昕悦®氨己烯酸口服溶液用散-儿童用药和罕见病用药

Listing process

In 1989, vigabatrin was approved in the UK for the treatment of epilepsy.

On August 21, 2009, it was approved by the US FDA with the trade name SABRILR (Vigabatrin).

In 2012, at the International Tuberous Sclerosis Consensus Conference, experts recommended vigabatrin as the first-line treatment for infantile spasms in patients with tuberous sclerosis.

On May 29, 2019, China's National Health Commission, Ministry of Industry and Information Technology, and State Food and Drug Administration jointly issued the "Second Batch of Clinically Urgent Overseas New Drug List", which included vigabatrin powder.

In May 2022, Chengdu Yuandong Biopharmaceutical Co., Ltd. was the first to apply for generic Xinyue® Vigabatrin Oral Solution Powder to be approved for marketing for the treatment of infantile spasms.

At the same time, vigabatrin is recommended by many authoritative guidelines and consensuses such as the International League Against Epilepsy (ILAE) and the Chinese Association against Epilepsy (CAAE).

This product is indicated for monotherapy in children 1 month to 2 years old with infantile spasms (when the potential benefit is greater than the potential risk of visual impairment). In addition, this product has clinically replaced adrenocorticotropin as an auxiliary therapeutic drug for infantile spasms and adult complex refractory partial seizures, and has been identified as a drug for children and a drug for rare diseases.

The dosage regimen for vigabatrin oral solution powder should be formulated based on the age range and body weight. When administering, a calibrated measuring device should be used to measure and prepare the dose, and the final concentration should be diluted to 50 mg/ml. The dosage needs to be adjusted in patients with renal insufficiency. During administration, the physician should inform the child's caregiver of the preparation and administration methods of vigabatrin oral solution powder to ensure that the child takes the correct dose. This product can be taken with or without food. If you want to stop using vigabatrin oral solution powder, the dose should be gradually reduced.

Mechanism of action

γ-Aminobutyric acid is the main inhibitory transmitter in the central nervous system. γ-Aminobutyric acid transaminase can decompose γ-aminobutyric acid. Vigabatrin oral solution can irreversibly combine with γ-aminobutyric acid aminotransferase in the form of covalent bonds, prevent γ-aminobutyric acid aminotransferase from decomposing γ-aminobutyric acid, increase the concentration of γ-aminobutyric acid in the brain, thereby inhibiting epileptic-like electrical activity in the brain, thereby treating various neurological diseases and exerting anti-epileptic and anti-spasmodic effects.

A retrospective study included 103 children with infantile spasms. On average, children have two or three types of epileptic seizures at the time of first visit. After starting to take vigabatrin, 80 patients had one seizure type, 15 had two, 8 had three, and no patients with four seizure types were observed, indicating that vigabatrin can effectively control and reduce seizure types, with a high overall effectiveness. During the follow-up, statistics showed that compared with baseline, the frequency of seizures was reduced by 83.3% at the first follow-up and by 96.7% at the last follow-up. Research results show that vigabatrin can effectively control infantile spasms, reduce spasm attacks to varying degrees and improve electroencephalogram hyperarrhythmia, and quickly control the patient's condition.

In addition, it has been clinically observed that the efficacy of vigabatrin monotherapy in the treatment of infantile spasms is equivalent to that of hormone preparations (such as glucocorticoids, adrenocorticotropic hormone, etc., the first-line treatment drugs for infantile spasms), but the recurrence rate is lower. It can also greatly reduce the adverse reactions of multiple organs and systems that may be caused by long-term or high-dose glucocorticoid use in children, such as hypertension, immunosuppression, electrolyte imbalance, irritability, infection, brain atrophy, development restriction, etc. It was observed in the trial that the efficacy and safety of vigabatrin monotherapy in the treatment of infantile spasms associated with tuberous sclerosis is significantly better than that of hormone therapy in the treatment of infantile spasms.

Jiang Yuwu, a member of the Genetic Committee of the International League Against Epilepsy and Director of the Center for Pediatrics and Childhood Epilepsy at Peking University First Hospital, said: "The main contradiction in the treatment of infantile spasms should be focused on, especially for epilepsy that is particularly difficult to treat and has a particularly poor prognosis. Treating the epilepsy first is the most important, with as short a course of treatment as possible and as little dosage as possible. If it is effective, try to reduce the risk. If it can be cured, the price of this risk is worth it, so you should give it a try. Therefore, it is particularly emphasized that once infantile spasms is diagnosed, symptomatic treatment with vigabatrin must be chosen early."