Alcami recently contributed to a special feature in the August / September issue of Chemicals Knowledge magazine, titled "Considerations for Approval of Biosimilars."

Biosimilars are sometimes incorrectly considered equivalent to a generic small molecule therapeutic, but for a biologic drug product, the clinical and regulatory pathways are quite different. Alcami Commercial Development Manager Cheryl Johnson explains the distinct differences in the FDA’s definition, development strategy, and regulatory approval pathways.

Alcami recently contributed to an eBook produced by Drug Development and Delivery. The eBook, titled "Analytical Testing in Drug Development," features four companies and explains how they each help the pharmaceutical and biotech industries comply with drug development regulatory requirements. 

Angela Moore, abuse-deterrence studies subject matter expert at Alcami, contributed to the eBook. Her article focuses on considerations for abuse-deterrent Category 1 studies as well as standard syringeability studies.

Since Joseph Juran’s concept of Quality by Design (QbD) was first adopted by the US Food and Drug Administration (FDA), the pharmaceutical industry has been interpreting its concepts into a variety of approaches for implementation. At Alcami, our QbD approach considers evolving global regulatory guidances with a collaborative fit-for-purpose and pragmatic approach that can evolve throughout the lifecycle of the product. QbD starts from day one in modern product development, culminating in a chemistry manufacturing controls (CMC) package that maximizes every opportunity for a successful regulatory review and approval. The value of this approach is simple: design of robust processes for the consistent production of safe and effective products for our patients and long term cost savings for our partners through improved yields, reduced deviations, and efficient change management.

The QbD process for product development is intended to build quality into pharmaceuticals based on characteristics that define safety and efficacy. Critical quality attributes (CQAs) – specific physical, chemical, biological, and other properties – may impact product safety and efficacy and can be managed by identifying critical process parameters (CPPs) through the use of risk management tools and controlling them within the proven acceptable process design space.

Abeona Therapeutics, Inc. is a fully-integrated gene and cell therapy company at the forefront of the rapidly-advancing field of genetic medicine. Their late-stage candidate, EB-101, is an autologous, ex-vivo, gene-corrected cell therapy poised to enter a Phase 3 clinical trial for Recessive Dystrophic Epidermolysis Bullosa (RDEB).

Read this case study to learn how Alcami's extensive experience with small and large molecule development services, regulatory compliance, and specialty techniques helped

Supporting a compound from discovery to clinical phases and potentially commercial phases requires an immense amount of time, energy, and money. Regulatory management is paramount during the drug development pipeline funnel, and recognizing Current Good Manufacturing Practices (CGMPs) is the foundation of drug safety and efficacy.

Every country has its own regulatory authority, responsible for enforcing rules and regulations and issuing guidelines for drug development, licensing, registration, manufacturing, marketing, and labeling of pharmaceutical products.^[1]

Alcami recently contributed to a special feature in Drug Development and Delivery magazine titled "Designing a CMC Strategy for Accelerated Development of Biotherapeutics."

As the pipeline of biologic drugs intended to treat complex disease expands, the typical timeline for drug development seems to be shortening more frequently and with more intensity. There are many drivers to accelerate development.

Alcami’s St. Louis, Missouri Center of Excellence for advanced analytical testing was issued a Certificate of Compliance by the European Medicines Agency (EMA) Tuesday, April 23, 2019, following the facility’s recent successful Good Manufacturing Practice (GMP) inspection. Certification confirms the site is compliant with GMP laboratory storage and testing practices, and enables the laboratory to process more commercial products for the European Union (EU). The outcome reflects the company’s unwavering commitments to quality, reliability, responsible operations, and strict adherence to regulatory compliance.

The new elemental impurities regulations stress the importance of controlling all sources of elemental impurities in drug products. The regulations also impact all upcoming and current commercial drug products for human use. To comply with these regulations, it is critical to have an efficient, cost-effective, and agency-accepted approach.

Elemental impurities can contaminate or leach into a drug product from multiple sources (i.e. drug substance, excipients, water, equipment, and container closure systems), therefore, it is no longer acceptable to monitor the inlet raw materials only. A conservative risk-based approach is recommended, which should address all potential sources of elemental impurities. If it can be demonstrated that the elemental impurities present in the drug product are under control (less than the control threshold for three lots), then analysis at release is not needed. 

The US Orphan Drug Act of 1983 characterizes an orphan disease as a rare medical condition that affects a population of fewer than 200,000 people. Rare diseases have various causes— most notably, genetics. However, the National Institutes of Health (NIH) states, “many rare diseases, including infections, some rare cancers, and some autoimmune diseases, are not inherited. While researchers are learning more each year, the exact cause of many rare diseases is still unknown” (NIH, 2017).

The term “orphan disease” resulted from pharmaceutical companies not having the resources to design and scale up formulations to treat these diseases, due to the lack of financial support for such highly specialized programs. The Orphan Drug Act (ODA) allows for the necessary financial backing of pharmaceutical companies to develop treatments for rare diseases (NIH, 2017), giving special status to a drug or biological product (drug) upon request of a sponsor. This status is referred to as orphan designation or orphan status.

Many pharmaceutical manufacturers are concerned about label design and placement to accommodate a serialized label. These are, of course, very legitimate concerns. However, there are much more complex issues at hand, including the importance of standardization, scaling information technology infrastructure, agility to accommodate regulatory changes, and data security.

Why Serialization Matters
The first objective of any pharmaceutical company is to deliver safe and effective medicines to patients. While historically there have been methods to provide product visibility and control along the supply chain, the processes have been highly manual, with paper-based record keeping being the prevalent modality.