On April 21, 2020, VISEN Pharma, a joint venture company dedicated to the endocrine-related treatment field, introduced the world's leading treatment methods and drugs to China, and was responsible for the endocrine treatment program of Ascendis Pharma in Greater China Development and promotion. Ascendis Pharma announced on April 15, 2020 that the US Food and Drug Administration (FDA) has granted TransCon Human Growth Hormone (lonapegsomatropin) orphan drug designation to treat growth hormone deficiency (GHD).
TransCon Human Growth Hormone is a research long-acting prodrug of growth hormone, which has been developed into a weekly GHD treatment drug. TransCon Human Growth Hormone maintains the same mode of action as daily human growth hormone treatment by releasing the same growth hormone molecules. Currently, there is no approved long-acting growth hormone therapy in the US or European market. TransCon Human Growth Hormone has been certified by the European Commission (EC) for the treatment of GHD in Europe in October 2019.
"TransCon Human Growth Hormone is now certified as an orphan drug in the United States and Europe. We believe this is an important affirmation that the world needs a long-acting therapy to deal with GHD and all endocrine health problems." Ascendis Pharma Senior Deputy President Dana Pizzuti, MD, said: "As the only long-acting growth hormone product in development that provides unmodified growth hormone, we believe that TransCon Human Growth Hormone has great potential to improve the lives of patients. We are still expected to be second in this year as planned Quarterly and fourth quarter submission of marketing applications for TransCon Human Growth Hormone in the US and Europe. "
Drugs approved by the FDA for orphan drugs refer to those that can safely, effectively treat, diagnose, or prevent rare diseases or conditions affecting less than 200,000 US citizens, and may be safer or more effective than approved products. The orphan drug designation provides certain benefits and incentives for drug developers, including a seven-year US market monopoly period from the date of market authorization, exemption from FDA user fees and tax credits for clinical research. Granting orphan drug designations will not change FDA ’s regulatory requirements to determine the safety and effectiveness of drugs through appropriate and well-controlled research to support approval and commercialization.
Lu Anbang, CEO of Weisheng Pharmaceutical, pointed out: "At present, more than 90% of rare diseases lack effective treatment. The approval of TransCon Human Growth Hormone by the US FDA orphan drug is of great significance for the treatment of growth hormone deficiency. The weekly TransCon Human Growth Hormone can reduce the burden of daily injections, giving patients a better chance of achieving normal adult height and overall endocrine health. Currently, TransCon Human Growth Hormone is used in China's Phase III clinical trial of childhood growth hormone deficiency The research has started at the end of 2019. As the exclusive authorized R & D and sales company of TransCon Human Growth Hormone in Greater China, Weisheng Pharmaceutical will fully promote the listing plan in China in the future and is committed to introducing the world's leading treatment methods and drugs China, hopes to allow more Chinese patients to benefit from the world's advanced and reliable treatment options earlier. "
About TransConTM technology
TransCon refers to "transient connection" (Transient Conjugation). The TransCon technology platform develops new treatments through innovative technologies to optimize treatment effects including effectiveness, safety, and frequency of administration.
The TransCon molecule consists of three parts: an unmodified prototype drug, an inert carrier molecule that protects the prototype drug, and a connection structure that temporarily connects the two. When the three parts are combined, the carrier molecule can inactivate the prototype drug and not be cleared by the body. When injected into the human body, under physiological conditions of pH and body temperature, the active, unmodified prototype drug will be released in a controlled manner. Since the prototype drug is unmodified, it can maintain its original pharmacological mechanism. TransCon technology can be widely applied to proteins, peptides or small molecules in various therapeutic fields, and can be applied systemically or locally.