FCS是一种严重且极为罕见的遗传性疾病，常由各种单基因突变引起。FCS导致极高的甘油三酯(TG)水平，通常超过880mg/dL。这种严重的升高会导致各种严重的体征和症状，包括急性和潜在致命的胰腺炎、慢性腹痛、糖尿病、肝脂肪变性和认知问题。目前，能够充分治疗FCS的治疗选择很有限，严格的低脂饮食是目前治疗这种疾病的一线方法。Olezarsen被批准作为一种附加治疗，以降低成人患者的甘油三酯(TG)水平。The FDA has approved Olezarsen from Ionis Pharmaceuticals for the treatment of familial chylomicronemia syndrome (FCS).
According to Zhitong Finance APP, the U.S. Food and Drug Administration (FDA) has approved Olezarsen, a drug for rare genetic diseases from Ionis Pharmaceuticals (IONS.US), marking the company's first wholly-owned drug.
It is reported that Olezarsen has been approved for the treatment of familial chylomicronemia syndrome (FCS). Trial results released in April showed that for patients with moderate hypertriglyceridemia at high cardiovascular risk, Olezarsen can significantly reduce triglycerides, apolipoprotein B, and non-HDL cholesterol levels, with no major safety issues reported.
FCS is a serious and extremely rare genetic disease often caused by various single-gene mutations. FCS leads to extremely high triglyceride (TG) levels, usually exceeding 880 mg/dL. This severe elevation can cause various serious signs and symptoms, including acute and potentially fatal pancreatitis, chronic abdominal pain, diabetes, hepatic steatosis, and cognitive problems. Currently, there are very limited treatment options available for FCS, with a strict low-fat diet being the first-line method for treating this condition. Olezarsen has been approved as an additional treatment to lower triglyceride (TG) levels in adult patients.
Buoyed by this news, Ionis Pharmaceuticals's stock rose nearly 7% in Post-Market Trading on Thursday.
Ionis Pharmaceuticals stated that it will announce pricing details of the drug during an investor conference call. The company's CEO, Brett Monia, stated, "We believe there are no supply obstacles. We have produced and are ready." Brett Monia added that patients still need to adhere to the recommended dietary restrictions.
However, BMO Capital Markets Analyst Kostas Biliouris believes that given the low prevalence of FCS and the fact that competing drugs will enter the market in the second half of next year, FCS treatment drugs will not be a significant opportunity for Ionis Pharmaceuticals. According to U.S. government data, there are fewer than 5,000 people with FCS in the USA. Kostas Biliouris estimates that the sales of Ionis Pharmaceuticals's FCS treatment drug could reach up to $0.341 billion.
