IntraBio’s Aqneursa Receives the EC Approval for Treating Niemann-Pick Disease Type C (NPC)

Shots: The EC has approved Aqneursa (levacetylleucine), available as 1g granules for oral suspension, with or without miglustat for treating adults & pediatric pts (≥6yrs.; ≥20kg) with NPC Approval was backed by P-III (IB1001-301) crossover study assessing Aqneursa vs PBO in NPC pts, which showed improved neurological symptoms & function after 12wks. as measured by…

ByRidhi Rastogi4 days ago

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Sobi Reports the EC Approval of Aspaveli for C3 Glomerulopathy and Primary IC-MPGN

Shots: The EC approved Aspaveli (pegcetacoplan) for pts (≥12yrs.) with C3 glomerulopathy or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), to be used with a RAS inhibitor unless such therapy is contraindicated or not tolerated Approval was based on P-III (VALIANT) trial assessing Aspaveli(twice weekly) vs PBO in C3G or primary IC-MPGN pts (≥12yrs., n=124) for…

ByRidhi RastogiJanuary 16, 2026

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Sanofi Secures China Approval for Myqorzo and Redemplo in oHCM and FCS

Shots: China’s NMPA has approved Sanofi-licensed Myqorzo (aficamten) for the treatment of obstructive hypertrophic cardiomyopathy (oHCM) and Redemplo (plozasiran) for triglyceride reduction in adults with familial chylomicronemia syndrome (FCS) on dietary control The approval of Myqorzo was based on the pivotal P-III (SEQUOIA-HCM) study in pts with symptomatic oHCM, while Redemplo’s approval was supported by…

ByDipanshu DixitJanuary 15, 2026

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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

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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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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

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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

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

Shots: Health Canada has approved Redemplo as an adjunct to diet to reduce triglycerides (TGs) in adults with FCS who had an inadequate response to standard triglyceride lowering therapies Approval was based on the P-III (PALISADE) trial assessing Redemplo (25mg & 50mg, SC, Q3M) vs PBO in 75 adults with genetically confirmed/clinically diagnosed FCS. Pts…

ByRidhi RastogiJanuary 6, 2026

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GSK Reports the NMPA Approval of Nucala (Mepolizumab) for Chronic Obstructive Pulmonary Disease (COPD)

Shots: The Chinese NMPA has approved Nucala as an add-on maintenance treatment of adults with inadequately controlled COPD characterised by eosinophilic phenotype based on the P-III (MATINEE & METREX) trial; EMA filing is under review MATINEE assessed Nucala (100mg, SC, Q4W) vs PBO in COPD pts (n=804) with type 2 inflammation while METREX evaluated it…

ByRidhi RastogiJanuary 5, 2026

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Vanda Pharmaceuticals Reports the US FDA Approval of Nereus (Tradipitant) to Prevent Vomiting Induced by Motion

Shots: The US FDA has granted Nereus for the prevention of vomiting induced by motion, backed by 3 trials, incl. 2 P-III real-world boat studies (Motion Syros & Motion Serifos) & 1 supporting study in pts with documented motion sickness In Motion Syros (n=365) & Motion Serifos (n=316), vomiting incidence was 18.3–19.5% & 10.4–18.3% vs 44.3%…

ByRidhi RastogiDecember 31, 2025

IntraBio’s Aqneursa Receives the EC Approval for Treating Niemann-Pick Disease Type C (NPC)

Sobi Reports the EC Approval of Aspaveli for C3 Glomerulopathy and Primary IC-MPGN

Sanofi Secures China Approval for Myqorzo and Redemplo in oHCM and FCS

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

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

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

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

GSK Reports the NMPA Approval of Nucala (Mepolizumab) for Chronic Obstructive Pulmonary Disease (COPD)

Vanda Pharmaceuticals Reports the US FDA Approval of Nereus (Tradipitant) to Prevent Vomiting Induced by Motion

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