Drug Quality and Safety Management

PharmaEssentia is committed to solving unmet medical needs. After achieving breakthroughs in the MPN domain, we adhered to the spirit of the Access to Medicine Index and continued to invest in research on blood disorders and solid tumors. We are also working with external research institutes and biotechnology companies to jointly develop cell therapies.
PharmaEssentia screens domestic and international research institutes with GLP, ISO 17025 (non-compulsory), and AAALAC certifications to ensure that research personnel rigorously adhere to all relevant regulations during R&D processes for new drugs and use humane procedures when conducting preclinical animal trials.

R&D Focus

The global biopharmaceutical industry is flourishing due to advances in medical technologies and aging populations, and therefore continuous innovation and development is an important mission for pharmaceutical companies. PharmaEssentia is a new drug developer and biologics manufacturer that is actively developing drugs for MPN. The four main types of MPN are:

Material Topics New Drug Research & Development and Innovation Management-R&D Product Pipelines

• Governance

  New drugs must pass rigorous quality, safety, efficacy, and regulatory controls over a long period of time that spans from drug discovery, feasibility studies, preclinical animal trials, clinical trials, product manufacturing, and market authorization reviews before launch. PharmaEssentia has established complete drug R&D governance organizations to ensure that all responsible units implement efficient management and progress at all stages of new drug development.

  PharmaEssentia Drug R&D to Marketing Process

  

• Strategy

  Innovation and R&D Focuses

  In 2024, PharmaEssentia completed Phase 3 ET clinical trials (LPLV) for Ropeg. We expect to apply for ET marketing authorizations in Taiwan, the US, Korea, and China in 2025 to bring more medical value to the MPN field and strengthen PharmaEssentia’s leadership in this domain.

  PharmaEssentia Innovation Research Center (PIRC)

  PharmaEssentia established the PharmaEssentia Innovation Research Center in 2023 to work with headquarters on breaking into new research fields. We hope to further expand our R&D and innovation capacity by combining AI (artificial intelligence) and ML (machine learning) to accelerate the process from new drug development to market launch. In 2024, we worked with Qiagen to utilize data analytics and AI in finding new indications for Ropeg, and presented the results at the ASH Annual Meeting. We also worked with DeepSeq.AI in using AI to design new-generation smart cytokines that could be used in cancer treatments.

• Risk Management

  Commitment to Animal Welfare in Preclinical Animal Experiments

  Adhere to our requirements and standards, such as whether they hold GLP (Good Laboratory Practice), ISO 17025 (Laboratory Quality Management System), and AAALAC (American Association for Accreditation of Laboratory Animal Care) certification, and then conduct online or on-site visits/audits to better understand the expertise of the CRO (Contract Research Organization) and expert team, trial schedule coordination, and market price rationales. We also review contracts and technical agreements with legal personnel, supervise trials and review data during trial periods, and compile reports for review once trials have concluded.

  Clinical Trial Quality Maintenance and Participant Safety in Clinical Trials

  

• Metrics and Targets

  

PharmaEssentia’s Taichung Plant rigorously adheres to the Quality Risk Management Procedure, Equipment Risk Assessment Procedure, and Change Control Procedure; has incorporated risk management procedures into production processes, environmental controls, and material supplies; and conducts annual quality reviews to minimize quality hazards.

PharmaEssentia Global Certification Landscape

We use practical actions to achieve vertical integration within our supply chain, gradually building our global market from production, quality control, filling, to delivery, actively demonstrating our vision to become a world-class pharmaceutical company.

• Regulatory Agency Inspections

  • Regulatory agency inspections
  • 2021
  • 2022
  • 2023
  • 2024

  • Number of inspections
  • 4
  • 5
  • 5
  • 2

  Note1：PharmaEssentia did not receive any Form 483s (or equivalent) in the last four years.
  Note 2：PharmaEssentia did not receive any Warning Letters (or equivalent) in the last four years.

International Standard Manufacturing Processes

PharmaEssentia’s product Ropeg undergoes four critical stages in production: fermentation and cell processing, P1040 extraction and purification, PEGylation, and Ropeg DS purification. Sterile filling, labeling, and packaging processes all rigorously comply with GMP and international standards for quality management and standard operating procedures. In response to commercialization and mass production demands, we continue to enhance all production stages as well as overall production capacity, reduce supply chain risks, and improve supply stability.

Process Optimization

Quality Control and Risk Management in Manufacturing Processes

PharmaEssentia’s Taichung Plant emphasizes product quality and safety management. Each year, our colleagues at the plant must undergo education and training related to product safety and GMP regulations so they can update their knowledge and incorporate quality management concepts in daily tasks. The Taichung Plant has developed work procedures and organizational operational procedures for factories in different countries that produce GMP-compliant APIs and drug formulations, which are detailed below. In 2024, we continued to implement, monitor, and report trends associated with production environments (HVAC systems), water systems, compressed air systems, and biosafety cabinets, requiring all systems to comply with design specifications and legal regulations. The Taichung Plant has also formulated the “Plant and Facility Emergency Response Management Standard” to establish emergency response mechanisms for natural disasters, equipment abnormalities, and other emergency hazards, thereby ensuring that all equipment can operate normally and all personnel can work in safe environments.

Secure Distribution Processes and Stable International Logistics & Transportation

Our secure distribution processes strictly adhere to GDP regulations and ensure proper management of pharmaceuticals throughout the transportation process. PharmaEssentia’s GDP-compliant Panco Healthcare Logistics Center in Taichung supplies clinical drugs and marketed products and also implements quality management regarding logistics management, warehousing management, and labeling processes.

Warehouse Management

PharmaEssentia has established the “Product Receipt and Storage Management Operation Standards” and “Storage and Distribution Policy” to prevent negative environmental and operational impacts on product quality. The Panco Logistics Center has established a “Storage Area Temperature Verification Plan” which stipulates that temperature verifications should be conducted twice every three years to ensure warehouse environment quality.

Product Entry and Storage Process

Quality Control in Shipping and Transportation

PharmaEssentia has established the “Product Shipping Operations Standards” to ensure that distribution processes for products manufactured at the Taichung Plant and by CMOs, as well as packaging and transportation processes at storage warehouses, are appropriately managed and all pharmaceuticals are transported under prescribed and suitable temperature conditions to uphold global drug transportation safety.

Pharmaceutical Risk Management Plan

PharmaEssentia implements drug safety risk standard operating procedures formulated by collaborating CROs and has established “Drug Risk Management Plans” in accordance with the pharmacovigilance regulations of each country. Our headquarters established pharmacovigilance plans in accordance with the “Regulations for Pharmacovigilance Management” in Taiwan, and completed amendments in 2024 to include pharmacovigilance organizational charts, operational processes and responsibilities at each stage, pharmacovigilance quality systems, pharmacovigilance personnel education and training processes, pharmacovigilance document management, and addition of document numbers, formats, effective dates, and other information in accordance with our pharmacovigilance quality documentation SOP. Our subsidiaries in the US, Japan, and other locations have also established “Drug Risk Management Plans” in accordance with local regulatory requirements.

Pharmacovigilance Management

Our Pharmacovigilance Team was established under the Medical Research Department and works in coordination with relevant units to carry out their duties according to our “Pharmacovigilance Policy,” “Drug Safety Functions and Training Standard Operating Procedures,” “Post-Marketing Safety Data Collection Standard Operating Procedures,” as well as Taiwan’s “Regulations for Reporting Serious Adverse Reactions of Medicaments” and “Regulations for the Management of Drug Safety Surveillance.” We also commission professional CROs to conduct pharmacovigilance tasks. Our pharmacovigilance procedures include both passive monitoring and active monitoring:

Pharmacovigilance Reporting Education & Training and Reporting Program

Taiwan’s pharmacovigilance regulations require CROs to formulate and implement drug safety management and regulatory authority reporting plans, organize regular employee pharmacovigilance education and training, and preserve all training records.

Drug Safety Reporting Mechanism

Product Traceability Mechanisms

PharmaEssentia has established a global product traceability mechanism and incorporated drug serialization. We regulate packaging and serialization processes at our Taichung injectables plant and CMOs to achieve our goal of full traceability for all individual product flows and usage records. Drug serialization has also been fully implemented for Ropeg sold in the US, Taiwan, Korea, and China, and a qualified injectables facility packages and serializes drugs in accordance with the Drug Supply Chain Security Act (DSCSA) to maintain drug quality and safety.

Product Recall Mechanisms

PharmaEssentia’s “Return and Recall Procedures” clearly stipulates the use of a product traceability system to complete drug recall mechanisms, which allows for rapid and effective drug recalls if product quality concerns emerge, providing an additional safety mechanism for patients when using medications. Recall simulation drills are conducted every year to ensure accuracy and familiarity with recall actions. None of our drugs were recalled in 2024 due to adverse events.

Product Marketing Ethics

PharmaEssentia’s ethical marketing framework is founded on integrity, transparency, and compliance, ensuring that all promotional practices prioritize patient welfare and meet the highest regulatory and ethical standards. The Company adheres to the following principles:

1. Prioritize patient medical care and well-being
2. Meet high quality, safety, and efficacy standards required by regulatory authorities
3. When interacting with related units or personnel, all behaviors should be ethical, appropriate, and professional. Provision or supply of any materials or labor that directly or indirectly result in negative impacts are prohibited
4. Be responsible for providing accurate, balanced, and scientifically valid product information
5. Product marketing activities should be ethical, accurate, and balanced, and should not be misleading. Product marketing information should include accurate product risks, risk-benefit assessments, and appropriate usage methods
6. Respect patient privacy and personal information
7. Sponsor/support clinical trials and scientific research aimed at pursuing new knowledge to enhance patient interests, promote advances in medical technologies, and maintain transparency of human clinical trials sponsored by the industry
8. Transparent Review and Approval Mechanisms

   PharmaEssentia implements a transparent reporting system under theMedical Legal Regulatory (MLR) Committee, which reviews and approves all written materials potentially regarded by the FDA as promotional or labeling for external use. This includes promotional materials, non-promotional materials, advertisements, and other external communications. The Committee ensures scientific accuracy, compliance with internal policies, and adherence to applicable laws and regulations. It also periodically reviews evolving regulatory frameworks and market needs to adjust its approval considerations accordingly.

9. Commitment to Safety and Risk Management

   Patient health and welfare remain a top priority. PharmaEssentia actively tracks and reports adverse drug events, fostering a “quality first, patient safety” culture. The Company integrates comprehensive risk management practices and communicates transparently on product approval status, while disclosing information on pharmacological action, indications, pregnancy classifications, dosage and administration, side effects, contraindications, and precautions to provide balanced benefit-risk reporting.

PharmaEssentia’s flagship product Besremi® is Taiwan’s first protein-based innovative drug approved by the European Union and the world’s first interferon-based therapy formally approved as a first-line treatment for polycythemia vera (PV). Detailed prescribing information is available via the E-Da Hospital “Drug Identification and Formulary” platform.