Nov 24, 2023

Aculys Pharma receives orphan drug designation for a diazepam nasal spray: an antiepileptic drug for epileptic seizures

Tokyo, Japan, November 24, 2023 Aculys Pharma, Inc., a clinical stage biopharmaceutical company focused on commercializing innovative treatments for neurological conditions, today announced that a diazepam nasal spray (compound development code: NRL-1), an antiepileptic drug currently under development for the treatment of epileptic seizures, has been designated as an orphan drug by the Ministry of Health, Labour and Welfare for the treatment of status epilepticus.

This drug is the first nasally administered antiepileptic under development in Japan to treat epileptic seizures. It has achieved the primary endpoint in the pre-specified interim analysis of the ongoing domestic Phase 3 clinical trials*.

Shinichiro Takeuchi, Head of Regulatory Affairs and Clinical Strategy at Aculys Pharma, said: "While epileptic seizures usually resolve spontaneously within a few minutes, they can last for abnormally long periods, or lead to status epilepticus, in which seizures occur repeatedly before consciousness is regained. There are concerns that, in addition to its impact on the QOL (Quality of Life) of patients and their families, status epilepticus may affect life prognosis and cause damage to the brain, so prompt therapeutic intervention is required1. Currently, there are several drugs which can be used outside the hospital setting, but those have issues with convenience and other aspects. As an intranasal formulation, this drug can be easily and appropriately administered by non-medical personnel such as family members and caregivers outside of medical institutions, such as when away from home or at school. As such, it is expected to become a new treatment option for status epilepticus patients. These advantages were evaluated, and we were able to obtain designation as an orphan drug. We will continue to strive to obtain early approval of this drug, so that we can help to resolve the concerns and issues faced by patients and their families."

For the interim analysis results of the Phase III clinical trial, please refer to the press release dated October 18, 2023 “Aculys Pharma delivers positive Phase 3 clinical study interim analysis result of a diazepam nasal spray: an antiepileptic drug for the treatment of epileptic seizures” ( ).

About diazepam and diazepam nasal spray

Diazepam has been used in Japanese medical practice for about 60 years as a treatment for epileptic seizures in the form of injections and other forms. It is also administered as a suppository by non-medical personnel, such as patients and caregivers, outside medical institutions.

Diazepam nasal spray was developed by the neuroscience company Neurelis, Inc. Aculys Pharma holds an exclusive license to develop and commercialize this treatment in Japan and the Asia-Pacific region*(excluding Greater China and Singapore). In 2020, the FDA approved Neurelis’ VALTOCO® (diazepam nasal spray) as acute treatment of intermittent, stereotypic episodes of frequent seizure activity (ie, seizure clusters, acute repetitive seizures) that are distinct from a patient’s usual seizure pattern in patients with epilepsy 6 years of age and older. In June 2023, China's National Medical Products Administration (NMPA) approved diazepam nasal spray as effective for the same indication.

(*Asia-Pacific region: Australia, Brunei, Cambodia, Indonesia, South Korea, Laos, Malaysia, Myanmar, New Zealand, Philippines, Thailand, Vietnam)

About epilepsy

Epilepsy is a chronic brain disorder that causes loss of consciousness and/or convulsions (epileptic seizures) as the result of excessive electrical excitation of nerve cells (neurons) in the brain. An estimated 600,000 to 1,000,000, or 5 to 8 in 1,000 people in Japan have epilepsy. As treatment has advanced, many people with epilepsy are able to control their seizures with proper diagnoses and antiepileptic medications and are able to lead normal social lives; however, about 30% of patients, as well as their families and caregivers, still need to deal with frequent, repeated seizures.2,3

The causes, symptoms, and severity of epilepsy vary greatly from one individual patient to another, and similarly, epileptic seizures are diverse in nature from patient to patient. Among the diverse seizure types, some people experience seizures that recur many times a day or that do not terminate after a certain period of time (status epilepticus). These patients are at increased risk of brain damage and reduced life expectancy.1 When repetitive seizures occur, people with epilepsy must seek emergency treatment by healthcare professionals. On average, in Japan it takes 20~40 minutes from the time an emergency call is placed until a person is transferred to a hospital for treatment.1 An extensive overseas survey of patients, their families, and physicians has reported that recurrent epileptic seizures cause significant emotional, social, and financial burdens on patients and their families.4

About Orphan Drugs

Based on Article 77-2 of the Pharmaceutical and Medical Device Act, the Minister of Health, Labor and Welfare designates drugs which meet the conditions that the number of eligible patients in Japan is less than 50,000 and that there is a particularly high medical need. In doing so, it refers to the opinions of the Pharmaceutical Affairs and Food Sanitation Council. If a drug is so designated, companies will be able to receive support measures such as grants, tax measures, priority examinations, and extensions of reexamination periods. For details of this designation system, please see the Ministry of Health, Labour and Welfare's website.

About Neurelis, Inc.

Neurelis, Inc., is a neuroscience company focused on the development and commercialization of therapeutics for the treatment of epilepsy and orphan neurologic disorders characterized by high unmet medical needs. For more information about Neurelis, please visit

About Aculys Pharma

Aculys is a clinical stage biopharmaceutical company focused on the commercialization of neurology innovations for patients in Japan. Aculys was formed in 2021 to catalyze innovative treatments for neurological and psychiatric disorders. We are committed to shortening the drug lag in Japan by developing and commercializing novel CNS treatments; applying blockchain and AI technologies to increase speed to market; and providing services to improve medical care for patients, their families, healthcare professionals, and society.

Company name: Aculys Pharma, Inc.
Location: The ARGYLE Aoyama 6F, 2-14-4 Kita-Aoyama, Minato-ku, Tokyo
Representative: Takeshi Takahashi
Establishment date: January 2021

Information concerning pharmaceutical product (compound under development) contained herein is not intended as advertising or as medical advice, but intended for disclosure of management information.


  1. Treatment guidelines for pediatric status epilepticus & pediatric convulsive status epilepticus 2023 (Japanese Society of Child Neurology) (Japanese only)
  2. Epidemiological survey on the prevalence, diagnosis, and treatment of epilepsy and issues to be solved to establish a healthcare system for the disease, 2013 (Japanese only) (Accessed on Nov 15, 2023)
  3. Kwan P., Brodie, M.J. Early identification of refractory epilepsy. N. Engl. J. Med., 2000 Feb 3; 342(5): 314–9.
  4. Penovich, P.E., Buelow, J., Steinberg, et al. Burden of seizure clusters on patients with epilepsy and caregivers survey of patient, caregiver, and clinician perspectives. The Neurologist. 2017; 22: 207–214.