Those with Dravet syndrome have epilepsy that is typically not well controlled. In addition to frequent seizures, many patients regularly experience status epilepticus, which are prolonged seizures lasting more than 5 minutes. This is a serious medical emergency which can be life threatening and requires emergency intervention with rescue medications. Patients may also experience seizure clusters, which are seizures that start and stop, but occur one right after another for an extended period of time. It is not uncommon for seizure clusters to evolve into status epilepticus. Both status epilepticus and seizure clusters have a significant impact on health and patient quality of life and often result in emergency room visits or hospital stays.
While medical professionals can give rescue medications intravenously, home-based treatments require a different approach. Recently, two new rescue treatment options have been approved by the FDA that can easily be administered by non-medical professionals outside of a hospital setting. These new rescue medications are in the form of a pre-measured, single use nasal spray that can be administered anywhere and do not pose the social challenges that older patients face with rescue medications that require rectal administration.
The first, NAYZILAM is produced by UCB. It received FDA approval in May of 2019 and became available in December 2019. Each dispensed box contains two single-use 5mg midazalom nasal spray devices that can be stored at room temperature. The nasal spray device has a plunger that is pressed once the device is inserted into the patient’s nostril. UCB offers a NAYZILAM training kit so that caregivers and family members can practice and feel comfortable with what to do during an emergency. (The training device does not contain any active medication, and it can be reset and practiced with again and again.) UCB also offers a Patient Savings Card for NAYZILAM for eligible patients. Full details on the program can be found on their website.
The second, VALTOCO is produced by Neurelis and was approved in January 2020 and became available through an Early Access Program with a specialty pharmacy partner (Maxor) in March. This was to ensure that patients, care partners and HCPs could get access to VALTOCO as soon as it was commercially available. The specialty pharmacy delivers directly to the patient via UPS (generally, the next day). It will be available in retail pharmacies by fall 2020. This diazepam nasal spray is available in 5 mg, 10 mg, 15 mg, and 20 mg doses. Many patients in our community have used Diastat as a rescue medication, which is a rectal form of diazepam.
You can sign up to receive updates on availability, as well as learn about patient services on their website. Their patient support services – myNEURELIS – is a flexible program that allows patients to select desired support services, including help checking insurance coverage, Patient Assistance Programs, education, and more. Neurelis is offering copay assistance to eligible patients along with a patient assistance program. For more information, you can visit http://www.valtoco.com.
For patients who experience frequent seizure emergencies, such as those in the Dravet community, additional rescue medication options offers the potential to help improve overall quality of life for patients and their families. As a parent of a Dravet patient, I personally find these new options exciting as they are designed to be easy to use and can be administered by non-medical personnel in any setting.
Please note, DSF does not recommend or endorse any specific product, test, physician or other medical information provided on our website. The contents of our website, including this blog, are intended for informational and educational purposes only, and not for the purpose of rendering medical advice. Before taking any new medications, consult with your physician for personalized medical advice.