X4 Pharmaceuticals’ Xolremdi Receives the EC’s Approval for WHIM Syndrome

Shots: The EC has approved Xolremdi (Mavorixafor), under exceptional circumstances, for the treatment of WHIM syndrome Approval was supported by the global pivotal P-III (4WHIM) trial, PBO-controlled 52-wk study evaluating mavorixafor’s efficacy & safety in pts ≥12 yrs with WHIM syndrome Xolremdi (PO, QD), a CXCR4 antagonist, is used in pts ≥12 yrs with WHIM syndrome to increase circulating mature neutrophils & lymphocytes Ref: X4…

ByDipanshu DixitApril 27, 2026

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Crinetics Pharmaceuticals Report the EC Approval of Palsonify for Acromegaly

Shots: The EC has approved Palsonify (paltusotine) for treating adults with acromegaly who are ineligible for or inadequately respond to surgery across all 30 EEA states EC approval was supported by P-III PATHFNDR-1 & PATHFNDR-2 trials evaluating Palsonify in previously treated & medically untreated adults with acromegaly, demonstrating rapid onset, reliable biochemical control & sustained efficacy; it also received ODD in EU Crinetics plans…

ByDipanshu DixitApril 23, 2026

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Telix Reports US FDA Acceptance of NDA for TLX101-Px (Pixclara) in Glioma Imaging

Shots: The US FDA has accepted the resubmitted NDA for TLX101-Px (Pixclara; 18F-FET), an investigational PET imaging agent for the detection and characterization of glioma in adult and pediatric patients The US FDA has assigned a PDUFA date of 11’Sep, 2026; TLX101-Px has received Orphan Drug and Fast Track designations, addressing unmet needs in distinguishing…

ByPrince GiriApril 10, 2026

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Santhera Pharmaceuticals Reports Swissmedic’s Approval of Agamree (Vamorolone) to Treat Duchenne Muscular Dystrophy

Shots: Swissmedic has approved Agamree for the treatment of pts (≥4yrs) with Duchenne muscular dystrophy and expects launch in H2’26 Approval was based on pivotal P-IIb (VISION-DMD) data and EMA assessments, granting 15-year Swiss orphan drug exclusivity; Santhera retains exclusive Swiss distribution. Trial met its 1EP of Time to Stand (TTSTAND) velocity vs PBO at…

ByDipanshu DixitJanuary 15, 2026

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Omeros Submits MAA to the EMA for Narsoplimab (OMS721) to Treat TA-TMA

S Shots: Omeros submitted an MAA to the EMA for narsoplimab to treat TA-TMA, with data demonstrating a 61% response rate and a 3x improvement in OS vs matched external controls; over 130 pts treated under expanded access data were also included EMA granted Narsoplimab ODD status, and CHMP review will start mid-July under centralized…

ByPrince GiriJune 30, 2025

X4 Pharmaceuticals’ Xolremdi Receives the EC’s Approval for WHIM Syndrome

Crinetics Pharmaceuticals Report the EC Approval of Palsonify for Acromegaly

Telix Reports US FDA Acceptance of NDA for TLX101-Px (Pixclara) in Glioma Imaging

Santhera Pharmaceuticals Reports Swissmedic’s Approval of Agamree (Vamorolone) to Treat Duchenne Muscular Dystrophy

Omeros Submits MAA to the EMA for Narsoplimab (OMS721) to Treat TA-TMA

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