China-based Yichang Humanwell Pharmaceutical Co., Ltd’s generic version of Sanofi’s clobazam (trade name: Frisium) has been approved by the National Medical Products Administration (NMPA) for use as a combination therapy for seizures in patients with Lennox-Gastaut syndrome (LGS) aged two years and older. This marks the first availability of this therapy for patients in China.
Disease Overview and Treatment Needs
Lennox-Gastaut syndrome (LGS) is a rare epileptic encephalopathy characterized by intractable seizures in childhood, with an incidence of approximately 2/100,000. It typically begins in childhood and most cases continue as epileptic encephalopathy throughout life. The etiology is complex and diverse, and viable drug treatments are limited.
Regulatory and Market Context
Clobazam was first approved in Australia in 1970 and was included in China’s second batch of pediatric drugs encouraged for development and fast-track treatment in 2017. Due to the narrow market, limited profits, and its classification as a Class II psychotropic drug, pharmaceutical companies were slow to develop the product. The drug went out of stock in July last year, prompting a letter signed by 1,042 patients’ parents to circulate on Weibo in November. In response, the National Healthcare Commission (NHC) and NMPA released plans for the temporary import of clinically urgently needed drugs, including clobazam, in late March this year. The temporarily imported clobazam was first prescribed on September 22 at Peking Union Medical College Hospital.
Humanwell’s Development and Approval Process
Humanwell Pharma decided to file for the development of clobazam in 2017, noticing the drug shortage, and began development work in 2018. The company achieved a final total yield more than 10%-15% higher than that reported in the literature. Humanwell filed for market approval in March this year, which was prioritized for review in May and approved in September in under six months.
Future Outlook and Patient Access
Humanwell plans to include clobazam in the National Reimbursement Drug List (NRDL) in 2023 and is collaborating with a third-party fund to provide assistance to financially challenged patients seeking access. This approval and planned inclusion in the NRDL highlight Humanwell’s commitment to improving patient access to critical treatments for rare pediatric diseases.-Fineline Info & Tech
