Pharmaceutical Technology Editors

VIVEbiotech, a gene transfer technologies contract development and manufacturing organization (CDMO) specializing in lentiviral vectors, announced on June 4, 2021 that it has opened new facilities in Spain for the manufacture of lentiviral vectors for the cell and gene therapy sector.

This expansion will allow the company to significantly increase its production capacity by 400% and deliver commercial-size batches of lentiviral vectors. The expansion also translates to an increase in average batch size per year from 20 batches in 2020 to 80 batches in 2022 of vector material. Furthermore, the facilities are capable of supplying material for early stage research as well as for good manufacturing practice (GMP) clinical- and commercial-scale quantities.

VIVEbiotech also plans to install the largest bioreactor currently available on the global market within the new facilities and seven cleanrooms specifically set up for lentiviral-vector manufacturing, the company stated in a press release. VIVEbiotech said it is using a flexible design that will allow it to further add more cleanrooms in the future as well as accommodate a larger number of batches.

The construction of these new facilities follows an authorization granted by the Spanish Agency of Medicines and Medical Products that allows VIVEbiotech to manufacture drugs to be administered 

 in clinical trials. VIVEbiotech has also been authorized to manufacture intermediate products. The company currently works in accordance with European Medicines Agency and FDA regulatory requirements.

“The continued expansion of the gene and cell therapy sector has resulted in a significant increase in the demand for lentiviral vectors. In order to meet the exponential growth of lentiviral demand to support the growth of the wider cell therapy industry, VIVEbiotech has invested in new, state-of-the-art, manufacturing facilities [that] will enable the commercial-scale production of our products,” said Gurutz Linazasoro, CEO, VIVEbiotech, in the press release.

“VIVEbiotech [is] one of the few companies that have fully specialized [capabilities] in the manufacture of lentiviral vectors, with expertise in all phases of development, from drug discovery to commercial scale-manufacturing. The new facilities will enable VIVEbiotech to develop new projects and increase our offering to our growing customer base,” Linazasoro added in the press release.

Articles

VIVEbiotech has opened its new lentiviral vector manufacturing facilities in Spain, expanding capacity for lentiviral vectors for use in cell and gene therapies.

VIVEbiotech Launches Lentiviral Vector Manufacturing Plant for Cell and Gene Therapies

 on June 3, 2021, that provide recommendations for complying with the Drug Supply Chain Security Act (DSCSA) in order to identify and trace drug products as they move through the supply chain. Two final guidance documents and two draft guidance documents were issued.

Product Identifiers Under the Drug Supply Chain Security Act, Questions and Answers

clarifies the DSCSA requirement that manufacturers and repackagers put a product identifier on drug packages that includes a national drug code, serial number, lot number, and expiration date.

Drug Supply Chain Security Act Implementation: Identification of Suspect Product and Notification

 offers certain trading partners aid in identifying a suspect product. It also describes how to notify FDA of illegitimate products.

Definitions of Suspect Product and Illegitimate Product for Verification Obligation under DSCSA

 describes terms used to define “suspect” and “illegitimate” products and revises the March 2018 draft guidance, and 

Enhanced Drug Distribution Security at the Package Level under DSCSA

 details requirements for enhanced drug distribution security at the package level and provides recommendations on the system attributes necessary for secure tracing of product at the package level.

The agency issued two final and two draft guidance documents to help identify and trace drug products in the supply chain.

FDA Publishes Guidance Documents to Enhance Supply Chain Security

On June 7, 2021, AGC Biologics, a contract development and manufacturing organization (CDMO), announced that it has partnered with BioNTech to further supply plasmid DNA (pDNA) starting material for the Pfizer-BioNTech COVID-19 vaccine at AGC’s Heidelberg, Germany, facility. pDNA starting material is an essential component of BioNTech’s mRNA-based vaccine manufacturing process.

“We are honored that BioNTech has entrusted us to become part of their global supply network in an effort to make the vaccine available to as many people as possible around the world,” said Mark Womack, AGC Biologics’ chief business officer, in a company press release. “We are very proud of our efforts to provide our global customers with the essential materials needed to rapidly deliver vaccines in the fight against the COVID-19 pandemic.”

“The team at Heidelberg is enthusiastically embracing this important project with BioNTech,” said Dieter Kramer, AGC Biologics’ general manager, Heidelberg, in the press release. “[We] look forward to supporting BioNTech in the production of their mRNA-based COVID-19 vaccine with [p]lasmid DNA.”

AGC Biologics has partnered with BioNTech to further supply plasmid DNA starting material from its Heidelberg, Germany, facility for the Pfizer-BioNTech COVID-19 vaccine.

AGC Biologics to Supply More Plasmid DNA for Pfizer-BioNTech COVID-19 Vaccine

Boehringer Ingelheim and Zealand Pharma announced on June 2, 2021 that FDA has granted fast track designation to BI 456906, an investigational glucagon-like peptide 1 (GLP-1)/glucagon dual agonist, for the treatment of non-alcoholic steatohepatitis (NASH). BI 456906 is currently in a Phase II study in adults with NASH and liver fibrosis with and without diabetes.

GLP-1/glucagon is a compound derived from oxyntomodulin, a natural gut hormone, that activates both the GLP-1 and glucagon receptors critical for controlling metabolic functions. The dual agonist BI 456906 has the potential to be a new, once-weekly treatment and is also being investigated as a potential treatment for adults with diabetes and for adults with obesity.

“The FDA [f]ast [t]rack [d]esignation for our dual agonist is an important step forward in addressing the high unmet medical need among the up to 444 million adults estimated to be living with NASH,” said Waheed Jamal, MD, corporate vice-president and head of CardioMetabolic Medicine, Boehringer Ingelheim, in a company press release. “Together with our partner Zealand Pharma, we look forward to working closely with [FDA] as we explore the potential of the GLP-1/glucagon agonist to improve outcomes for adults with NASH.”

“Boehringer Ingelheim and Zealand Pharma are committed to delivering innovative solutions that address public health challenges of cardiometabolic diseases, including NASH,” said Adam Steensberg, executive vice-president and chief medical officer at Zealand Pharma, in the press release. “By combining Boehringer Ingelheim’s expertise in drug development in the cardio-metabolic area with our strength in the discovery of innovative peptide-based medicines, we have the potential to bring forward a novel therapy option in an area with limited available treatments.”

FDA has granted fast track designation to a GLP-1/glucagon dual agonist in development by Boehringer Ingelheim and Zealand Pharma for the treatment of NASH.

Boehringer Ingelheim and Zealand Pharma Receive FDA Fast Track Designation for NASH Treatment

 on June 4, 2021, that it has approved Ryplazim (plasminogen, human-tmvh) to treat plasminogen deficiency type 1. Also known as hypoplasminogenemia, the rare disorder can impair tissue and organ function and may lead to blindness, according to the agency. Ryplazim was granted Orphan Drug and Fast Track designations as well as Priority Review and a Rare Pediatric Disease Priority Review Voucher. 

Ryplazim’s active ingredient, plasminogen, is purified from human plasma. Ryplazim helps increase the level of plasminogen in patients who lack the protein, the agency says, “enabling a temporary correction of the plasminogen deficiency and reduction or resolution of the lesions.”

FDA based the safety and effectiveness of the drug on one single-arm, open-label clinical trial that included 15 adult and pediatric patients with plasminogen deficiency type 1. Patients received the drug every two to four days for 48 weeks. A 50% improvement of lesions was seen in all 11 patients who had lesions at baseline. No recurrent or new lesions were seen in any of the 15 patients through the time of treatment.

“Until now, there were no FDA-approved treatment options for patients with plasminogen deficiency type 1,” said Peter Marks, MD, PhD, director of FDA’s Center for Biologics Evaluation and Research. “Today’s approval helps address an unmet medical need for individuals affected by this rare genetic disease.”

FDA approved Ryplazim (plasminogen, human-tmvh) for the treatment of plasminogen deficiency type 1.

FDA Approves Treatment for Plasminogen Deficiency Type 1

The Janssen Pharmaceutical Companies of Johnson & Johnson announced on June 1, 2021 that FDA has granted breakthrough therapy designation for teclistamab, an off-the-shelf, T-cell redirecting, bispecific antibody, for treating relapsed or refractory multiple myeloma. FDA’s designation follows a priority medicines (PRIME) designation granted by the European Medicines Agency in January this year. In 2020, the European Commission and FDA each granted teclistamab orphan drug designation for the treatment of multiple myeloma.

Teclistamab targets both B-cell maturation antigen (BCMA), which is expressed at high levels on multiple myeloma cells, and CD3 receptors. The bispecific antibody redirects CD3-positive T-cells to BCMA-expressing myeloma cells to induce killing of tumor cells. Results from preclinical studies demonstrated that teclistamab kills myeloma cell lines and bone marrow-derived myeloma cells from heavily pretreated patients. Teclistamab is currently in Phase II clinical development and is also being explored in combination studies.

“We are pleased to have received [b]reakthrough [t]herapy and PRIME [d]esignations for our novel bispecific antibody, teclistamab,” said Peter Lebowitz, MD, PhD, Global Therapeutic Area head, Oncology, Janssen Research & Development, in a company press release. “This program exemplifies our commitment to advancing science for patients living with multiple myeloma, and it builds upon our robust portfolio in this disease.”

FDA has granted Janssen breakthrough therapy designation for teclistamab, an investigational bispecific antibody under development for treating relapsed or refractory multiple myeloma.