Pharmaceutical Technology Editors

Roquette, a French excipient provider, announced on Sept. 20, 2022 that it would be fully acquiring Crest Cellulose, an Indian manufacturer of excipients. This move, which follows Roquette’s decision to purchase a majority stake in 2018, will make the company a wholly owned subsidiary of Roquette. According to a company press release, Roquette identified India as a major area of growth in the near future and purchased Crest to reinforce their presence in India and Asia overall.

“At Roquette, we believe true innovation can only be achieved in an environment where ingredient safety is completely assured,” said Paul Smaltz, vice-president, Roquette Pharma Solutions, in the press release. “There’s no room for compromise or shortcuts when tackling the world’s greatest health challenges. The team at Crest Cellulose share our conviction that quality is king, always striving to optimize processes and redefine what’s possible in drug delivery. As a global leader in health and nutrition, we’re excited to harness our newly expanded capabilities to support our network of customers and partners in India, Asia, and the rest of the world.”

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Roquette’s acquisition of Crest Cellulose is designed to reinforce its presence in India and other countries in Asia.

Roquette Acquires Indian Excipient Manufacturer

Aprecia, a pharmaceutical manufacturer specializing in 3D printing (3DP), announced on Sept. 20, 2022 that it had hired Chris Lockwood as head of technical services. Lockwood, who has 26 years of experience in the pharmaceutical industry, will bring his experience to Aprecia’s 190,000 ft² manufacturing facility in Blue Ash, Ohio.

“Chris’ extensive leadership and pharmaceutical experience will guide our technical services moving forward. We know he will contribute significantly to our current and future growth,” said Kyle Smith, president and chief operating officer, Aprecia, in a company press release.

“I am excited to work with Aprecia’s disruptive 3DP manufacturing technology platforms, as these enable a new spectrum of product and manufacturing possibilities compared to traditional manufacturing approaches,” said Lockwood.

Aprecia has hired Chris Lockwood as head of technical services at its Ohio facility.

Chris Lockwood to Lead Technical Services at Aprecia

FDA and the National Institutes of Health (NIH) announced the launch of the Critical Path for Rare Neurodegenerative Diseases (CP-RND), a public-private partnership, on Sept. 14, 2022. The goal of this partnership is to advance the understanding and develop treatments for amyotrophic lateral sclerosis (ALS) and other rare degenerative diseases.

The Critical Path Institute (C-Path) will convene the partnership, bringing together rare neurodegenerative disease experts, including patient communities, advocacy organizations, private entities, and more. According to an agency press release, areas of focus will include patient-focused drug development, characterization of neurodegenerative diseases and their natural history, the identification of molecular targets for neurodegenerative disease, and increased efficiency, predictability, and productivity of clinical development of therapies.

“There is a crucial need to develop new treatments that can improve and extend the lives of people diagnosed with rare neurodegenerative diseases, including ALS. Collaboration across public and private sectors can accelerate the progress to address this urgent need,” said Hilary Marston, chief medical officer, FDA, in the release. “The partnership we are announcing today will leverage the shared expertise of all participants to create a path towards new breakthroughs in treating these diseases. We look forward to working with NIH, C-Path, and other public and private partners to carry out this important effort.”

“This public-private partnership will convene the entire ALS community to develop novel strategies and approaches to therapy development and clinical testing with the goal to finally produce a treatment that stops the tragic progression of ALS,” said Walter Koroshetz, director of the National Institute of Neurological Disorders and Stroke, part of the NIH, in the release.

The Public-Private Partnership for Rare Neurodegenerative Diseases will work to enhance understanding and develop treatments for rare neurodegenerative diseases.

FDA and NIH Enter Rare Neurodegenerative Diseases Partnership

FDA announced that it had granted approval of SKYSONA (elivaldogene automecel) to bluebird bio for the treatment of active cerebral adrenoleukodystrophy (CALD) on Sept. 16, 2022. The treatment, a one-time infusion, is set to have a wholesale acquisition cost of three million dollars, making it one of the most expensive treatments on the market.

CALD is a rare, progressive, neurodegenerative disease that primarily affects young boys and causes irreversible, devastating neurologic decline, including major functional disabilities such as loss of communication, cortical blindness, requirement for tube feeding, total incontinence, wheelchair dependence, or complete loss of voluntary movement. According to a company press release, elivaldogene automecel is intended to slow the neurodegenerative progression of the disease.

According to the release, FDA based its approval on a Phase II/III study, ALD-102, which found that individuals who received the treatment had an estimated 72 percent likelihood of major functional disability (MFD)-free survival at 24 months from time of treatment. This was found to be a significant gain compared to untreated patients, who had an estimated 43 percent likelihood of MFD-free survival.

“Children with CALD and their families have been at the heart of bluebird’s mission since the company was founded more than a decade ago,” said Andrew Obenshain, CEO, bluebird bio, in the release. “This long-awaited approval represents significant hope and offers families a new option where, for many, there had been none. We are grateful to every individual who was involved in the development of [elivaldogene automecel] and are committed to working with providers and payers to make this important treatment option available to patients and their families.”

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FDA granted accelerated approval to Skysona (elivaldogene autotemcel), which is designed to slow the progression of neurologic dysfunction in adolescents with CALD.

FDA Approves Bluebird Bio’s $3 Million CALD Treatment

Lonza announced on Sept. 14, 2022 that it has expanded its Highly Potent API (HPAPI) multipurpose suite in Visp, Switzerland. The expansion adds development and manufacturing capacity for ADC payloads.

Lonza develops and produces all components of these therapies, including cytotoxic payloads, antibodies, and the required linkers. According to a company press release, the ADC market is expected to grow strongly, due to the growing demand for novel cancer therapeutics with higher targeting abilities. The expansion increased the capacity of the kilogram-scale HPAPI multipurpose manufacturing suite in anticipation for this growth.

The new HPAPI suite enables handling compounds with occupational exposure levels down to 1 ng/m3, with a variety of containment solutions and a flexible setup. On-site equipment includes reactors sized 1L to 50L with a temperature range of -80°C to 150°C isolation and drying equipment, and lyophilization and chromatography equipment.

“Lonza’s expanded multipurpose suite enables customers to meet the growing demand for targeted cancer treatments by developing and manufacturing ADC payloads with increased speed, capacity, and agility,” said Iwan Bertholjotti, senior director, commercial development and strategic marketing – bioconjugates, Lonza, in the release. “With this new fully integrated offer in Visp, we simplify the supply chain under one roof: from the production of an antibody to the chemical synthesis of complex payload-linkers and bioconjugation.”

Lonza has expanded its HPAPI multipurpose suite for payload-linker manufacturing. 

Lonza Expands HPAPI Multipurpose Suite

Quality Executive Partners (QxP), global management consulting and educational technology firm, announced on Sept. 20, 2022 that Boehringer Ingelheim has officially deployed its Virtuosi VR education platform for employee training. Boehringer Ingelheim is set to deploy the technology at three aseptic sites across its network.

Virtuosi is a cloud-based learning platform that incorporates 56 courses and 26 VR immersive experiences in more than 100 hours of educational content. According to a company press, its modules are self-paced and fully learning management software-integrated for reporting and evaluation. Additionally, learners receive real-time coaching via responsive formative evaluation, coaching, and haptic feedback.

"QxP is proud to partner with Boehringer Ingelheim, a leading research-driven biopharmaceutical company. Virtuosi is helping Boehringer Ingelheim to further advance the skills and knowledge necessary in sterile manufacturing using tech-forward innovation," said Crystal Mersh, CEO, QxP, in the release.

Boehringer Ingelheim will use Virtuosi to train employees at three aseptic sites across its network.