Ultragenyx Receives FDA Accelerated Approval for GENGLYCOS Gene Therapy to Treat the Underlying Cause of GSDIa

Shots: Ultragenyx reported the US FDA accelerated approval to GENGLYCOS (pariglasgene brecaparvovec-opnr; DTX401) for adults and pediatric patients (≥8yrs., n=46) with glycogen storage disease type Ia (GSDIa) The approval was supported by the P-III (GlucoGene) study, in which GENGLYCOS (n=20) demonstrated a statistically significant reduction in cornstarch requirements at 48wks. vs PBO (n=24) (p<0.001). The…

ByPrince Giri6 hours ago

NEWS

NORROY BIOSCIENCE Receives FDA Breakthrough Therapy and Fast Track Designations for 68Ga-NYM096 in Kidney Cancer Imaging

Shots: NORROY BIOSCIENCE reported that the FDA has granted Breakthrough Therapy and FTD to 68Ga-NYM096, a radiopharmaceutical imaging agent targeting CAIX for PET imaging of clear cell renal cell carcinoma (ccRCC) 68Ga-NYM096 is being developed as a non-invasive PET imaging agent to characterize indeterminate renal masses identified by CT or MRI and help differentiate ccRCC…

ByPrince Giri7 hours ago

NEWS

Harbour BioMed Reports NMPA IND Approval for HBM7004 to Treat Solid Tumors

Shots: The NMPA has approved the IND application for HBM7004 for the treatment of advanced solid tumors HBM7004 is a B7H4xCD3 bispecific antibody developed using HBM’s HBICE platform, designed to offer a differentiated cancer immunotherapy approach with potential efficacy & safety benefits In preclinical studies, HBM7004 induced B7H4-dependent intratumoral T-cell activation & demonstrated strong anti-tumor…

ByRidhi Rastogi2 days ago

NEWS

AbelZeta Gains FDA IND Clearance for Bispecific CAR-T C-CAR039 in LBCL

Shots: AbelZeta has received FDA clearance for the IND application for C-CAR039 (prizloncabtagene autoleucel), an anti-CD20/CD19 bispecific CAR-T therapy, for r/r large B-cell lymphoma (LBCL) AbelZeta regained global development, regulatory, manufacturing, & commercialization rights to C-CAR039 in Jul 2026 & is working with the FDA to finalize clinical protocols for 3L or later LBCL pts…

ByRidhi Rastogi2 days ago

NEWS

UroGen Submits NDA for UGN-103 to the US FDA for Recurrent LG-IR-NMIBC

Shots: The US FDA has received an NDA for UGN-103 (mitomycin; intravesical) to treat adults with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) The NDA is supported by the P-III (UTOPIA) trial in adults with recurrent LG-IR-NMIBC, where UGN-103 achieved a 77.8% 3mos. CR rate & a 94.5% 6mos. DoR by Kaplan-Meier estimate, consistent…

ByRidhi Rastogi2 days ago

NEWS

Lantheus Reports the US FDA Approval of Tauklarify as a PET Imaging Agent for Alzheimer’s Disease

Shots: The US FDA has approved Tauklarify (florquinitau F 18 injection/ MK-6240) for PET of the brain in adults with cognitive impairment who are being evaluated for Alzheimer’s disease to identify pts with tau neurofibrillary tangle (NFT) pathology Approval was supported by Study 1 & 2 assessing Tauklarify (IV, 185 MBq) PET images from >500…

ByRidhi Rastogi3 days ago

NEWS

Pfizer and Valneva Report EMA’s MAA Validation for PF-07307405 Against Lyme Disease

Shots: The EMA has validated the MAA for PF-07307405, a 6-valent outer surface protein A (OspA)-based Lyme disease vaccine candidate and will begin its assessment of the application developed in collaboration between Pfizer & Valneva The application is supported by >70% efficacy in the P-III (VALOR) trial, which evaluated PF-07307405 in individuals aged ≥5 years.…

ByRidhi RastogiAugust 14, 2026

NEWS

BMS Reports the US FDA Accelerated Approval for Zenbexus (Iberdomide) Combination in R/R Multiple Myeloma (MM)

Shots: FDA has granted accelerated approval to Zenbexus (iberdomide) + daratumumab & hyaluronidase-fihj + dexamethasone (ZDd) for the treatment of pts with MM who received ≥1L of prior therapy incl. a proteasome inhibitor & an immunomodulatory agent Approval was supported by the P-III (EXCALIBER-RRMM) trial assessing ZDd (n=207) vs daratumumab + bortezomib + dexamethasone (DVd;…

ByRidhi RastogiAugust 14, 2026

NEWS

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 RastogiAugust 13, 2026

NEWS

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 RastogiAugust 12, 2026

Ultragenyx Receives FDA Accelerated Approval for GENGLYCOS Gene Therapy to Treat the Underlying Cause of GSDIa

NORROY BIOSCIENCE Receives FDA Breakthrough Therapy and Fast Track Designations for 68Ga-NYM096 in Kidney Cancer Imaging

Harbour BioMed Reports NMPA IND Approval for HBM7004 to Treat Solid Tumors

AbelZeta Gains FDA IND Clearance for Bispecific CAR-T C-CAR039 in LBCL

UroGen Submits NDA for UGN-103 to the US FDA for Recurrent LG-IR-NMIBC

Lantheus Reports the US FDA Approval of Tauklarify as a PET Imaging Agent for Alzheimer’s Disease

Pfizer and Valneva Report EMA’s MAA Validation for PF-07307405 Against Lyme Disease

BMS Reports the US FDA Accelerated Approval for Zenbexus (Iberdomide) Combination in R/R Multiple Myeloma (MM)

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

Rhythm Pharmaceuticals’ Imcivree Receives Expanded UK Authorization for Hypothalamic Obesity

Contact US

FOLLOW US