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Amicus Therapeutics Secures Japan Approval for Pombiliti + Opfolda Combination Therapy for Late-Onset Pompe Disease

Drug ApprovalRare Disease
  • Japan's Ministry of Health, Labour and Welfare has approved Pombiliti + Opfolda for treating adult patients with late-onset Pompe disease, expanding global access to this innovative therapy.
  • The approval was based on data from the Phase 3 PROPEL study, which uniquely studied both treatment-naïve and treatment-experienced patients in a controlled setting.
  • Pombiliti + Opfolda is now approved in seven major markets including the US, EU, UK, Canada, Australia, Switzerland, and Japan.
  • The two-component therapy combines a recombinant human GAA enzyme with enhanced muscle cell uptake and an oral enzyme stabilizer designed to maintain enzyme activity in blood.

Everest Medicines Advances AI-Powered mRNA Platform with Cancer Vaccine Breakthroughs

Drug ApprovalCAR-TAI
  • Everest Medicines unveiled major breakthroughs in its proprietary AI-powered mRNA platform during its 2025 R&D Day, showcasing a fully integrated system spanning antigen design to manufacturing.
  • The company's personalized cancer vaccine EVM16 demonstrated robust immunogenicity and mutation-specific T-cell responses in early clinical trials, while off-the-shelf vaccine EVM14 received FDA IND clearance.
  • Everest's innovative in vivo CAR-T program achieved high T-cell transduction efficiency and effective B-cell clearance in preclinical studies, offering a scalable alternative to conventional CAR-T therapies.
  • The company is pursuing global partnerships with top 20 pharmaceutical companies to accelerate international deployment of its clinically validated mRNA assets.

Alembic Pharmaceuticals Acquires UTILITY Therapeutics to Commercialize First New UTI Antibiotic in 20 Years

Drug Approval
  • Alembic Pharmaceuticals has acquired UTILITY Therapeutics to expand its U.S. commercial portfolio with PIVYA, the first antibiotic approved for uncomplicated urinary tract infections in approximately 20 years.
  • PIVYA received FDA approval in April 2024 for treating female patients 18 years and older with uUTI caused by specific bacterial strains, featuring a unique mechanism targeting gram-negative bacteria cell walls.
  • The acquisition addresses a critical unmet medical need as current UTI therapies are failing and antibiotic options remain limited for millions of women affected annually.
  • PIVYA is expected to become available in the U.S. market in the fourth quarter of 2025, with positioning as a first-line treatment in infectious disease guidelines.

AstraZeneca and Summit Therapeutics in Advanced Talks for $15 Billion Ivonescimab Licensing Deal

OncologyDrug Approval
  • AstraZeneca is negotiating with Summit Therapeutics for a potential $15 billion licensing deal for ivonescimab, an experimental lung cancer drug that has shown promising results in late-stage trials.
  • The deal could include an upfront payment of several billion dollars plus milestone payments, representing one of the largest pharmaceutical licensing agreements in recent years.
  • Ivonescimab demonstrated statistically significant improvement in progression-free survival when combined with chemotherapy in previously treated lung cancer patients, though it failed to achieve statistical significance for overall survival.
  • Summit secured rights to ivonescimab through a $5 billion deal with China-based Akeso in 2022, and the drug is already approved in China as of May 2023.

Aquestive Therapeutics Receives FDA Approval for Anaphylm New Drug Application, Targeting $2 Billion Severe Allergic Reaction Market

Drug Approval
  • The U.S. Food and Drug Administration has approved Aquestive Therapeutics' New Drug Application for Anaphylm, a sublingual epinephrine product for treating severe allergic reactions.
  • Anaphylm represents a non-device-based alternative to traditional autoinjectors like EpiPen and Auvi-Q, offering wallet portability and rapid onset for emergency anaphylaxis treatment.
  • The FDA has set a Prescription Drug User Fee Act date of January 31, 2026, with the product positioned to capture market share in a $2 billion addressable market.
  • Clinical data demonstrates that Anaphylm achieves outcomes comparable to existing autoinjector products while providing unique sublingual administration advantages over nasal spray formulations.

Delhi High Court Orders CDSCO to Review Safety of GLP-1 Diabetes Drugs Used for Weight Loss

Drug Approval
  • The Delhi High Court has directed India's Central Drugs Standard Control Organisation (CDSCO) to decide within three months on a petition challenging the licensing of GLP-1 receptor agonist drugs for weight loss treatment.
  • The petition raises safety concerns about semaglutide, tirzepatide, and liraglutide being approved for weight management despite limited India-specific clinical trial data and inadequate safety evaluation.
  • These drugs were originally developed for Type 2 diabetes treatment but have been repurposed for obesity management, with the petitioner citing risks including pancreatitis, gastrointestinal damage, and potential cancers.
  • The court emphasized that CDSCO must consult experts and relevant stakeholders before making its decision on the regulatory concerns raised in the petition.

Hikma Receives FDA Approval for TYZAVAN™, First Ready-to-Infuse Vancomycin Formulation for Sepsis Treatment

Drug Approval
  • Hikma Pharmaceuticals received FDA approval for TYZAVAN™, the first ready-to-infuse vancomycin formulation that requires no preparation, targeting critical sepsis treatment where survival rates decrease by 15% after 87-113 minutes.
  • The glycopeptide antibiotic is indicated for septicemia, infective endocarditis, skin infections, bone infections, and lower respiratory tract infections in adults and pediatric patients over one month old.
  • TYZAVAN™ offers room-temperature stability with 16-month shelf life, seven dosing presentations (0.5g-2g), and compatibility with automated dispensing cabinets to streamline hospital workflows.
  • The US vancomycin injection market was valued at approximately $200 million in 2024, with sepsis affecting one person every 20 seconds and causing death every two minutes in the United States.

FDA Approves Takeda's GAMMAGARD LIQUID ERC, First Ready-to-Use Low IgA Immunoglobulin Therapy for Primary Immunodeficiency

Drug Approval
  • The U.S. FDA has approved GAMMAGARD LIQUID ERC as the only ready-to-use liquid immunoglobulin therapy with low IgA content for primary immunodeficiency treatment in patients aged two and older.
  • The therapy contains less than or equal to 2 µg/mL IgA in a 10% solution and can be administered both intravenously and subcutaneously without reconstitution.
  • Takeda plans to discontinue its first-generation GAMMAGARD S/D product by December 2027, with commercialization of the new therapy beginning in the U.S. in 2026.
  • Primary immunodeficiency affects approximately 1 in 1,200 people in the United States and comprises more than 550 rare chronic disorders affecting immune system function.

China Approves ORPATHYS-TAGRISSO Combination for EGFR-Mutated Lung Cancer with MET Amplification

Drug ApprovalTargeted Therapy
  • China's National Medical Products Administration approved the ORPATHYS-TAGRISSO combination for treating EGFR-mutated non-small cell lung cancer patients with MET amplification after disease progression on EGFR inhibitor therapy.
  • The Phase III SACHI trial demonstrated a 66% reduction in disease progression risk compared to chemotherapy, with median progression-free survival of 8.2 months versus 4.5 months.
  • This represents the first all-oral, chemotherapy-free treatment option for this patient population, addressing a critical resistance mechanism in lung cancer therapy.
  • The approval triggers an $11 million milestone payment from AstraZeneca to HUTCHMED and marks the third indication for ORPATHYS in China.

A trial to learn how well osimertinib with savolitinib works in people with non-small cell lung cancer (NSCLC) that has gotten worse after initial treatment with osimertinib.

2024-512581-34-00Active, Not RecruitingPhase 2
AstraZeneca AB

Clinical Study on Savolitinib + Osimertinib in Treatment of EGFRm+/MET+ Locally Advanced or Metastatic NSCLC

NCT05009836RecruitingPhase 3
Hutchison Medipharma Limited
Posted 9/6/2021

AZD9291 in Combination With Ascending Doses of Novel Therapeutics

NCT02143466Active, Not RecruitingPhase 1
AstraZeneca
Posted 8/5/2014

Savolitinib Plus Osimertinib Versus Platinum-based Doublet Chemotherapy in Participants With Non-Small Cell Lung Cancer Who Have Progressed on Osimertinib Treatment

NCT05261399RecruitingPhase 3
AstraZeneca
Posted 8/3/2022

Savolitinib for Treating Locally Advanced or Metastatic Non-small Cell Lung Cancer (NSCLC) Patients

NCT04923945CompletedPhase 3
Hutchison Medipharma Limited
Posted 8/19/2021

Savolitinib for Treating Gastric Cancer and Esophagogastric Junction Adenocarcinoma Patients

NCT04923932Active, Not RecruitingPhase 2
Hutchison Medipharma Limited
Posted 7/27/2021

Study on Savolitinib Combined With Osimertinib in Treatment of Advanced NSCLC With MET Amplification

NCT05015608Active, Not RecruitingPhase 3
Hutchison Medipharma Limited
Posted 11/22/2021

MEDI4736 Combinations in Metastatic Renal Cell Carcinoma

NCT02819596CompletedPhase 2
Queen Mary University of London
Posted 5/3/2016

A Phase II Study of HMPL-504 in Lung Sarcomatoid Carcinoma and Other Non-small Cell Lung Cancer

NCT02897479Unknown StatusPhase 2
Hutchison Medipharma Limited
Posted 12/1/2016

Savolitinib vs. Sunitinib in MET-driven PRCC.

NCT03091192Active, Not RecruitingPhase 3
AstraZeneca
Posted 7/25/2017

Iterum Therapeutics Appoints Christine Coyne as Chief Commercial Officer to Lead ORLYNVAH Launch

Drug Approval
  • Iterum Therapeutics has appointed Christine Coyne as Chief Commercial Officer to lead the upcoming U.S. launch of ORLYNVAH, an FDA-approved oral antibiotic for uncomplicated urinary tract infections.
  • Coyne brings over 30 years of pharmaceutical commercial experience, including previous roles as Chief Commercial Officer at Innoviva and SCYNEXIS, with specific expertise in anti-infective portfolios.
  • ORLYNVAH represents a novel oral penem antibiotic targeting multi-drug resistant pathogens, including those with extended spectrum beta-lactamase resistance, addressing significant unmet medical needs.
  • The appointment comes at a pivotal time as Iterum prepares for commercial launch with partner EVERSANA, positioning the company for sustained growth in the anti-infective market.
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