Setmelanotide 塞美拉肽 MC4R激动剂 遗传肥胖/罕见病 API ≥98%

Molecular Formula

C50H68N14O10

Molecular Weight

1025.17 g/mol

Sequence

Ac-Arg-Cys(1)-D-Ala-His-D-Phe-Arg-Trp-Cys(1)-NH2 (cyclic disulfide: Cys2-Cys8)

Appearance

White to off-white lyophilized powder

Solubility

Soluble in water and aqueous buffers (pH 4-6)

Storage

Store at -20°C, protected from light and moisture

Pharmacological Class

Melanocortin-4 receptor (MC4R) agonist

Indication

Chronic weight management in rare genetic disorders of obesity (POMC, PCSK1, LEPR deficiencies)

Administration Route

Subcutaneous (SC) injection once daily

Purity

>= 98.0% (by HPLC)

First FDA-Approved MC4R Agonist 首个FDA批准MC4R激动剂

First and only FDA-approved therapy targeting melanocortin-4 receptor for rare genetic obesity disorders, including POMC, PCSK1, and LEPR deficiencies. 首个也是唯一一个靶向黑皮质素-4受体治疗罕见遗传性肥胖症的FDA批准疗法，包括POMC、PCSK1和LEPR缺乏症。

Pioneering position in the rapidly expanding market for precision obesity therapeutics with strong orphan drug exclusivity. 在快速扩张的精准肥胖治疗市场中占据开创性地位，享有强有力孤儿药独占权。

Precision Obesity Targeting 精准肥胖靶向

Restores MC4R pathway function in patients with genetic defects upstream of the melanocortin circuit, regulating appetite and energy expenditure. 修复黑色素皮质素通路上游遗传缺陷患者的MC4R通路功能，调节食欲和能量消耗。

Dramatic and sustained weight reduction demonstrated in clinical trials - patients achieved 23.1% mean weight loss at one year. 临床试验显示显著且持续的体重减轻——患者在一年内平均减重23.1%。

Cyclic Peptide Stability 环肽稳定性

Engineered cyclic disulfide structure provides enhanced metabolic stability and receptor selectivity compared to linear alpha-MSH analogs. 工程化环状二硫键结构相比线性α-MSH类似物提供增强的代谢稳定性和受体选择性。

Superior pharmacokinetic profile with once-daily subcutaneous dosing, improving patient compliance and commercial viability. 优越的药代动力学特征，每日一次皮下给药，提高患者依从性和商业可行性。

Rare Disease Premium Pricing 罕见病溢价定价

Orphan drug designation with targeted indication in ultra-rare genetic obesity populations creates favorable pricing dynamics. 孤儿药资格认定，针对超罕见遗传性肥胖人群的靶向适应症创造有利的定价动态。

Attractive commercial opportunity for specialty pharmaceutical companies focused on rare disease and metabolic therapeutics. 对专注于罕见病和代谢治疗的专业制药公司具有吸引力的商业机会。

Validated Clinical & Regulatory 经验证的临床和法规

Robust Phase III clinical data package with established efficacy and safety profile; FDA-approved and EMA-authorized. 强有力的III期临床数据包，确立的疗效和安全性特征；已获FDA批准和EMA授权。

De-risked regulatory pathway for international registrations, with reference to existing approved drug dossier. 利用已获批药物文件，国际注册的风险降低。

GMP Bulk Supply GMP散装供应

Multi-kilogram GMP manufacturing capacity with validated processes, comprehensive stability data, and full regulatory documentation. 多公斤GMP生产能力，经验证的生产工艺、全面的稳定性数据和完整的法规文件。

Supports commercial launch scale-up and international market expansion with reliable supply chain and consistent quality. 支持商业上市规模化和国际市场扩张，供应链可靠，质量一致。

Full-Service Procurement 全服务采购

End-to-end supply chain offering API, reference standards, analytical methods, and regulatory submission support. 端到端供应链，提供API、对照品、分析方法和法规申报支持。

Streamlines product development for formulation partners entering the metabolic and rare disease therapeutic space. 简化进入代谢和罕见病治疗领域的制剂合作伙伴的产品开发流程。