Pharmaceutical Technology Editors

On July 29, 2021, Emergent BioSolutions announced that FDA is allowing the company’s Bayview manufacturing facility in Baltimore, Md., to resume production of Johnson & Johnson’s (J&J) COVID-19 vaccine bulk drug substance. FDA’s decision comes after doing extensive reviews and is based on the diligent work and close coordination Emergent has conducted with J&J and the agency to execute a quality enhancement plan.

Since production was paused, Emergent has been working closely with FDA and J&J to address quality concerns and has developed and executed an action plan as well as committed extensive resources to bring operations up to FDA standards. The company expects to continue working with the agency throughout the manufacturing process to help ensure the stability of the J&J COVID-19 vaccine supply chain.

“We are proud to be resuming production of bulk COVID-19 vaccine batches following additional reviews and collaboration with FDA and our manufacturing partners,” said Robert Kramer, Emergent’s CEO, in a company press release. “We are in the unique business of producing life-saving medications for catastrophes that we hope never occur [such as] anthrax attacks, opioid overdoses, and COVID-19.”

“The American people should have high expectations of the partners its government chooses to help prepare them for disaster, and we have even higher expectations of ourselves,” Kramer added. “We have fallen short of those lofty ambitions over the past few months, but resumption of manufacturing is a key milestone, and we are grateful for the opportunity to help bring this global pandemic to an end. We’d like to thank our government partners as well as Johnson & Johnson for their support.”

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Emergent BioSolutions is being allowed to restart manufacturing operations for the J&J COVID-19 vaccine at its Bayview, Baltimore, Md., facility.

FDA Gives Green Light to Emergent BioSolutions to Resume COVID-19 Vaccine Manufacturing

Entries are open for the 18th Annual CPhI Pharma Awards, which are slated to take place on November 9, 2021, in Milan, Italy, at the Stella Polare Convention Centre. Awards in fifteen categories will be given out to innovators and thought leaders who are exploring uncharted territory in the pharma/biopharma industries.

Anyone involved in design, development, manufacture, supply, and distribution of large- and small-molecule drug products may apply. Winners will be selected by a panel of expert judges. The deadline for applications is August 6th, 2021.

This event is part of CPhI Worldwide, a pharma exhibition taking place November 9–11, 2021. Last year, the convention was cancelled as a result of the COVID-19 pandemic, with the entirely virtual CPhI: Festival of Pharma held in its place. This year’s event will be a hybrid of in-person and virtual activities.

“We host our awards because it’s important to recognize the innovative and inspirational work that is produced across the pharmaceutical industry” said Tara Dougal, head of content and insights, Informa Markets, in a July 19, 2021 company press release. “After a challenging year, the industry has been more committed than ever to advancing health and improving patient outcomes. It’s been a year in which we have seen the very best of pharma, with a record pace of innovation, discovery, and commercialization.”

The full awards list and submission requests can be found at 

Entries are now open for the 18th Annual CPhI Pharma Awards in Milan.

18th Annual CPhI Pharma Awards to Take Place in Milan, Italy

On July 29, 2021, VICO Therapeutics, a Netherlands-based biotech company focused on the development of RNA-modulating therapies for rare neurological disorders, announced that FDA had granted orphan drug designation (ODD) for VO659, an investigational antisense oligonucleotide therapy (AOT), in the treatment of Huntington’s disease (HD).

HD is a rare degenerative brain disease that results in motor disorders, impaired cognition, dementia, and various psychiatric manifestations. It is caused by a mutation to the CAG-trinucleotide repeat in the coding region of exon 1 of the HTT gene, resulting in a mutant huntingtin protein with an elongated polyglutamine (polyQ) stretch at its N-terminus. This grants a toxic gain-of-function to mutant protein forms, which results in widespread neuronal death.

VO659 is designed to suppress mutant proteins with the intent of slowing or halting the progression of disease. A month prior to this announcement, the drug had also received orphan drug designation for the treatment of spinocerebellar ataxia, a group of hereditary polyQ disorders that result in similar neuron degeneration.

ODD is granted to drugs intended for the safe and effective treatment, diagnosis, or treatment of rare (fewer than 200,000 cases) diseases or disorders. This designation grants various benefits, including seven years of market exclusivity following regulatory approval, exemption from FDA application fees for Huntington’s disease, and tax credits for qualified clinical trials.

“We are delighted that [FDA] has granted this orphan drug designation following the ODD for SCA one month ago,” said Rupert Sandbrink, chief medical officer at VICO, in a company press release. “This is affirmation of the potential of our [antisense oligonucleotide platform] approach.”

VICO Therapeutics’ drug candidate, VO659, for treating Huntington’s Disease was granted orphan drug designation by FDA. 

FDA Grants Orphan Drug Designation to VICO Therapeutics for Huntington’s Disease Drug 

Thermo Fisher Scientific has introduced Ion Torrent Oncomine immune repertoire assays, a new suite of next-generation sequencing (NGS) assays that are designed to detect potentially malignant clones of T cells and B cells, key players in immune response. The pan-clonality assays use proprietary Ion AmpliSeq technology to target multiple parts of B- and T-cell immune receptors. This is done using a single reaction with ultra-high sensitivity, which increases the probability of detecting malignant clones and decreasing the time to results, Thermo Fisher announced in a July 19, 2021 press release.

Detection of distinct receptor DNA sequences can inform diagnostic, prognostic, and therapy development studies, which can illuminate the progression of lymphoid cancers, including leukemias, lymphomas, and diseases such as multiple myeloma. Using NGS technology, detection sensitivity can be increased—compared to traditional testing methods, such as flow cytometry or quantitative polymerase chain reaction.

One of the challenges of current NGS-based immune repertoire analysis has been the need to run multiple secondary tests because of the high test failure rates of a typical single NGS assay, the company explained in the press release. The new Oncomine pan-clonality assays overcome this challenge by uniquely targeting multiple parts of the receptor to reduce the need for secondary tests and increase the probability of a single assay detecting a potentially malignant clone.

"Genomic testing can make a significant impact on researching and understanding these [blood] cancers—but we need to bring more testing to in-house laboratories where results can be made available quickly to healthcare professionals," said Garret Hampton, president of clinical next-generation sequencing at Thermo Fisher Scientific, in the press release. "The new Oncomine immune repertoire assays unlock the power of NGS to help researchers rapidly obtain a tremendous amount of insight about a lymphoid cancer sample, including its prevalence and an understanding of how it is evolving."

"With the ability to sequence multiple targets in a single reaction, our team can now get much more information from one assay when compared to single-target NGS tests," said John DeCoteau, medical director, University of Saskatchewan, Advanced Diagnostic Research Laboratory, in the press release. "Using the Oncomine BCR Pan-Clonality Assay, we were able to achieve a positive clonality detection rate of 93 percent in a cohort of multiple myeloma samples, without the need for secondary testing. That provides a remarkable improvement for laboratory efficiency and allows us to get results faster."

Thermo Fisher Scientific has added a new suite of immune repertoire assays that offers high detection rates of malignant clones, allowing researchers to better assess blood cancers.