In recent years 505(b)(2) approvals have experienced double-digit growth. Many companies are electing to pursue 505(b)(2) applications to obtain a new drug approval (NDA). A 505(b)(2) application is a hybrid between the FDA's traditional NDA and an abbreviated new drug application (ANDA). These submission types were made possible in 1984 by the adoption of the Hatch-Waxman Act. The act was created to allow for generic competition and increased innovation in the pharmaceutical arena. A true generic or ANDA demonstrates that the drug is identical to an already approved product based on bioequivalence data and chemical equivalence. 

Signed into law on January 4, 1983, the Orphan Drug Act (ODA) allows for granting special status to a drug or biological product (drug) to treat a rare disease or condition upon request of a sponsor. This status is referred to as orphan designation, or orphan status. For a drug to qualify for orphan designation, both the drug and the disease or condition must meet certain criteria specified in the ODA and US Food and Drug Administration’s (FDA) implementing regulations per 21 CFR Part 316. The FDA Office of Orphan Products Development (OOPD) evaluates the scientific and clinical data submission from sponsors to identify and designate products as promising for rare diseases or conditions.

The pharmaceutical product landscape is rapidly and fundamentally changing with profound impact on research and development strategy and technology needs. After a precipitous fall in new drug approvals during the last two decade of the 20th century, there has been a gradual recovery going into the second decade of the 21st century.Figures 1 and 2 map drug approvals by molecule type and route, excerpted from the publication by Ouyang et al¹ based on 34,673 drug products approved by the FDA over a 37-year period from 1980 through 2017. The number of biopharmaceutical products show a considerable growth accounting for nearly a quarter of the new molecular entities approved in 2017. While oral delivery route (62%) is still the most prevalent amongst marketed drug products, alternative adminstrations such as injection (22%), cutaneous, mucosal, inhalation, and others are making up a greater percentage as the industry works to improve bioavailability and subsequent efficacy.

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 helpedensure the quality, safety, and efficacy of raw materials used in Abeona's EB-101 manufacturing batches.

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.