Pharming Group Launches Joenja in Japan for APDS

Shots: Pharming Group has launched Joenja in Japan for activated PI3K delta syndrome (APDS) patients aged 4 years and older following NHI reimbursement listing As per the agreement with Pharming, OrphanPacific serves as Joenja’s Marketing Authorization Holder in Japan &, alongside Pharming, manages the product’s supply & distribution Leniolisib is under regulatory review for pediatric…

ByRidhi Rastogi15 hours ago

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Definium Therapeutics Reports P-III (Voyage) Study on DT120 ODT in Generalized Anxiety Disorder (GAD)

Shots: Definium has reported topline P-III (Voyage) trial results assessing DT120 (lysergide, Orally Disintegrating Tablet, 100µg; n=107) vs PBO (n=107) in 214 adults with GAD Voyage met its 1EP, with DT120 improving HAM-A scores at Wk. 12 (-11.6 vs -6.2). Efficacy emerged by Day 2 & was sustained through all post-baseline timepoints in Part A Key…

ByRidhi Rastogi16 hours ago

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Freenome’s SimpleScreen Lung Cancer Screening Test Secures Breakthrough Device Designation

Shots: The US FDA has granted Breakthrough Device Designation to SimpleScreen Lung to detect early signals of lung cancer in a blood sample from a standard blood draw SimpleScreen Lung builds on its base-level methylation & next-generation sequencing platform, combining methylation & proteomics with an AI/ML-powered classifier to detect lung cancer signals Freenome presented initial…

ByRidhi Rastogi17 hours ago

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Summit and Akeso Reports NMPA Approval for Ivonescimab + CT for 1L Squamous NSCLC

Shots: Chinese NMPA has approved ivonescimab + CT for the 1L treatment of pts with advanced squamous NSCLC (sqNSCLC), based on the global P-III (HARMONi-6) trial The trial, sponsored by Akeso, assessed ivonescimab + CT vs Tevimbra + CT to treat Chinese pts with 1L sqNSCLC irrespective of PD-L1 expression, showing improved OS & PFS;…

ByRidhi Rastogi19 hours ago

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Taiho Oncology, Taiho Pharmaceutical and Cullinan Report P-III (REZILIENT3) Trial Data on Zipalertinib for 1L EGFRm NSCLC

Shots: The companies have reported the global P-III (REZILIENT3) trial assessing zipalertinib + Pt-based CT vs CT alone in the 1L treatment of 285 adults with previously untreated, locally advanced or metastatic NSCLC harboring EGFR ex20ins mutations Trial met its 1EP of PFS, showing improvement with the zipalertinib-containing regimen at interim analysis, with the IDMC recommending unblinding the…

ByRidhi Rastogi20 hours ago

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PTC Therapeutics to Acquire Sangamo Therapeutics’ ST-920 Gene Therapy Program

Shots: PTC Therapeutics has been selected as the winning bidder to acquire ST-920, a BLA-stage, one-time AAV gene therapy for Fabry disease, from Sangamo Therapeutics through a competitive bankruptcy auction As per the terms, PTC Therapeutics will pay $111M upfront, with up to $100M in contingent milestone payments tied to certain regulatory approvals; expected to…

ByRidhi Rastogi21 hours ago

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Elevation Spine Receives FDA Clearance for Saber-XA ALIF System

Shots: The US FDA has granted 510(k) clearance to Saber-XA, an anterior lumbar interbody fusion (ALIF) system combining an expandable cage with a zero-profile anterior lumbar plate, enabling intra-operative customization of implant height & lordosis Saber-XA allows surgeons to independently adjust implant height & lordotic angle based on patient anatomy, with multiple configurations across its…

ByRidhi Rastogi2 days ago

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Rhythm Pharmaceuticals’ Imcivree Receives Expanded UK Authorization for Hypothalamic Obesity

Shots: The MHRA has expanded market authorization for Imcivree (setmelanotide) to incl. the treatment of obesity & control of hunger in pts (≥4yrs.) with acquired hypothalamic obesity due to hypothalamic injury or impairment Approval was backed by the global P-II/III (TRANSCEND) trial assessing Imcivree vs PBO in 120 pts with acquired HO, meeting its 1EP with…

ByRidhi Rastogi2 days ago

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Belenos Biosciences Reports P-II Trial Results on BEL512 for CRSwNP

Shots: P-II trial, conducted by Belenos’partner Keymed Biosciences, assessed BEL512 (SC; 300mg, Q12W/Q24W or 600mg, Q24W) vs PBO, both on top of intranasal corticosteroid (QD), in 120 Chinese pts with CRSwNP with NPS of ≥5 (≥2 polyps/nostril) & a NCS of 2-3 Trial met its 1 & 2EPs, with the BEL512 showing statistically significant improvements…

ByRidhi Rastogi2 days ago

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Belite Bio Receives the US FDA Priority Review for Tinlarebant to Treat Stargardt Disease Type 1

Shots: The US FDA has accepted & granted Priority Review to the NDA for tinlarebant for the treatment of Stargardt Disease Type 1 (STGD1), with a PDUFA target date of Feb 12, 2027 NDA was supported by the P-III (DRAGON) trial assessing tinlarebant vs PBO in STGD1 pts, showing 35.7% reduction in atrophic retinal lesion growth…

ByRidhi Rastogi2 days ago

Ridhi Rastogi

Pharming Group Launches Joenja in Japan for APDS

Definium Therapeutics Reports P-III (Voyage) Study on DT120 ODT in Generalized Anxiety Disorder (GAD)

Freenome’s SimpleScreen Lung Cancer Screening Test Secures Breakthrough Device Designation

Summit and Akeso Reports NMPA Approval for Ivonescimab + CT for 1L Squamous NSCLC

Taiho Oncology, Taiho Pharmaceutical and Cullinan Report P-III (REZILIENT3) Trial Data on Zipalertinib for 1L EGFRm NSCLC

PTC Therapeutics to Acquire Sangamo Therapeutics’ ST-920 Gene Therapy Program

Elevation Spine Receives FDA Clearance for Saber-XA ALIF System

Rhythm Pharmaceuticals’ Imcivree Receives Expanded UK Authorization for Hypothalamic Obesity

Belenos Biosciences Reports P-II Trial Results on BEL512 for CRSwNP

Belite Bio Receives the US FDA Priority Review for Tinlarebant to Treat Stargardt Disease Type 1

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