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Yunnan Province Increases Pediatric Medical Service Prices for Children Under 6
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The Yunnan Provincial Healthcare Security Administration and the Health Commission of Yunnan Province have issued a notification to implement a policy that allows for increased pricing on certain pediatric medical services for children under the age of 6 in public medical institutions, effective from October 10, 2024. This policy aims…
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NHC Releases Updated List of Pediatric Drugs for Research and Development Incentives
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The National Health Commission (NHC) has issued a notification regarding the fifth batch of pediatric drugs encouraged for research and development. The list includes 15 varieties, encompassing systemic anti-infective drugs, respiratory system medications, anti-tumor drugs, and immune modulators. This edition features 25 specifications and 8 dosage forms, and is notable…
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Takeda Prepares to File Dengue Fever Vaccine for US Approval, Expanding Global Regulatory Reach
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Takeda Pharmaceutical, a leading Japanese pharmaceutical company listed on the NYSE (NYSE: TAK), is reportedly preparing to submit its dengue fever vaccine for regulatory approval in the United States, according to the Nikkei Asian Review. At this juncture, specifics regarding the timing of the filing or the anticipated decision date…
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Servier’s Voranigo Receives FDA Nod for IDH Inhibitor as Brain Tumor Treatment
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The U.S. Food and Drug Administration (FDA) has granted first-time approval for Voranigo (vorasidenib), a therapy developed by Servier Pharmaceutical plc. Voranigo, an inhibitor of isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2), has been approved for the treatment of adult and pediatric patients aged 12 years and older with Grade…
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Day One Biopharmaceuticals Expands Pipeline with MabCare Therapeutics Licensing Deal
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Day One Biopharmaceuticals (NASDAQ: DAWN), a California-based biotech company that recently celebrated its first drug approval, has announced an expansion of its pipeline through a licensing agreement with Shanghai-based MabCare Therapeutics. Day One will acquire exclusive development, manufacturing, and commercialization rights to MTX-13, an antibody-drug conjugate (ADC) targeting protein-tyrosine kinase…
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Amgen’s Blincyto Secures New Indication Approval for B-ALL Consolidation Treatment in the US
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Amgen (NASDAQ: AMGN) has announced that its bispecific T-cell engager (BiTE) Blincyto (blinatumomab) has received a new indication approval from the U.S. Food and Drug Administration (FDA) for the U.S. market. The drug is now approved for the treatment of adult and pediatric patients aged one month or older with…
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Pfizer’s Gene Therapy for Duchenne Muscular Dystrophy Misses Late-Stage Trial Endpoints
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Pfizer Inc. (NYSE: PFE) has announced that a late-stage clinical trial for its investigational gene therapy, fordadistrogene movaparvovec, in children aged 4 to 7 with Duchenne muscular dystrophy (DMD) failed to meet its primary endpoint of improving motor function. Additionally, secondary endpoints related to motor function showed no significant improvement…
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AstraZeneca’s Farxiga Receives FDA Approval for Pediatric Type-2 Diabetes Treatment
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The US Food and Drug Administration (FDA) has granted regulatory approval to AstraZeneca (AZ; NASDAQ: AZN) for its SGLT2 inhibitor, Farxiga (dapagliflozin), expanding its use to pediatric patients aged 10 and above with type-2 diabetes (T2D). This approval supplements the drug’s original indication for adult patients. The FDA’s decision was…
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RareStone Group’s Pitolisant Secures NMPA Approval to Treat Narcolepsy in Children and Adolescents
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RareStone Group, a China-based company specializing in rare diseases (formerly known as Citrine Medicine), has secured marketing approval from the National Medical Products Administration (NMPA) for pitolisant, its in-licensed drug candidate. The histamine 3 (H3) receptor antagonist/inverse agonist is now approved for treating excessive daytime sleepiness (EDS) or cataplexy in…
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Eisai’s Fycompa Gains New Indication for Generalized Tonic-Clonic Seizures in China
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Eisai Co Ltd (TYO: 4523), a Japanese pharmaceutical company, has announced that its antiepilepsy drug (AED) Fycompa (perampanel) has received a new indication approval from China’s National Medical Products Administration (NMPA). The drug is now approved for use as an additional treatment for adults and children aged 12 and above…
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Pfizer Pauses Dosing in DMD Gene Therapy Trial Following Participant’s Sudden Death
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This week, Pfizer Inc. (NYSE: PFE) announced the unfortunate sudden death of a participant in a Phase II study for its investigational gene therapy, fordadistrogene movaparvovec, used to treat Duchenne muscular dystrophy (DMD). The patient, aged between 2 and 4 years, had received the treatment in the previous year. In…
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Wanbangde Pharmaceutical’s Huperzine A Earns FDA Rare Pediatric Disease Designation for HIE Treatment
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China’s Wanbangde New Building Materials Co., Ltd (SHE: 002082) has announced that its subsidiary, Wanbangde Pharmaceutical Group, has received a rare pediatric disease (RPD) designation from the US Food and Drug Administration (FDA) for its drug huperzine A. This designation is for the treatment of neonatal hypoxic-ischemic encephalopathy (HIE), a…
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Novartis Receives Positive Interim Results for Antimalarial Coartem in Infants
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Swiss pharmaceutical company Novartis (NYSE: NVS) has announced interim results from a Phase II/III clinical trial for its antimalarial drug Coartem (artemether + lumefantrine) in infants with uncomplicated Plasmodium falciparum malaria. The trial data indicates that the tablet formulation meets pharmacokinetic (PK), efficacy, and safety requirements, achieving the primary PK…
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Novartis’s Lutathera Secures FDA Approval for Pediatric Gastroenteropancreatic NETs
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The US Food and Drug Administration (FDA) has granted regulatory approval to Novartis (NYSE: NVS) for its radioligand therapy (RLT) Lutathera (lutetium Lu 177 dotatate) for the treatment of SSTR+ gastroenteropancreatic neuroendocrine tumors (GEP-NETs) in patients aged 12 and older. This expansion of the drug’s original indication for adult patients…
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China’s CDE Issues Guidelines for Pediatric Drug Labeling Updates
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China’s Center for Drug Evaluation (CDE) has issued a notice outlining the “Detailed Rules for Adding Pediatric Medication Information to the Labeling of Listed Drugs.” The notice provides clarity on the procedural steps for updating pediatric labeling information on drugs. Under the new guidelines, originator companies are permitted to directly…
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AstraZeneca’s Fasenra Gains FDA Approval for Pediatric Severe Asthma Treatment
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AstraZeneca (AZ; NASDAQ: AZN) has secured an indication extension from the U.S. Food and Drug Administration (FDA) for its anti-eosinophil biologic Fasenra (benralizumab). The drug is now approved as an add-on maintenance treatment for severe asthma in pediatric patients aged 6 to 11 years with an eosinophilic phenotype, expanding its…
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Shanghai’s 9th People’s Hospital Launches Groundbreaking Gene Therapy Trial for Congenital Deafness
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In a medical first, Shanghai’s 9th People’s Hospital has initiated the world’s inaugural clinical trial for a gene-editing therapy targeting congenital deafness attributed to otoferlin (OTOF) gene mutations. The therapy, developed by HuidaGene Therapeutics Co., Ltd, represents a significant advance in the treatment of auditory neuropathy, a leading cause of…
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GSK’s ViiV Healthcare Expands Dovato Indication to Include Adolescents with HIV-1 Infection
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GlaxoSmithKline’s (GSK; NYSE: GSK) ViiV Healthcare has received an indication extension from the US Food and Drug Administration (FDA) for its antiretroviral treatment Dovato (dolutegravir + lamivudine). The drug is now approved for use in virologically suppressed or treatment-naive adolescents aged 12 years and older with HIV-1 infection, a demographic…
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European Commission Approves Pfizer’s Prevenar 20 for Pediatric Use
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The European Commission (EC) has granted regulatory approval to Pfizer (NYSE: PFE) for its 20-valent pneumococcal vaccine, Prevenar 20, aimed at preventing invasive disease, pneumonia, and acute otitis media caused by Streptococcus pneumoniae in individuals aged 6 weeks to 18 years. This vaccine was previously approved for use in adults…
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Amgen Reports Positive Results for Otezla in Pediatric Psoriasis Trial
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Amgen (NASDAQ: AMGN) provided an update last week on a late-stage trial evaluating its PDE4 inhibitor Otezla (apremilast) for the treatment of moderate to severe plaque psoriasis in patients aged 6 to 17 years. The one-year data revealed that 56.3% of participants achieved clear or almost clear skin, while 71.4%…
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China Aims to Insure 80% of Newborns with Basic Medical Insurance by 2024
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The National Healthcare Security Administration (NHSA) and the National Health Commission (NHC), along with three other bureaus, have issued a Notice on “Carrying out the Special Action for Children to Participate in Basic Medical Insurance (BMI)”. The notice outlines an ambitious target to ensure that 80% of newborns in China…
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Novartis Gene Therapy Zolgensma Shows Continued Benefits in Broader SMA Patient Group
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Swiss pharmaceutical giant Novartis (NYSE: NVS) has presented Phase III data this week, demonstrating that the clinical benefits of its gene therapy Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy (SMA) extend beyond patients up to 2 years of age. The new study has shown similar benefits in older children, including…
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Hainan Poly Pharm Secures Tentative FDA Approval for Generic Version of UCB’s Briviact
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Hainan Poly Pharm Co., Ltd (SHE: 300630), a Chinese pharmaceutical company, has announced that it has received tentative marketing approval from the US Food and Drug Administration (FDA) for its generic version of Belgium-based UCB’s Briviact (brivaracetam). Briviact is recognized as a selective high-affinity synaptic vesicle protein 2A (SV2A) ligand,…
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FDA Approves Takeda’s Eohilia as First Oral Therapy for Eosinophilic Esophagitis in Patients Aged 11 and Up
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The U.S. Food and Drug Administration (FDA) has granted approval to Takeda Pharmaceutical Company (TYO: 4502)’s Eohilia (budesonide oral suspension) for the treatment of eosinophilic esophagitis (EoE). This marks Eohilia as the first and currently only oral therapy available for EoE patients aged 11 and older. Previously, Novartis (NYSE: NVS)’s…
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Dupixent by Sanofi and Regeneron Approved by FDA for Eosinophilic Esophagitis in Younger Pediatric Patients
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The US Food and Drug Administration (FDA) has concluded a priority review and granted approval to Sanofi (NASDAQ: SNY) and Regeneron (NASDAQ: REGN) for their interleukin blocker Dupixent (dupilumab) as a treatment for eosinophilic esophagitis (EoE) in patients aged 1 to 11 years. This indication expansion makes Dupixent the sole…
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Eli Lilly’s Subsidiary Akouos Reports Positive Preliminary Results for Gene Therapy in Hearing Loss
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Eli Lilly (NYSE: LLY) has announced preliminary results from a Phase I/II study conducted by its subsidiary Akouos for gene therapy AK-OTOF, which is being investigated for the treatment of sensorineural hearing loss due to mutations in the otoferlin gene (OTOF). The press release reported that the first participant in…
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Drug Farm’s DF-003 Earns FDA Rare Pediatric Disease Designation for ROSAH Syndrome
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Sino-US biotech company Drug Farm has announced that it has received Rare Pediatric Disease Designation (RPDD) from the US Food and Drug Administration (FDA) for its first-in-class drug candidate DF-003, which is being investigated as a potential treatment for the ROSAH (retinal malnutrition, optic edema, splenomegaly, anhidrosis, and headache) syndrome.…
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China’s NHC Unveils Strategy to Enhance Pediatric Healthcare by 2025
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The National Health Commission (NHC) has issued a set of Opinions aimed at “Promoting the High Quality Development of Children’s Medical and Health Services,” outlining a comprehensive plan to enhance the children’s healthcare system across national, regional, and local levels by 2025. The initiative focuses on improving the allocation of…
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Thederma’s TAP-1503 Cream Receives Priority Review Status for Pediatric Atopic Dermatitis Treatment in China
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The Center for Drug Evaluation (CDE) website has indicated that China-based Thederma’s TAP-1503 cream is poised to receive priority review status for the local topical treatment of atopic dermatitis (AD) in patients aged two years and above. This priority status is granted due to the drug’s classification under “new varieties,…
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EMA’s CHMP Gives Green Light to Sanofi’s Fexinidazole for Acute Sleeping Sickness Treatment
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Last week, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion on Sanofi’s (NASDAQ: SNY) Fexinidazole Winthrop (fexinidazole) as a 10-day treatment for acute sleeping sickness caused by Trypanosoma brucei (T.b.) rhodesiense in both adults and children. The EMA is…
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Vertex Pharmaceuticals and CRISPR Therapeutics Receive Conditional Approval for CRISPR Gene-Editing Therapy
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Vertex Pharmaceuticals (NASDAQ: VRTX) and CRISPR Therapeutics (NASDAQ: CRSP) have received conditional approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) for their jointly developed therapy, Casgevy (exagamglogene autotemcel), a treatment for sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) and for transfusion-dependent beta thalassemia (TDT) in…
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Roche’s Gene Therapy Elevidys Misses Primary Endpoint in DMD Trial
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Switzerland-headquartered pharmaceutical giant Roche (SWX: ROG) has announced that a Phase III trial for its gene transfer therapy Elevidys (delandistrogene moxeparvovec) did not meet its primary endpoint in Duchenne muscular dystrophy (DMD) ambulatory patients aged 4 to 7 years. The trial aimed to measure the change from baseline in motor…
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Pfizer’s Penbraya Receives FDA Approval for Meningococcal Immunization
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Pfizer Inc. (NYSE: PFE) announced last week that it has successfully registered its pentavalent meningococcal vaccine, Penbraya, with the US Food and Drug Administration (FDA). The vaccine is designed for active immunization to prevent invasive disease caused by Neisseria meningitidis serogroups A, B, C, W, and Y in individuals aged…
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Bayer Partners with HALO Precision Diagnostics to Develop Gadoquatrane for MRI Applications
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Germany-based Bayer (ETR: BAYN) has announced a strategic partnership with US radiology expert HALO Precision Diagnostics to jointly develop gadoquatrane, a late-stage contrast agent candidate. This collaboration focuses on advancing three Phase III studies aimed at evaluating the pharmacokinetics, safety, and efficacy of this gadolinium-based molecule in both adults and…
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AstraZeneca Reports Positive Phase III Results for Forxiga in Children with Type 2 Diabetes
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UK-based AstraZeneca (AZ, NASDAQ: AZN) has announced promising results from the Phase III T2NOW study (NCT03199053) for its diabetes medication, Forxiga (dapagliflozin), in adolescents and children aged 10 to 17 with type 2 diabetes (T2D). Enrolling 256 patients, this study stands as one of the largest investigations into type 2…
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Eli Lilly’s Insulin Biosimilar YouBiAn Approved for Diabetes Treatment in China
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US pharmaceutical major Eli Lilly (NYSE: LLY) has announced that it has received market approval from the National Medical Products Administration in China for its insulin glargine product, YouBiAn. This treatment is indicated for adults with type 1 or type 2 diabetes requiring insulin therapy, as well as for children…
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Cutia Therapeutics Files for Market Approval of CU-10201 with NMPA for Acne Treatment
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Cutia Therapeutics (HKG: 2487), a developer focused on dermatology therapies, has announced the submission of a market approval filing for its CU-10201 (4% minocycline foam agent for external use) to the National Medical Products Administration (NMPA) in China. The drug, which targets the treatment of non-nodular moderate to severe acne…
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CDC Provisionally Recommends Pfizer’s Abrysvo for Maternal Immunization Against RSV
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The US Centers for Disease Control and Prevention (CDC) has issued a provisional recommendation for the use of Pfizer’s (NYSE: PFE) bivalent respiratory syncytial virus (RSV) vaccine, Abrysvo, as a seasonal maternal immunization strategy. This measure aims to prevent RSV lower respiratory tract infection in infants, a significant cause of…
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FDA Extends Pfizer’s Comirnaty Vaccine Indication for Omicron XBB.1.5 Variant
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The US Food and Drug Administration (FDA) has granted Pfizer (NYSE: PFE) an indication extension for its SARS-CoV-2 Omicron XBB.1.5 single-dose monovalent vaccine, Comirnaty. The vaccine is now approved for individuals aged 12 years and older, in addition to receiving an emergency use authorization (EUA) for children between 6 months…
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Bayer Initiates US Recall of TRK Inhibitor Vitrakvi Due to Mold Contamination
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Germany-based Bayer (ETR: BAYN) has announced a voluntary recall of a specific lot of its tropomyosin receptor kinase (TRK) inhibitor Vitrakvi (larotrectinib) in the US market following the detection of contamination by the Penicillium brevicompactum mold in the oral solution. While no adverse events have been reported, Bayer has advised…
