Sentynl Therapeutics Reports US FDA Approval of Zycubo to Treat Pediatric Menkes Disease

Shots: The US FDA has approved Zycubo (copper histidinate) for the treatment of pediatric patients with Menkes disease Approval was supported by clinical data showing improved OS with early Zycubo treatment vs an untreated external control, reflecting ~80% reduction in the risk of death & a mOS of 177.1 vs 17.6mos. Zycubo is a bioavailable…

ByRidhi RastogiJanuary 14, 2026

NEWS

Summit Therapeutics Reports the US FDA’s BLA Submission for Ivonescimab to Treat EGFRm Non-Squamous NSCLC

Shots: The US FDA has received BLA of ivonescimab + CT for ≥2L treatment of pts with EGFR-mutated locally advanced or metastatic non-squamous NSCLC; decision is expected by Q4’26 BLA was supported by P-III (HARMONi) trial assessing ivonescimab + Pt-doublet CT vs PBO + Pt-doublet CT in pts with EGFR-mutated, locally advanced or metastatic NSCLC…

ByRidhi RastogiJanuary 13, 2026

NEWS

Merck KGaA Reports US FDA’s NDA Acceptance of Pimicotinib for Tenosynovial Giant Cell Tumor (TGCT)

Shots: The US FDA has accepted the NDA of Merck KGaA's Pimicotinib (PO) for the systemic treatment of adults with TGCT following NMPA approval NDA was based on the global 3-part P-III (MANEUVER) trial in TGCT pts who require systemic therapy & have not received prior anti-CSF-1/CSF-1R therapy, in which Part 1 randomized 94 pts…

ByRidhi RastogiJanuary 13, 2026

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Sanofi’s Teizeild (Teplizumab) Receives the EC Approval to Delay Progression of Type 1 Diabetes

Shots: The EC has approved Sanofi's Teizeild (Tzield outside the EU) to delay the onset of stage 3 type 1 diabetes (T1D) in adult & pediatric pts (≥8yrs.) with stage 2 T1D; regulatory review is ongoing in the other regions Approval was based on the P-II (TN-10) trial assessing Teizeild (QD, n=44) vs PBO (n=32)…

ByRidhi RastogiJanuary 12, 2026

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Annexon Seeks European Approval of Tanruprubart for Guillain-Barré Syndrome

Shots: The EMA has received MAA of tanruprubart for the treatment of Guillain-Barré syndrome (GBS) The MAA dossier demonstrated rapid neuroinflammation control & improved recovery, supported by a PoC study & a P-III trial against PBO, favorable risk/benefit, robust US & Southeast Asian biomarker data, PK (US/EU/SE Asia), & RWE matching P-III outcomes to IVIg…

ByRidhi RastogiJanuary 9, 2026

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Fondazione Telethon’s Waskyra Receives the EC Approval for Wiskott-Aldrich Syndrome

Shots: The EC has approved Waskyra (etuvetidigene autotemcel), an ex vivo gene therapy for Wiskott-Aldrich Syndrome (WAS) Waskyra is a single-administration therapy that uses a patient’s CD34+ hematopoietic stem and progenitor cells, modified with a lentiviral vector carrying the WAS gene The EC’s decision follows a CHMP opinion issued in November 2025 recommending marketing authorization, with the same therapy also approved by…

ByDipanshu DixitJanuary 9, 2026

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BioNet’s VacPertagen Receives the EC Approval for Pertussis Disease

Shots: The EC has approved VacPertagen as a booster aganist Pertussis for pts (age≥12) and for passive infant protection via maternal immunization during pregnancy CHMP recommendation was supported by data from three clinical trials and nine supportive studies, all consistently demonstrating that VacPertagen is safe for use in teenagers, adults, and pregnant women across populations VacPertagen is an acellular pertussis vaccine with two components, including recombinant…

ByDipanshu DixitJanuary 9, 2026

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Arrowhead Pharmaceuticals Receives the NMPA Approval for Redemplo (Plozasiran) to Treat Familial Chylomicronemia Syndrome (FCS)

Shots: The Chinese NMPA has approved Redemplo to reduce triglycerides (TGs) in adults with FCS; regulatory review is ongoing in other regions Redemplo will be commercialized by Sanofi in Greater China under an agreement with Arrowhead, with NMPA approval triggering a $10M milestone payment from Sanofi to Arrowhead’s subsidiary Visirna Therapeutics Redemplo is an siRNA…

ByRidhi RastogiJanuary 8, 2026

NEWS

GSK Reports the MHLW Approval of Exdensur (Depemokimab) for Severe Asthma and CRSwNP

Shots: GSK’s Exdensur was approved for sev. or refractory bronchial asthma unresponsive to existing therapies & for CRSwNP in pts whose disease remains inadequately controlled with SoC, based on the P-III (SWIFT & ANCHOR) trials, respectively; filings under regulatory review in the EU & China SWIFT-1 (n=382) & SWIFT-2 (n=380) trials showed depemokimab reduced asthma…

ByRidhi RastogiJanuary 6, 2026

NEWS

Takeda and Protagonist Therapeutics Report the US FDA’s NDA Submission for Rusfertide to Treat Polycythemia Vera

Shots: The US FDA has received an NDA of rusfertide (SC) for the treatment of adults with polycythemia vera (PV) NDA was followed by 32wk. primary analysis & 52wk. data from the global P-III (VERIFY) trial (n=293) as well as P-II (REVIVE) study, where VERIFY met its 1EP & all key 2EPs, showing higher response…

ByRidhi RastogiJanuary 6, 2026

Sentynl Therapeutics Reports US FDA Approval of Zycubo to Treat Pediatric Menkes Disease

Summit Therapeutics Reports the US FDA’s BLA Submission for Ivonescimab to Treat EGFRm Non-Squamous NSCLC

Merck KGaA Reports US FDA’s NDA Acceptance of Pimicotinib for Tenosynovial Giant Cell Tumor (TGCT)

Sanofi’s Teizeild (Teplizumab) Receives the EC Approval to Delay Progression of Type 1 Diabetes

Annexon Seeks European Approval of Tanruprubart for Guillain-Barré Syndrome

Fondazione Telethon’s Waskyra Receives the EC Approval for Wiskott-Aldrich Syndrome

BioNet’s VacPertagen Receives the EC Approval for Pertussis Disease

Arrowhead Pharmaceuticals Receives the NMPA Approval for Redemplo (Plozasiran) to Treat Familial Chylomicronemia Syndrome (FCS)

GSK Reports the MHLW Approval of Exdensur (Depemokimab) for Severe Asthma and CRSwNP

Takeda and Protagonist Therapeutics Report the US FDA’s NDA Submission for Rusfertide to Treat Polycythemia Vera

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