Novartis siRNA Therapy Receives EU Marketing Approval

Novartis announced on the 12th that its siRNA therapy Leqvio (inclisiran) has been approved by the European Commission for the treatment of adult hypercholesterolemia and mixed dyslipidemia. This is the first batch of this "first-in-class" therapy in the world. It is worth mentioning that after the initial injection and the third month of injection treatment, the patient is expected to receive only two treatments a year to reduce his "bad cholesterol" level.

"Bad cholesterol" refers to "low-density lipoprotein cholesterol" (LDL-C), which is an important risk factor for cardiovascular disease. Even though statins that lower LDL-C levels are very popular, heart disease is still the cause of death worldwide One of the primary factors. Despite the widespread use of statins, 80% of high-risk patients still fail to reach the LDL-C target level recommended by the guidelines, and they urgently need more innovative therapies.

As a "first-in-class" siRNA therapy, inclisiran can bind to the mRNA encoding PCSK9 protein, reduce its level through RNA interference, and prevent the liver from producing PCSK9 protein. The role of PCSK9 protein is to inhibit the recovery and reuse of LDL receptors. Therefore, lowering the level of PCSK9 protein allows more LDL receptors to return to the surface of liver cells, bind to more LDL, and remove them from the blood.

The European Commission's decision to approve this therapy is based on the results of the ORION clinical development project. In clinical trials, inclisiran has shown a positive effect-for patients who are unable to achieve LDL-C levels after treatment with the maximum tolerated dose of statin, inclisiran can reduce LDL-C levels by up to 52%. Earlier this year, the clinical results of the project were also published in the New England Journal of Medicine.

In the press release, Ms. Marie-France Tschudin, Global President of Novartis Pharmaceuticals, said: “Cardiovascular disease is still the main cause of death so far. This shows that patients with substandard LDL-C have an urgent need for innovative treatments.” As a “first” -in-class" drug, inclisiran can effectively and continuously reduce LDL-C levels, and has great potential in improving the treatment outcome of patients with ASCVD (atherosclerotic cardiovascular disease).

So far, many RNAi therapies have been approved worldwide, but most of them are used to treat rare diseases with a small number of patients. The approval of Inclisiran for marketing indicates that RNAi therapy is expected to expand its therapeutic field and change the treatment of common diseases, which is of great significance. In the future, we also expect more innovative therapies to emerge in an endless stream to benefit global health!