Pharmaceutical Technology Editors

Bio-Rad Laboratories Pioneer Antibody Discovery Platform is an antibody discovery service specifically designed to develop best-in-class biologic candidates. The platform, which includes more than 200 billion unique sequences, is capable of identifying high-affinity antibody candidates via Bio-Rad’s SpyDisplay selection technology; this technology is optimized for candidates suitable for drug development.

The platform is tailored to specific client requirements and uses proprietary TrailBlazer technology to provide prototypes of therapeutic antibodies in various formats for streamlined lead identification. Final immunoglobulin G antibodies are delivered functionally characterized with a complete data package and sequences. Customers retain exclusivity and ownership over antibody sequences produced from the platform.

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Bio-Rad Laboratories’ Pioneer Antibody Discovery Platform is an antibody discovery service specifically designed to develop best-in-class biologic candidates.

Biologics-Oriented Antibody Discovery Platform

Emer Cooke, the executive director of the European Medicines Agency (EMA), shared her optimism for the new year ahead, considering the multiple changes and challenges that the COVID-19 pandemic has brought since it started.

Cooke highlighted how seven COVID-19 vaccines and four vaccines tailored to the specific strains of the SARS-CoV-2 virus were authorized for use in the European Union since the start of the pandemic, bringing the population to a much better place than previous years. 

“Thanks to the rapid pace of scientific progress, and to EMA’s and the regulatory network’s ability to adapt during these times of crisis, we are able to experience some return to normality again,” Cooke said in a press release. “The knowledge we gained with COVID-19 also helped us mount a rapid response to mpox (monkeypox), declared a public health emergency by the World Health Organization (WHO) in the middle of the year.”

Additionally, 89 human medicines have been recommended for approval by the EMA, and the organization also analyzed a new vaccine against dengue and two diabetes treatments that address important public health issues outside the EU.

Cooke recognized EMA’s efforts and successes during the pandemic, with an extended mandate that helped formalize their existing procedures and provided new tools to respond effectively to future public health crises, tackled medicine shortages, and improve the monitoring of medical devices and their authorization procedures.

She also mentioned the Clinical Trials Regulation and the Clinical Trials Information System (CTIS) launching at the same time to help energize the clinical research area of the EU.

“It is a major change management initiative, a move from an entirely national system to a system with a single EU submission, coordinated assessment between the Member States, and high levels of transparency never seen before for clinical trials,” Cooke said in a press release. “We are working hard to deliver the next CTIS milestone: the mandatory use of the system as of 31 January 2023.”

Cooke concluded that she hopes to continue supporting a work environment that prevents burnout and promotes the wellbeing of all staff members. This can range from listening directly to staff feedback, personal challenges, and struggles that help apply the best approaches.

EMA Executive Director Sends Message of Promoting Stronger Public Presence for Safety, Efficacy of Medicines

The management board of the European Medicines Agency (EMA) meet on December 14–15, 2022 in Amsterdam to discuss recent activities related to the response to COVID-19, including seven vaccines, four adapted vaccines, and eight treatments authorized for use in the European Union.

The meeting went into detail about multiple topics, such as a new budget and updated multiannual programming document for 2023–2025, which looks at looking at the growing workload from COVID-19 and the availability of resources.

In addition, the Clinical Trials Regulation is set to become a single-entry point for sponsors and regulators in January 2023 for the submission and assessment of all new clinical trials.

The Committee for Advanced Therapies Report also presented key highlights and achievements of the Committee from 2017-2022 to the Board. The Board took time to acknowledge the work of the Committee in the last 6 years and its continued efforts to support developers through the production of guidance documents and the organization of workshops and training.

Highlights of December 2022 EMA Management Board Meeting 

The European Medicines Agency’s (EMA’s) human medicines committee (CHMP) recommended five medicines for approval at the committee’s December 2022 meeting.

Hemgenix (etranacogene dezaparvovec) as an advanced therapy medicinal product and as the first gene therapy for the treatment of severe and moderately severe Hemophilia B

Imjudo (tremelimumab) to be used in combination with Imfinzi (durvalumab) for the treatment of adults with hepatocellular carcinoma

Tremelimumab AstraZeneca (tremelimumab) for the treatment of metastatic non-small lung cancer in combination with Imfinzi and platinum-based chemotherapy

Pombiliti for the treatment of glycogen storage disease type 2, or Pompe disease

Dimethyl fumarate Accord (dimethyl fumarate) for the treatment of multiple sclerosis.

With these positive reviews, the CHMP also recommended the refusal of a marketing authorization for Omblastys for the treatment of neuroblastoma, which is a rare type of cancer.

In terms of extensions, the committee recommended Adcirca, Dupixent, Edistride, Enhertu, Fintepla, Forxiga, Hemlibra, Imfinzi (including two new indications), Kerendia, Spikevax and Triumeq.

EMA Recommends Five Medicines for Approval

The US District Court for the Western District of Oklahoma entered a consent decree against Qualgen LLC, an outsourcing facility with a history of violations, which prohibits the company from directly or indirectly distributing adulterated drugs in interstate commerce.

The consent decree follows a complaint filed by the US Department of Justice on behalf of FDA against Qualgen, its majority owner, Shaun Riney, and its Director of Quality, Jasen Lavoie. The complaint states the company introduced adulterated drugs into interstate commerce that were manufactured, processed, packed, or held under conditions that defy current good manufacturing practice (CGMP) requirements.

“While drugs compounded by outsourcing facilities are not subject to pre-market review and approval by the FDA for safety and effectiveness, they must comply with rigorous manufacturing quality assurance requirements,” said Jill P. Furman, JD, acting director of the Office of Compliance in FDA’s Center for Drug Evaluation and Research, in a press release. “The FDA’s commitment to ensuring compliance with current good manufacturing practice requirements is a critical protection for patients. We will remain vigilant and hold all manufacturers accountable to best protect the public health.”

Looking at section 503B of the Federal Food, Drug, and Cosmetic Act (FD&C Act), a compounder can register as an outsourcing facility, which is defined as a facility at one geographic location or address that is engaged in the compounding of sterile drugs, has elected to register as an outsourcing facility, and complies with all the requirements of section 503B of the FD&C Act.

Qualgen is currently registered with FDA as an outsourcing facility.

Drugs that are compounded by an outsourcing facility can qualify for exemptions from FDA approval requirements and the requirements to label products with adequate directions for use, but not from CGMP requirements, according to FDA.

During several inspections conducted since 2015, the agency cited Qualgen for violations of CGMP requirements, issuing a safety alert in 2015 and a warning letter in 2016. Most recently, FDA inspected Qualgen’s Oklahoma facilities, which ended in September 2022, where it manufacturers and ships compounded drugs. 

Although compounded drugs can be crucial for patients whose medical needs cannot be met by an FDA-approved drug product, they are not approved by FDA themselves and have not been evaluated for safety or efficacy.

The complaint states the company introduced adulterated drugs into interstate commerce that were manufactured, processed, packed, or held under conditions that defy current good manufacturing practice (CGMP) requirements. 

Consent Decree Against Oklahoma Drug Compounder Enters Federal Court

Albireo Pharma, Inc. announced on December 19, 2022 the submission of a supplemental New Drug Application (sNDA) to FDA and a variation application to the European Medicines Agency (EMA) for approval for a second Bylvay (odevixibat) indication for use in patients with Alagille syndrome (ALGS). 

“The clear results from our ASSERT Phase III data allowed the Company to swiftly submit FDA and EMA regulatory filings,” said Ron Cooper, president and chief executive officer of Albireo, in a press release. “We are getting one step closer to expanding access to Bylvay beyond PFIC [progressive familial intrahepatic cholestasis] patients to the Alagille patient community and delivering on our mission of bringing hope to families with cholestatic liver diseases.”

Bylvay has minimal systemic exposure and acts locally in the small intestine as a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi). The drug is already approved in the United States for the treatment of pruritis in patients three months of age and older in all types of PFIC and in Europe for the treatment of all types of PFIC in patients six months of age and older.

New positive data from the Phase III ASSERT study showed that Bylvay provided early, rapid, clinically meaningful and sustained improvements in pruritus and significant reductions in bile acids and improvements in sleep quality across the two most prominent genetic types in Alagille syndrome: JAG1 and NOTCH2. The study showed that more than 90% of patients were pruritus responders and Bylvay was well tolerated. Further, the overall incidence of treatment emergent adverse events was like the placebo. There were no patients who discontinued the study, and 96% transitioned over to the open-label extension study.

Albireo Pharma, Inc. hopes to approve Bylvay in the second half of 2023, and it is being evaluated in the Phase III BOLD study in biliary atresia.

Bylvay has minimal systemic exposure and acts locally in the small intestine as a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi). 