF Follow RASLSS for more updates: https://lnkd.in/de5zNWmK

Anemia's Dosing in Phase II Study of RVU120 in Lower-Risk Myelodysplastic Syndromes The REMARK Phase II trial, led by Ryvu Therapeutics in collaboration with the European Myelodysplastic Neoplasms Cooperative Group and overseen by hematologist Prof. Uwe Platzbecker, aims to evaluate the safety and efficacy of RVU120, a cyclin-dependent kinase 8/19 inhibitor, in treating anemia in lower-risk myelodysplastic syndromes patients. Conducted across 25 global sites, including countries like Germany, Spain, Poland, Italy, and France, the trial expects to enroll around 40 patients. RVU120 has shown promise in improving hematologic outcomes and reducing red blood cell transfusion dependence in previous trials involving high-risk MDS and acute myeloid leukemia. If successful, the drug could offer a new treatment option for LR-MDS patients and possibly expand its applications in other hematologic conditions. For more details please click the link! https://lnkd.in/dNhZQEUc #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical 

📢 FDA approves osimertinib for locally advanced, unresectable (stage III) NSCLC following chemoradiation therapy ➤ Tagrisso (osimertinib, AstraZeneca), for adult patients with locally advanced, unresectable (stage III) NSCLC whose disease has not progressed during or following concurrent or sequential platinum-based chemoradiation therapy and whose tumors have EGFR exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test ➤ Efficacy was evaluated in LAURA (NCT03521154), a double blind, randomized, placebo-controlled trial in 216 adult patients ➤ Osimertinib demonstrated a statistically significant improvement in PFS vs. placebo with a hazard ratio of 0.16 (95% CI: 0.10, 0.24; p-value <0.001). The median PFS was 39.1 months (95% CI: 31.5, not estimable [NE]) in the osimertinib arm and 5.6 months (95% CI: 3.7, 7.4) in the placebo arm ➤ While OS results were immature at the current analysis, with 36% of pre-specified deaths for the final analysis reported, no trend towards a detriment was observed #oncology #biotech #biotechnology #regulatoryaffairs #pharma #drugdevelopment #oncologytrials #oncologyresearch #cancer #oncology #regulatoryintelligence #regulatoryprecedent https://lnkd.in/eECxiacc

#News: The FDA on Tuesday expanded the label of Takeda ’s #Iclusig (#ponatinib). The #kinaseinhibitor can now be used with #chemotherapy to treat adult patients with Philadelphia chromosome-positive acute lymphoblastic leukemia in the first-line setting. The #labelexpansion was granted under the #FDA’s #acceleratedpathway based on Iclusig’s ability to induce minimal residual disease (MRD)-negative complete remission (CR). #Takeda will need to run confirmatory studies to verify the clinical benefit of Iclusig in this indication to ensure continued approval. Awny Farajallah, MD, FACP , CMO of oncology at Takeda, in a statement called Tuesday’s label expansion an “incredibly exciting milestone” as it will provide newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) patients with “an approved, targeted treatment option in the frontline.” Read more from BioSpace 👇🏽 https://lnkd.in/ebZkhXtT