Genentech’s Zelboraf® (vemurafenib) is a small molecule B-RAF inhibitor and is FDA-approved to treat patients with metastatic or unresectable melanoma whose tumors express the B-RAF V600E mutation. To reduce the likelihood of cancer cells becoming drug resistant, Genentech wanted to combine vemurafenib with cobimetinib, a small molecule MEK inhibitor that targeted a different part of … Continued
In 2020, the US FDA approved Risdiplam (Evrysdi®) as the first orally administered drug to treat SMA in patients ≥2 months old, followed by the European Medicine Agency. Risdiplam addresses the underlying cause of SMA: a reduced amount of survival motor neuron (SMN) protein.
非典型溶血性尿毒症综合征（aHUS）是一种极其罕见的遗传疾病，会导致全身小血管内异常血凝块的形成，从而导致肾衰竭、其他器官损伤和过早死亡。在项目开始时，美国食品及药物管理局尚未批准治疗 aHUS 的药物。Furthermore, as only a few thousand aHUS patients are diagnosed … Continued
原发性胆汁性胆管炎（PBC）是一种慢性疾病，会阻碍胆汁从肝脏流出，导致胆汁酸浓度升高，从而造成细胞损伤。PBC 如不及时治疗，可导致肝功能衰竭和死亡。在项目开始时，唯一获批的 PBC 治疗方法并非对所有患者都有效。
A large biopharmaceutical company required insight on the current market access landscape in a rare disease, as well as feedback on a pipeline product that will launch as a second-to-market agent in the rare disease.
