TransConTM hGH Received Orphan Drug Designation in the US and EC as Treatment for Growth Hormone Deficiency

2020-04-21

TransConTM hGH (lonapegsomatropin) now has orphan designation in both the U.S. and Europe for the treatment of growth hormone deficiency (GHD)

【SHANGHAI, China, Apr 20, 2020】VISEN Pharma developing and commercializing through multiple license agreements Ascendis Pharma’s endocrinology rare disease therapies in Greater China, hailed the move and pledged to accelerate the drug’s launch in China, as part of its mission provide more Chinese patients quick access to advanced and reliable treatment solutions. On Apr 15, 2020, Ascendis Pharma A/S (Nasdaq: ASND) announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to TransConTM hGH (lonapegsomatropin), as a treatment for growth hormone deficiency (GHD).

TransConTM hGH is an investigational long-acting prodrug of somatropin (human growth hormone or hGH) that is being developed as a once-weekly treatment for GHD. TransConTM hGH is designed to maintain the same mode of action as daily hGH therapies by releasing the same growth hormone molecule, somatropin. Currently, there is no approved long-acting growth hormone treatment on the market in the U.S. or Europe. TransConTM hGH received Orphan Designation (OD) for the treatment of GHD in Europe from the European Commission (EC) in October 2019.

“TransConTM hGH has now been granted orphan designation in both the U.S. and Europe, which we believe is an important acknowledgement of the global need for a long-acting therapy to address GHD and overall endocrine health,” said Dana Pizzuti, M.D., Ascendis Pharma’s Senior Vice President of Development Operations. “As the only long-acting growth hormone product in development that delivers somatropin, we believe TransConTM hGH has significant potential to improve patients’ lives. We remain on track to file our marketing applications for TransConTM hGH in the U.S. and Europe, as planned, in the second and fourth quarters of this year, respectively.”

The FDA grants orphan designation to drugs that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States, and potentially may be safer or more effective than already approved products. Orphan designation provides a drug developer with certain benefits and incentives, including a seven-year period of U.S. marketing exclusivity from the date of marketing authorization, waiver of FDA user fees, and tax credits for clinical research. The granting of orphan designation does not alter the FDA’s regulatory requirements to establish safety and effectiveness of a drug through adequate and well-controlled studies to support approval and commercialization.

“At a time when over 90% of rare diseases lack effective treatment, receiving Orphan Drug Designation (ODD) from the FDA is a major step forward for TransConTM hGH in the treatment of GHD,” said Pony Lu, CEO of VISEN Pharma. “Once-weekly dosing can reduce the burden of daily injections, giving patients a better chance of achieving general adult height and improved overall endocrine health. In late 2019, VISEN Pharma launched the TransConTM hGH China Phase 3 study for the treatment of pediatric growth hormone deficiency (PGHD). As the company with exclusive authorization to develop and commercialize TransConTM hGH in Greater China, VISEN will speed up the launch of TransConTM hGH in China, as we continue our commitment to the treatment of endocrine-related diseases. We look forward to introducing leading treatment methods and drugs to the China market as we provide more Chinese patients quick access to advanced and reliable treatment solutions.”

About TransConTM Technology

TransConTM refers to “transient conjugation.” The proprietary TransConTM platform is an innovative technology to create new therapies designed to optimize therapeutic effect, including efficacy, safety and dosing frequency. TransConTM molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (i.e., pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action is expected to be maintained. TransConTM technology can be applied broadly to a protein, peptide or a small molecule in multiple therapeutic areas, and can be used systemically or locally.

About VISEN Pharma

VISEN Pharmaceuticals is committed to the treatment of endocrine-related diseases, introducing the world’s leading treatment methods and drugs into the China market and hoping to provide more Chinese patients quick access to the advanced and reliable treatment solutions.

In 2018, VISEN Pharmaceuticals (VISEN) was formed by Ascendis Pharma A/S (Nasdaq: ASND) and an investor syndicate led by Vivo Capital (along with participation by Sofinnova Ventures), to develop and commercialize Ascendis Pharma’s endocrinology rare disease therapies in Greater China, which includes mainland China, Hong Kong, Macau, and Taiwan.

Ascendis Pharma, headquartered in Copenhagen, Denmark with offices in Heidelberg, Germany and Palo Alto, California, is a leading fully integrated biopharmaceutical company that uses its innovative platform technology to make a real difference to patients’ lives. Guided by its core values of patients, science and passion, the company uses its TransConTM technologies to create new, potentially best-in-class therapies.

Founded in 1996, Vivo Capital is a global healthcare investment firm focused on investing in and building high quality companies. Vivo Capital is headquartered in Palo Alto, California, with offices in Beijing, Shanghai, Hong Kong, and Taipei. 

VISEN Pharmaceuticals is headquartered in Shanghai, and its main business centers in mainland China are in Beijing and Shanghai.