Recent Facet Successes – Just a Few Highlights

Facet Helps Small Biologics Company Secure Breakthrough Designation on Phase 2 Data

Facet assisted a sponsor in successfully obtaining Breakthrough Therapy Designation for their gene therapy product. In conjunction with the Breakthrough Therapy Designation (BTD), Facet helped the company discuss the results of a Phase 2 study with FDA in a formal meeting.  Subsequently, Facet worked with the company to develop a proposal for the submission of a BLA based on the results of the Phase 2 study as pivotal and multiple ex-US clinical studies as supportive of the efficacy and safety of the product. If successful, the plan will allow the company to save several years of development time and seek approval for the product for treatment of a serious unmet medical need.

Facet Helps Company Establish Strategic Plan to Increase Product Sales

Facet helped a small commercial-stage company evaluate issues associated with sluggish market uptake of their recently approved product. Through a careful landscape and market analysis, Facet and the company identified several limitations of the current product distribution model and brainstormed alternatives. Then, Facet pressure tested each alternative by contacting medical practices, insurance companies, and pharmacies to evaluate the market viability of each option. Plus, Facet evaluated the regulatory implications of each approach, the tactical considerations for changing the distribution model, and the estimated regulatory costs associated with each approach. Facet also recommended the company seek federal and local government feedback and legal counsel prior to making their final decision. Ultimately, Facet provided the company with rank-ordered recommendations based on estimated probabilities of overall success. This integrated approach was instrumental in understanding the multidimensional nature of the product distribution issue and provided the company with actionable guidance to increase product sales, physician awareness, and patient access.

Industry Focus

Update on BIOSECURE Act

On September 9, 2024, the House of Representatives passed the BioSecure Act, aiming to prevent the U.S. government from obtaining services or equipment from biotechnology companies associated with foreign adversaries that pose national security threats, such as China, Cuba, Iran, North Korea, and Russia. The BioSecure Act specifically identifies companies like BGI, MGI, Complete Genomics, WuXi AppTech, and WuXi Biologics but allows for additional companies to be listed in the future. It also prohibits federal grant recipients from purchasing equipment from these companies. If enacted, the BioSecure Act will restrict the use of listed biotechnology companies in the U.S., impacting the development and marketing of drugs, biologics, and medical devices. Read the full article: Update on BIOSECURE Act (September 2024)

Gene Therapies in the Spotlight

A Major Trend in 2024

Cellular and gene therapies are a major trend in 2024, offering the potential to revolutionize healthcare with treatments for genetic disorders and rare diseases. Over 4,000 such therapies are in development worldwide, with FDA already approving 38 products and overseeing 1,500 ongoing clinical trials. To support this growth, FDA has reorganized its resources under the new Office of Therapeutic Products (OTP). Additionally, FDA offers the Regenerative Medicine Advanced Therapy (RMAT) designation to expedite development for promising therapies. Facet Life Sciences is actively helping developers navigate this evolving regulatory landscape and advance innovative treatments. Read the full article: Gene Therapies in the Spotlight – A Major Trend in 2024

FDA Roundup

Director Jeff Shuren, CDRH Retires

On July 23, 2024, Jeff Shuren, Director of the Center for Devices and Radiological Health (CDRH) at FDA announced his intent to retire from FDA. During his tenure at FDA, Dr. Shuren was instrumental in helping lead innovation and regulatory modernization efforts within CDRH.

Dr. Michelle Tarver is currently serving as acting Director of CDRH.  She is board-certified in ophthalmology and holds a doctorate in epidemiology from Johns Hopkins University School of Medicine and Bloomberg School of Public Health. Read the full article: Director Jeff Shuren, CDRH Retires

New FDA Guidance: Control of Nitrosamine Impurities in Human Drugs (September 2024)

FDA has released Revision 2 of its guidance for the control of nitrosamine impurities in human drugs. Nitrosamines are potent genotoxins, and some are classified as probable or possible human carcinogens. FDA first began investigating these impurities in 2018, starting with the discovery of N-nitrosodimethylamine (NDMA) in valsartan. The updated guidance offers recommendations on root causes, sources, and strategies for mitigating and controlling nitrosamines, including a new focus on nitrosamine drug substance-related impurities (NDSRIs). Facet Life Sciences has helped organizations develop strategies for managing these impurities in their products.  Read more on the website at: Control of Nitrosamine Impurities in Human Drugs (September 2024)

New Draft FDA Guidance: Chemical Analysis for Biocompatibility Assessment of Medical Devices (September 20, 2024)

FDA has issued draft guidance on chemical analysis for biocompatibility assessment of medical devices. This guidance helps manufacturers assess the biocompatibility of devices or components that come into contact with the human body as part of premarket submissions. While in vitro and in vivo testing are commonly used, chemical characterization is presented as an alternative or complement to biological testing, potentially reducing animal testing. The guidance covers approaches to chemical characterization, including extraction, analysis, and reporting methodologies. Facet Life Sciences is currently assisting Sponsors with biocompatibility testing strategies. Read the full article: New Draft FDA Guidance: Chemical Analysis for Biocompatibility Assessment of Medical Devices (September 2024)

In Case You Missed It

Starting October 1, 2025, all De Novo Classification Requests, unless exempted, must be submitted as electronic submissions using the electronic Submission Template (eSTAR). The eSTAR is an interactive PDF that helps device Sponsors prepare and submit comprehensive and complete applications and information to FDA. Facet has made many eSTAR submissions to FDA. Ask us how we can help you prepare and submit a complete and compliant eSTAR for your device to speed your product to clearance or approval!

Facet by the Numbers

Total ESG submissions to FDA July – September 2024: Over 37!

Early-Stage Development Highlights

  • Over 5 formal FDA meetings and 2 breakthrough meetings
  • Over 28 IND amendments (e.g., new protocols/amendments, CMC updates, nonclinical study reports, information amendments, IND annual reports, orphan designation annual reports, safety reporting/DSURs)
  • Prepared several gap analyses and regulatory development plans
  • Ongoing review of public-facing information (e.g., websites, flyers, conference presentations) for investigational products
  • Responded to numerous FDA information requests
  • Provided regulatory strategy and guidance for FDA meeting planning
  • Provided IND strategy and planning
  • Secured 2 breakthrough designations
  • Secured 1 orphan designation
  • Secured 1 rare pediatric disease designation
  • Performed several clinical trial simulations to improve trial design and probability of success.

Late-Stage Development Highlights

  • Late-stage CMC meeting with FDA
  • Completed 1 FDA meeting clarification request
  • Prepared several gap analyses
  • Phase 1, 2, and 3 clinical regulatory strategies
  • Phase 3 clinical trial oversight (i.e., independent validation of tables, listings, figures, and SDTM/ADaM data packages)
  • Programmatic data verification to enable efficient site monitoring and query generation/resolution
  • Clinical trial safety reporting
  • Used statistical modeling and simulations to design Phase 3 clinical trials
  • Supported registration batch manufacturing
  • Human factors study design recommendations
  • Medical device development strategy for over 3 products
  • Active regulatory submission planning for 3 NDAs/BLAs
  • Designed and authored several nonclinical protocols, clinical protocols, statistical analysis plans, and clinical study reports
  • Maintained CT.gov postings
  • Actively engaged in investment/funding partnership support
  • Expanded access program support and website content
  • Ongoing pre-approval ad promo strategy and content review
  • Pre-approval commercial landscape assessment and marketing evaluation.

Post-Approval and Commercial Highlights

  • Several NDA CMC supplements
  • Post-approval labeling supplement
  • Post-marketing requirement and commitment resolution
  • Ongoing advertising and promotional review
  • Commercial landscape assessment and marketing evaluation

Investor Support Pack for Companies Seeking Funding

A common problem for early life sciences organizations is how to attract and secure investment to support product development.  Investors know that product development is time-consuming and expensive. They also know that most products fail in development. As such, investors look for sound organizations that are developing products that have a high probability producing a favorable return on their investment. So, how do you demonstrate that your company and product is worthy of investment? At Facet, we help early life sciences organizations prepare an Investor Support Pack that summarizes the available scientific data, provides a high-level regulatory plan, and outlines the commercial market assessment and strong business case for the product. Talk to Facet today about how the Investor Support Pack can help you secure investment!

Facet Team News

Facet Presentation on Orphan Drug Development

Dr. Jason Mercer is giving a talk on Nov 14, 2024 at the North Carolina Regulatory Affairs Forum (NCRAF) round table dinner event.  The title of his talk is, “Orphan Product Development”. All are welcome to register and attend on location. The meeting will be held at Apella by Alexandria, 12 Davis Drive, Durham, NC 27709.

Facet Attendance at Oncology-Focused Lunch and Learn

On October 1, 2024 Dr. Venita DePuy attended a Lunch & Learn at the NC Biotechnology Center, focused on FDA’s Expectations for Drug Approval, dose optimization, accelerated approval, and adaptive designs for non-cytotoxic oncology therapeutic products. The presenters discussed different methodologies for finding the optimal dose including the Bayesian Optimal Interval (BOIN) approach and efficacy-toxicity utility contours. Recommendations included using these methods to allow for adaptive dosing with fewer subjects treated at a subtherapeutic dose than the traditional 3+3 design based on dose-limiting toxicities.

We’re Hiring

At Facet, our mission is to make life sciences dreams a reality.  We are looking for a regulatory affairs team leader and lead CMC regulatory strategists who want to use their knowledge and experience to help small organizations get their innovative products from concept into development, through FDA, and on to US commercial launch.

For over 10 years, Facet has leveraged our strong scientific, development, and regulatory expertise to help small organizations reach successful outcomes, from product concept to FDA approval.  We love to tell a good story and great science makes for a great story.

Come help us create great success stories – both for our customers and for you! Join our Team

About Facet Life Sciences

Facet’s strategic and tactical regulatory affairs, statistics, regulatory writing, and commercialization experts lead small organizations to faster and more efficient product development, US registration, and commercial launch.