China-based Yichang Humanwell Pharmaceutical Co., Ltd’s generic version (oral suspension) of Sanofi’s clobazam (trade name: Frisium) has been approved by the National Medical Products Administration (NMPA) to be assessed in a clinical study as a combination therapy for seizures in patients with Lennox-Gastaut syndrome (LGS) two years of age and older. This marks a significant step forward in addressing the unmet needs of patients with this rare and severe form of epilepsy.
Lennox-Gastaut Syndrome
LGS is a rare epileptic encephalopathy that presents with intractable seizures in childhood, with an incidence of approximately 2/100,000. It is characterized by onset during childhood, and most cases are epileptic encephalopathy that continues for life. The etiology is complex and diverse, with viable drug treatments limited. Clobazam, first approved in Australia in 1970, was included in China’s second batch of pediatric drugs encouraged for development and fast-track treatment in 2017.
Regulatory and Market Context
The National Healthcare Commission (NHC) and NMPA released “Work Plan for the Temporary Import of Clinically Urgently Needed Drugs” and “Work Plan for the Temporary Import of Clobazam” in late March this year. Under these rules, the NHC can select medical institutions to file for temporary import approval from the NMPA. The temporarily imported clobazam was first prescribed on September 22 at Peking Union Medical College Hospital. Humanwell filed for marketing approval for its generic version (tablets) in March, which was granted in September, marking the first generic of the drug in China.
Future Development
The approval from the NMPA underscores Humanwell’s commitment to advancing treatments for rare pediatric diseases. By developing a generic version of clobazam, Humanwell aims to improve access to this life-saving drug for patients with LGS, potentially enhancing treatment options and patient outcomes.-Fineline Info & Tech
