A regulatory starting material is a chemical compound that marks the point in the synthesis of an active pharmaceutical ingredient (API) where current Good Manufacturing Practices (cGMP) begin. It is formally designated in regulatory submissions and must be scientifically justified based on its impact on the quality and safety of the final drug product. 

FDA’s Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) program was established in 2012 as a financial incentive to encourage the development of therapeutics for rare pediatric diseases. The program officially ended as of September 30, 2024 due to a sunset clause. 

On December 15, 2025, the FDA published the final guidance on the “Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices” Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices  Dr. Makary said that the FDA is, “removing unnecessary barriers that have prevented us from using powerful real-world evidence to get life-changing treatments to patients faster.”

An overview of the key regulatory intelligence updates from FDA & EMA released in 2025.

FDA’s draft guidance streamlines biosimilar approval by reducing the need for comparative efficacy studies (CES). The guidance provides revised criteria outlining the circumstances in which conducting CES may be unnecessary for a Biologics License Application submitted through the 351(k) biosimilar and interchangeable biosimilar pathway.

This November, Scendea had the pleasure of attending BIO-Europe Autumn 2025 in Vienna, Austria. The focus of BIO-Europe Autumn 2025 was gene therapy, precision medicine, funding strategies and challenges across the industry. 

When market protection ends, it triggers a shift in the competitive landscape for a product and often has a significant effect on the originator company which can see an overnight loss in revenue and potential biosimilar manufacturers. There are generally two types of market protection.

Eight CAR-T products have been approved in the US thus far, and CAR-T products account for approximately 55% of all Gene Therapy INDs. A combination of factors, including manufacturing challenges, clinical development timelines and differences in regional regulatory requirements, complicate the development of CAR-T products.

The 8^th annual symposium for Life Science Innovation and Development was a day packed with timely topics for small and emerging biotech companies. As the welcome meeting highlighted, “With private investment still stalled and changes in federal funding along with tariffs and job reductions, the challenges for the industry have never been more serious”. 

The FDA has announced a new review process for rare
diseases based on a molecule’s “plausible mechanism”
which would allow the FDA to consider approval on a conditional basis. This initiative is designed to help speed up the approval of drugs to treat rare diseases and provide clear guidance on how evidence of efficacy can be demonstrated with a small patient population.

A prefilled syringe (PFS) is a disposable syringe that comes preloaded with a specific dose of medication, ready for injection. This design simplifies the process of administering injectable drugs, as it eliminates the need to draw medication from a vial into a syringe before use.

Vaccines are defined as medicinal products intended for prevention, post-exposure prophylaxis and/or treatment of disease caused by an infectious agent and which contain antigen(s) or genetic information for an antigen(s), either of biological or synthetic nature, that induce a specific immune response against the causative infectious agent(s) or its toxins (EMA 2023). 

On the 17th of June 2025 the FDA Commissioner Marty Makary announced a new initiative aimed at enhancing the health interests of the United States (US) population. The Commissioner’s National Priority Voucher (CNPV) will be granted to US companies developing drugs that are: Addressing a health crisis in the US, Delivering more innovative cures for the American people, Addressing unmet public health needs, Increasing domestic drug manufacturing as a national security issue.

Biotech companies, who are looking to secure investment to enable development of their medicinal products, must navigate a competitive funding landscape while optimizing their fundraising strategies. To de-risk and support fundraising strategies globally it’s imperative that product development and regulatory strategy are carefully considered and enacted upon.

In August 2024, the US Food and Drug Administration (FDA) released new guidance for the Project Optimus initiative, titled “Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases”.