FDA Approves Orphan Drug ATryn, First Biologic Developed in Transgenic Animals
The US Food and Drug Administration issued its first approval on February 6 for a biological product produced by genetically engineered animals.
The US Food and Drug Administration issued its first approval on February 6 for a biological product produced by genetically engineered animals.
ATryn, manufactured by GTC Biotherapeutics, Inc. (Framingham, MA), is a therapeutic protein derived from the milk of genetically engineered goats milk. Antithrombin is a protein that naturally occurs in healthy individual and helps to keep blood from clotting in the veins and arteries.
GTC received approvals from two FDA centers. The Center for Biologics Evaluation and Research (CBER) approved the human biologic based on its safety and efficacy, and the Center for Veterinary Medicine (CVM) approved the rDNA construct in the goats that produce ATryn.
ATryn is used for the prevention of blood clots in patients with a rare disease known as hereditary antithrombin (AT) deficiency. These patients are at high risk of blood clots during medical interventions, such as surgery, and before, during, and after childbirth.
Because hereditary AT deficiency occurs in a small population (approximately 1 in 5,000 people in the US), the FDA granted ATryn an orphan drug designation. The orphan drug designation system encourages the development of medications for patients with a rare disease or condition.
A summary of the information on which the FDA made its approval decision for the rDNA construct in the goats, and CVM’s guidance on the regulation of GE animals containing heritable rDNA constructs are available at http://www.fda.gov/cvm/GEAnimals.htm.
ATryn previously received approval from the European Medicines Agency for use in preventing clotting conditions during surgical procedures in patients with hereditary AT deficiency.
The Future of Cleanroom Construction: How Hybrid Solutions are Changing the Game
May 14th 2025Imagine a world where cleanroom facilities—essential for pharmaceutical manufacturing, biotechnology, and high-tech industries—are built with unparalleled speed, precision, and efficiency. That world is here, thanks to the hybrid construction approach. By blending traditional stick-built methods with modular and prefabricated solutions, companies are overcoming the limitations of conventional construction while ensuring compliance with stringent industry regulations. In this interview, we explore how hybrid cleanroom construction is transforming the industry, offering faster project timelines, improved quality control, and significant cost advantages. Join us as we delve into this game-changing approach with industry experts who are leading the charge in revolutionizing cleanroom infrastructure.
Mastering Antibody-Drug Conjugates
December 19th 2024In this episode, we explore BIOVECTRA’s capabilities in antibody-drug conjugate (ADC) manufacturing, from complex conjugation chemistry to synthesis of highly potent payloads. We’ll also showcase how BIOVECTRA’s extensive experience in complex chemistries and specialized small molecule manufacturing gives them a unique perspective, strengthening their approach to ADC production and ensuring clients receive custom solutions across all project stages.
