Capricor Therapeutics Reports the Topline P-III (HOPE-3) Trial Data on Deramiocel for Duchenne Muscular Dystrophy

Shots: Capricor Therapeutics has reported the topline P-III (HOPE-3) trial data assessing Deramiocel (IV, Q3M) vs PBO in boys & young men (n=106) with DMD for 12mos. across the US Trial met its 1EP with a 54% slowing of upper-limb functional decline (PUL v2.0) among 105 evaluable pts at 12mos. & achieved its 2EP with…

ByRidhi RastogiDecember 4, 2025

NEWS

Eli Lilly’s Jaypirca Receives the FDA Approval for R/R Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma (CLL/SLL)

Shots: The US FDA has approved label expansion of Jaypirca (pirtobrutinib; 100 & 50mg) for the treatment of adults with r/r CLL/SLL who were previously treated with a covalent BTK inhibitor & converted the accelerated approval of Dec 2023 to a full approval Label expansion was based on the P-III (BRUIN CLL-321) study assessing Jaypirca…

ByRidhi RastogiDecember 4, 2025

NEWS

Formosa Pharmaceuticals Collaborates with Rxilient to Commercialize APP13007 Across ASEAN Markets

Shots: Formosa has entered into an exclusive licensing agreement with Rxilient to commercialize APP13007, a 0.05% clobetasol propionate ophthalmic suspension for the treatment of inflammation & pain after ocular surgery As per the deal, Rxilient will gain exclusive rights to commercialize the suspension across key ASEAN markets, incl. Indonesia, Philippines, Singapore, & Thailand, with the deal…

ByRidhi RastogiDecember 3, 2025

NEWS

Imvax Reports P-IIb Trial Findings on IGV-001 to Treat Newly Diagnosed Glioblastoma

Shots: Imvax has reported the topline data from the P-IIb trial assessing IGV-001 vs PBO in 99 pts with newly diagnosed glioblastoma (ndGBM) Trial showed an mOS of 20.3 vs 14mos., with an mFU of 22mos. for all pts, plus IGV-001 demonstrated measurable patient benefit across various metrics; safety was consistent with previous studies Additionally,…

ByRidhi RastogiDecember 3, 2025

NEWS

Ionis’ Zilganersen Receives the US FDA’s Breakthrough Therapy Designation for Alexander Disease

Shots: The US FDA has granted BTD to zilganersen for the treatment of Alexander disease (AxD) Designation was supported by the topline P-III trial data, which assessed zilganersen (25 or 50mg) vs control in 54 AxD pts (1.5-53yrs.). Zilganersen (50mg) met the 1EP of gait speed as assessed by 10-Meter Walk Test at 61wks., plus…

ByRidhi RastogiDecember 3, 2025

NEWS

Francis Medical’s Vanquish Water Vapor System Receives the US FDA 510(k) Clearance for Prostate Tissue Ablation

Shots: FDA has granted 510(k) clearance to Vanquish Water Vapor System for prostate tissue ablation in pts with intermediate-risk, localized prostate cancer Clearance was supported by 12mos. follow-up data on 110 pts first enrolled in the ongoing VAPOR 2 trial (n=235) assessing Vanquish System across the US, with longer-term to support PMA submission 6mos. biopsy…

ByRidhi RastogiDecember 3, 2025

NEWS

The US FDA Approves Additional Presentation of Celltrion’s Omlyclo (Biosimilar, Xolair)

Shots: The US FDA has approved Omlyclo, an interchangeable biosimilar version of Xolair (omalizumab), in PFS presentation (300mg/2ml, SC) for the treatment of pts with allergic asthma, chronic spontaneous urticaria (CSU), chronic rhinosinusitis with nasal polyps (CRSwNP), & IgE-mediated food allergy Omlyclo previously received the US FDA approval in Mar 2025 for PFS injections at…

ByRidhi RastogiDecember 3, 2025

NEWS

AstraZeneca Reports the US FDA’s NDA Acceptance with Priority Review of Baxdrostat for Hard-to-Control Hypertension

Shots: The US FDA has accepted NDA & granted priority review to Baxdrostat as an add-on treatment of pts with uncontrolled or treatment-resistant hypertension (PDUFA: Q2’26 following use of PRV) NDA was supported by the P-III (BaxHTN) trial assessing baxdrostat (1 or 2mg, QD, PO) vs PBO on top of SoCs in 796 pts with…

ByRidhi RastogiDecember 3, 2025

NEWS

Roche’s Point-of-Care PCR Test Gains FDA’s 510(k) Clearance with CLIA Waiver and European CE Mark to Diagnose Bordetella infections

Shots: Roche has received 510(k) clearance with CLIA waiver & CE-IVDR certification for its point-of-care test to diagnose whooping cough (pertussis) & other Bordetella infections The PCR test on the cobas liat system delivers results in 15min. in GP clinics & emergency rooms, allowing real-time diagnosis during consultation & supporting immediate antibiotic decisions to help…

ByRidhi RastogiDecember 2, 2025

NEWS

Belite Bio Reports Topline P-III (DRAGON) Trial Data on Tinlarebant for Stargardt Disease Type 1 (STGD1)

Shots: Trial assessed tinlarebant (5mg, QD, PO; n=69) vs PBO (n=35) in 104 STGD1 pts (12-20yrs.), meeting its 1EP of 36% reduction in lesion growth per retinal imaging, quantified as 35.7% using the pre-specified MMRM model & confirmed at 35.4% with a post-hoc autoregressive-MMRM analysis Trial also showed reduced lesion growth rate by 33.6% in fellow…

ByRidhi RastogiDecember 2, 2025

Capricor Therapeutics Reports the Topline P-III (HOPE-3) Trial Data on Deramiocel for Duchenne Muscular Dystrophy

Eli Lilly’s Jaypirca Receives the FDA Approval for R/R Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma (CLL/SLL)

Formosa Pharmaceuticals Collaborates with Rxilient to Commercialize APP13007 Across ASEAN Markets

Imvax Reports P-IIb Trial Findings on IGV-001 to Treat Newly Diagnosed Glioblastoma

Ionis’ Zilganersen Receives the US FDA’s Breakthrough Therapy Designation for Alexander Disease

Francis Medical’s Vanquish Water Vapor System Receives the US FDA 510(k) Clearance for Prostate Tissue Ablation

The US FDA Approves Additional Presentation of Celltrion’s Omlyclo (Biosimilar, Xolair)

AstraZeneca Reports the US FDA’s NDA Acceptance with Priority Review of Baxdrostat for Hard-to-Control Hypertension

Roche’s Point-of-Care PCR Test Gains FDA’s 510(k) Clearance with CLIA Waiver and European CE Mark to Diagnose Bordetella infections

Belite Bio Reports Topline P-III (DRAGON) Trial Data on Tinlarebant for Stargardt Disease Type 1 (STGD1)

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