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CONTROL STRATEGY AND OTHER TECHNICAL CONSIDERATIONS

The CM system and its control strategy were designed to control parameters that impact the 602

manufacture and quality of the drug substance, including impurity profile and physicochemical 603

properties. The overall control strategy was developed in accordance with the main guideline and 604

ICH Q7–Q11.

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2.1. Equipment Design and Integration 606

 Reaction 1: Starting materials 1 and 2 are coupled in a PFR to produce Intermediate 1.

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Diversion Point D1 is located after the PFR to permit material diversion when PFR 611

conditions are outside predefined acceptance criteria. The reaction is quenched as an 612

integrated operation after the PFR, and unwanted by-products are removed by liquid-liquid 613

 Reaction 2: Intermediate 1 and Intermediate 2 (prepared upstream through separate batch 617

unit operations) are coupled in a second PFR to form the crude drug substance. The online 618

PAT near the reactor exit (T1) monitors conversion of Intermediate 1 to the crude drug 619

substance. Diversion Point D2 located after PAT is used to divert non-conforming material.

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 Drug Substance Isolation: The crude drug substance is purified by carbon filtration and 622

continuous two-stage crystallisation. The crystal slurry is filtered by using two identical 623

filtration units running in an alternating fashion. This setup enables continuous processing 624

of the drug substance after crystallisation by allowing the collection of crystallised products 625

on one filter unit at the same time product isolated on the second filter is discharged.

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Diversion Points D3 and D4 allow for material diversion at the crystalliser and just before 627

batch operations, respectively. A batch dry milling operation is used to achieve the desired 628

particle size distribution of the crystallised drug substance.

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Three surge points (each containing multiple surge tanks) are used: one before Reaction 2, another 631

before the two-stage continuous crystallisation, and one just before final batch operations. These 632

are important components of the system design and control strategy, as they improve process 633

robustness and mitigate temporary differences in mass flow rates by decoupling upstream and 634

downstream operations.

635 636

The design of the overall system and each unit operation, along with the control strategy, optimise 637

material quality. For example, PFR design elements (i.e., dimension and configuration) allow 638

precise control of temperature, mixing and reactant flows. These parameters were shown during 639

development to be important to the drug substance impurity profile.

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2.2. Process Control and Monitoring 641

Holistic controls used across Reactions 1 and 2 ensure consistent operations and quality of the 642

resulting crude drug substance. The stoichiometry of Reaction 1 is controlled precisely via control 643

of concentrations and flow rates of the feeds. Conversion of starting materials to Intermediate 1 644

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with minimal impurity formation is ensured through control of the reaction temperature. Reaction 645

2 is controlled through feedback control of the addition rate of Intermediate 2 based on the PAT 646

measurement of Intermediate 1 levels. This ensures correct stoichiometry for that reaction and 647

minimises the impact of variability of the Intermediate 1 feed solution on drug substance purity.

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The PAT also measures levels of crude drug substance and process impurities, which confirm 649

successful operation of all preceding steps and consistent product quality.

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RTD was used to develop a suitable strategy for disturbance detection, corrective actions, and 652

material diversion. RTD characterisation was based on mathematical modeling of all unit 653

operations and surge points across the entire CM process over planned mass flow rates. The RTD 654

was then confirmed through experimental tracer studies for appropriate segments of the 655

commercial equipment. Decisions for triggering material diversion are based on comparing 656

process parameters and PAT measurements to predefined acceptance criteria with timing and 657

Understanding of process dynamics and its impact on quality attributes of material produced 661

throughout the entire process was also used to guide start-up and shutdown strategies. For example, 662

during start-up of Reactions 1 and 2, a small amount of Intermediate 1 or crude drug substance is 663

diverted at Diversion Points 1 or 2, respectively, to allow those materials to reach the target 664

concentrations before processing into subsequent operations. The criteria for diversion were 665

established based on time considering the RTD. This approach was supported by development 666

studies and confirmed in commercial process equipment. PAT monitoring after Reaction 2 667

provides additional verification that appropriate criteria have been met during start-up. Collection 668

of material proceeds to the end of the process as subsequently described.

669 670

Sampling and process measurement needs were evaluated, considering relevant factors such as 671

residence times (RTs)/RTD, surge points, process dynamics, and the type and purpose of the 672

measurement. The measurement frequency of the PAT at Reaction 2 is sufficient to detect 673

disturbances, inform process adjustments, and ensure timely diversion of material based on 674

predefined criteria. The criteria for material diversion are based on the magnitude and duration of 675

the disturbance, an understanding of process dynamics and RTD for downstream unit operations 676

and surge points, and the impurity purging capability of the crystallisation operation. As a result 677

of this control strategy, all crude drug substance solution that enters continuous crystallisation 678

meets acceptable quality criteria and can be forward processed through the crystalliser.

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Appropriate controls and monitoring requirements for the continuous crystallisation were 681

extensively investigated during development in similar, but smaller scale equipment and verified 682

using commercial equipment. Process development included spiking studies using impurity-683

enriched feed solutions and intentional perturbations in process parameters (i.e., feed flow rates, 684

their ratios, and temperatures). An evaluation of the encrusted solids in the crystalliser over 685

extended run times demonstrated the solids were the same form and purity as the free-flowing drug 686

substance slurry. The set of process parameters and ranges identified by these studies were 687

appropriately scaled up. Implementation of these controls along with post-crystallisation material 688

tests (e.g., crystal form, purity) ensure consistent quality of the resulting drug substance throughout 689

continuous crystallisation and filtration.

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The resulting material is collected at Surge Point 3 and is dried and milled using batch operations 692

to provide a drug substance of the appropriate particle size for use in drug product 693

manufacturing. Procedures were developed to allow diversion of material at Diversion Points D3 694

or D4 in the event desired process conditions or material attributes are not met. However, diversion 695

of the drug substance from the crystalliser was found to be unnecessary either during start-up or 696

shutdown.

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2.3. Consideration of Other Controls 698

Process robustness and performance over time are important considerations. A risk assessment 699

was performed to ensure that adequate controls are in place to support the proposed run time 700

(which can be up to several months). It identified a number of considerations and corresponding 701

controls/measures. Examples are summarised in Table 2.

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Table 2: Examples of other controls for consideration 704

Consideration Controls/Measures

Cleaning and fouling potential

 Establishment of a risk-based cleaning strategy, including

understanding of the impact of build-up on drug substance quality

 Additional monitoring to assess fouling and cleanliness (e.g., pressure sensors at the discharge of feed pumps, periodic visual checks for the continuous crystalliser)

 Reduction of other risk factors (e.g., filtering feed streams to further reduce fouling risk)

Stability of in-process materials

 Hold times at key points in the process (e.g., feed streams;

accumulated material at the surge points, reactors, and crystalliser) managed through batch record and process automation

 Risk assessment of microbiological growth (i.e., negligible risk based on the nature of the process materials and conditions)

Calibration and potential for changes/drift in instrumentation

 Periodic checks at selected points (e.g., process parameter

measurements for the PFR, system suitability for the PAT analyser)

 Dual sensors at selected locations (e.g., temperature probes for the PFR) so that appropriate corrective actions can be taken

Equipment maintenance

Maintenance requirements for target run time

Use of redundant equipment (e.g., backup pumps) at key locations to enable continuous operation

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Additionally, specifications for input materials were evaluated during process development. There 706

were no differences between batch and continuous processing for this example.

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Collectively, the process understanding developed along with implementation of the various 709

controls described provide a robust and reliable control strategy. This ensures consistent quality of 710

the resulting drug substance including the impurity profile, physicochemical properties, and ability 711

of the system to identify and appropriately react to unexpected events.

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2.4. Process Validation 713

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The combination of process controls, online PAT measurements, comprehensive monitoring of 714

process parameters and material attributes, and end-product testing results in a data-rich 715

environment for this process. Together with system understanding generated during development, 716

this enabled the use of a traditional process validation for commercial product launch and 717

continuous process verification to validate process changes over the product lifecycle.

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A range of batch sizes was initially established based on material demands and the quantities of 720

material necessary to match input needs of the final batch unit operations. The process was 721

validated using a fixed number of batches. A single planned start-up and shutdown of the 722

commercial CM system was used to manufacture the process validation batches. This approach 723

was supported by the totality of evidence demonstrating the start-up and shutdown capabilities of 724

the system. This included development work on similar equipment, commercial equipment and 725

system qualification data, results of a prevalidation demonstration run, and extensive process 726

monitoring of the CM system that can verify success of each start-up and shutdown in real time.

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Subsequently, a continuous process verification approach was adopted after product approval to 729

support increases in batch size with extension of run time. This approach used a risk assessment 730

for the longer run time, which concluded that process performance and material quality would not 731

be impacted. Under the continuous process verification approach, data generated during the 732

manufacture of each batch was used to support successful validation of that batch with the 733

extended run time. This included information such as system performance monitoring and data 734

logs along with other controls that ensure material quality with appropriate detection and corrective 735

action. Additionally, appropriate regulatory actions were taken to communicate this manufacturing 736

change and use of the continuous process verification approach.

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3. REGULATORY CONSIDERATIONS 738

Refer to Section 4 of the main guideline. In consideration of the specific CM process design, 739

additional elements may need to be included in a dossier. For instance, in this example, the 740

influence of surge points on the material diversion and collection strategy, including the fate of 741

materials, was described.

742

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ANNEX II: CONTINUOUS MANUFACTURING FOR DRUG PRODUCTS