-European Commission (EC) has granted Orphan Drug Designation (ODD) to TransConTM C-Type Natriuretic Peptide (CNP) as a treatment for Achondroplasia (ACH).
VISEN Pharmaceuticals (“VISEN”) , 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 to provide more Chinese patients access as quickly as is safe and feasible to the world’s advanced treatment solutions. Ascendis Pharma A/S (Nasdaq: ASND) announced the European Commission (EC) has granted Orphan Designation to TransConTM C-Type Natriuretic Peptide (CNP) for the treatment of achondroplasia, the most common form of dwarfism1 on August 12, 2020. TransConTM CNP is an investigational long-acting prodrug of CNP, designed to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose. TransConTM CNP also received orphan designation for the treatment of achondroplasia in the United States (U.S.) in February 2019 from the FDA. Currently, there is no medical therapy approved for the treatment of achondroplasia2 .
“Ascendis Pharma now has orphan designations in Europe and the U.S. for TransConTM CNP in achondroplasia, which is of great significance for the treatment of achondroplasia and related complications in patients with achondroplasia,” said Dr. Jun Yang, CMO of VISEN. “At present, there is no effective etiological treatment for this disease. The accelerated development of TransConTM CNP brings patients hope. VISEN Pharma has entered the preparation stage of the global observational study ACHieve and phase 2 ACcomplisH China Trial , and strives to offer a new treatment option for patients with achondroplasia as soon as possible.”
Orphan Designation in the European Union (EU) is granted to therapies aimed at the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating, affects no more than five in 10,000 persons and for which no satisfactory therapy is available. The medicine also must be expected to provide significant benefit to those affected by the condition. Orphan medicines have 10 years of market exclusivity after they receive marketing authorization in the EU. Under certain conditions, market exclusivity for pediatric indications may be extended for an additional two years.
“Although the incidence of ACH is low in China, the mortality is high in infants.Patients suffer from impaired chondrogenesis and endochondral bone formation, which lead to a variety of severe skeletal complications and comorbidities at different ages,” said Pony Lu, CEO of VISEN. “Due to the lack of effective drug treatments, many patients bear enormous physical and psychological pressure throughout their lives. This demand for treatment needs to be solved urgently. Thus, EC’s designation for TransConTM CNP as an orphan drug has great significance for ACH treatment. As the company with exclusive authorization to develop and commercialize TransConTM CNP in Greater China, VISEN plans to take part in conducting the clinical trial in China. We look forward to introducing the new treatment to Chinese patients as early as possible.”
About TransConTM Technology
TransConTM refers to “transient conjugation.” The proprietary TransConTM platform is an innovative technology designed to create new therapies that 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 (e.g., 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 may be maintained. TransConTM technology is designed to be applied broadly to a protein, peptide or small molecule in multiple therapeutic areas, and to be used systemically or locally.
Achondroplasia is the most common form of dwarfism, affecting approximately 250,000 people worldwide. Individuals living with achondroplasia may experience severe skeletal complications and comorbidities. For example, abnormal skeletal development can lead to sleep apnea, chronic back and leg pain from lower spine impingement and sudden infant death from cervical compression. Chronic ear infections due to abnormal eustachian tubes can lead to hearing loss and speech delay3 .
The condition is caused by an autosomal dominant activating mutation in the fibroblast growth factor receptor 3 (FGFR3) gene that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways. Preclinical and clinical data show that the CNP pathway stimulates growth. Increased CNP counteracts the effects of the FGFR3 mutation downstream, thus promoting bone growth.1
About VISEN Pharmaceuticals
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 world's most 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.
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