NEURELIS CONGRATULATES INTRAVAIL® LICENSEE, ARS PHARMACEUTICALS, FOR FDA ACCEPTANCE OF INTRANASAL EPINEPHRINE NDA
2022-12-08
孤儿药上市批准申请上市
® Absorption-Enhancing Technology
SAN DIEGO, Dec. 8, 2022 /PRNewswire/ -- ARS Pharmaceuticals, Inc., announced that the U.S. Food & Drug Administration (FDA) has accepted its New Drug Application (NDA) for neffy (previously referred to as ARS-1), for the emergency treatment of severe type I allergic reactions in children and adults who weigh 30 kilograms (66 pounds) or more. In accordance with FDA guidelines, an anticipated target action date of mid-2023 has been set for the PDUFA (Prescription Drug User Fee Act).
"The FDA's acceptance of the neffy NDA marks a major milestone in the development of a non-injectable anaphylactic rescue medication. The potential quality of life implications for millions of people with severe type I allergic reactions is profound," said Neurelis CEO, Craig Chambliss. "If approved, neffy will be the latest FDA approved product utilizing Intravail, following the recent successful introduction of VALTOCO® (diazepam nasal spray) to the epilepsy community."
neffy has the potential to be the first non-injectable treatment available to patients with severe type I allergic reactions, including anaphylaxis, and is designed for convenience, ease-of-use, and rapid administration. As compared to currently available epinephrine treatment options, this is a simple to use, needle-free, low-dose aqueous formulation of epinephrine for nasal administration, developed with Neurelis' Intravail transmucosal absorption enhancement technology. Intravail enhances drug absorption through the nasal mucosa, enabling noninvasive delivery of a broad range of protein, peptide, and small-molecule drugs.
The NDA submission to the FDA was based on data from four primary registration studies, demonstrating that a 2mg intranasal dose of neffy met all clinical endpoints recommended by regulators and that its pharmacokinetics were within the range of approved epinephrine injection products. These data included studies in adults with self-administration and caregiver administration, as well as in children with type I allergies ≥30 kg (66 lbs). In addition, neffy has been well-tolerated to date, with more than 500 individuals receiving at least one dose and many with repeat dosing. The majority of adverse events in clinical trials were mild in nature without any meaningful nasal irritation or pain.
About Allergic Reactions (type I), including AnaphylaxisAnaphylaxis
Type I severe allergic reactions are serious and potentially life-threatening events that can occur within minutes of exposure to an allergen and require immediate treatment with epinephrine, the only FDA-approved medication for these reactions. While epinephrine autoinjectors have been shown to be highly effective, there are well published limitations that result in many patients and caregivers delaying or not administering treatment in an emergency situation. These limitations include fear of the needle, lack of portability, needle-related safety concerns, lack of reliability, and complexity of the devices. There are approximately 25 to 40 million people in the United States who experience Type I severe allergic reactions. Of those, only 3.3 million currently have an active epinephrine autoinjector prescription, with only half consistently carrying their prescribed autoinjector. Even if patients or caregivers carry an autoinjector, more than half either delay or do not administer the device when needed in an emergency.
About ARS Pharmaceuticals
ARS Pharmaceuticals is dedicated to empowering at-risk patients and caregivers to better protect themselves from severe allergic reactions that could lead to anaphylaxis. The company is developing neffy
® (previously referred to as ARS-1), an intranasal epinephrine product in clinical development for patients and their caregivers with Type I allergic reactions including food, medications and insect bites that could lead to life-threatening anaphylaxis. For more information, visit www.ars-pharma.com.
About Neurelis
Neurelis, Inc., is a commercial-stage neuroscience company focused on the development and commercialization of therapeutics for the treatment of epilepsy and orphan neurologic disorders characterized by high unmet medical need. In 2020, the FDA approved Neurelis' VALTOCO® (diazepam nasal spray) as an acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) that are distinct from an individual's usual seizure pattern in adult and pediatric patients 6 years of age and older. VALTOCO is a proprietary formulation of diazepam incorporating the science of INTRAVAIL®. Intravail's transmucosal absorption enhancement technology enables the noninvasive delivery of a broad range of protein, peptide and small-molecule drugs. In its approval of VALTOCO, the U.S. Food and Drug Administration also granted Neurelis Orphan Drug Exclusivity and recognized VALTOCO's intranasal route of administration as a clinically superior contribution to patient care over the previously approved standard-of-care treatment (a rectal gel formulation of diazepam). For more information on VALTOCO, please visit http://www.valtoco.com/. In addition to VALTOCO, Neurelis is developing NRL-4 as a noninvasive rescue therapy to address the escalation of acute agitation symptoms associated with schizophrenia and bipolar 1 mania in adults. In addition, Neurelis is developing NRL-1049 (previously known as BA-1049), an investigational, pre-clinical stage small molecule Rho kinase (ROCK) inhibitorRho kinase (ROCK) inhibitor, for the treatment of cerebral cavernous malformations (CCMS), a rare disorder of the central nervous system (CNS). For more information on Neurelis, please visit http://www.neurelis.com/. For the latest scientific information on VALTOCO, please visit http://www.neurelismedicalaffairs.com/.
Important Safety Information about VALTOCO:
Indication
VALTOCO® (diazepam nasal spray) is indicated for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., 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.
WARNING: RISKS FROM CONCOMITANT USE WITH OPIOIDS; ABUSE, MISUSE, AND ADDICTION; and DEPENDENCE AND WITHDRAWAL REACTIONS
Concomitant use of benzodiazepines and opioids may result in profound sedation, respiratory depression, coma, and death. Reserve concomitant prescribing of these drugs for patients for whom alternative treatment options are inadequate. Limit dosages and durations to the minimum required. Follow patients for signs and symptoms of respiratory depression and sedation.
The use of benzodiazepines, including VALTOCO, exposes users to risks of abuse, misuse, and addiction, which can lead to overdose or death. Abuse and misuse of benzodiazepines commonly involve concomitant use of other medications, alcohol, and/or illicit substances, which is associated with an increased frequency of serious adverse outcomes. Before prescribing VALTOCO and throughout treatment, assess each patient's risk for abuse, misuse, and addiction.
The continued use of benzodiazepines may lead to clinically significant physical dependence. The risks of dependence and withdrawal increase with longer treatment duration and higher daily dose. Although VALTOCO is indicated only for intermittent use, if used more frequently than recommended, abrupt discontinuation or rapid dosage reduction of VALTOCO may precipitate acute withdrawal reactions, which can be life-threatening. For patients using VALTOCO more frequently than recommended, to reduce the risk of withdrawal reactions, use a gradual taper to discontinue VALTOCO. |
Contraindications: VALTOCO is contraindicated in patients with:
Hypersensitivity to diazepam
Benzodiazepines, including VALTOCO, may produce CNS depression. Caution patients against engaging in hazardous activities requiring mental alertness, such as operating machinery, driving a motor vehicle, or riding a bicycle, until the effects of the drug, such as drowsiness, have subsided, and as their medical condition permits.
Suicidal Behavior and Ideation
Antiepileptic drugs (AEDs), including VALTOCO, increase the risk of suicidal ideation and behavior. Patients treated with any AED for any indication should be monitored for the emergence or worsening of depression, suicidal thoughts or behavior, and/or unusual changes in mood or behavior.
Benzodiazepines, including VALTOCO, can increase intraocular pressure in patients with glaucoma. VALTOCO may only be used in patients with open-angle glaucoma only if they are receiving appropriate therapy. VALTOCO is contraindicated in patients with narrow-angle glaucoma.
Risk of Serious Adverse Reactions in Infants due to Benzyl Alcohol Preservative
VALTOCO is not approved for use in neonates or infants. Serious and fatal adverse reactions, including "gasping syndrome," can occur in neonates and low-birth-weight infants treated with benzyl alcohol-preserved drugs, including VALTOCO. The "gasping syndrome" is characterized by central nervous system depression, metabolic acidosis, and gasping respirations. The minimum amount of benzyl alcohol at which serious adverse reactions may occur is not known.
The most common adverse reactions (at least 4%) were somnolence, headache, and nasal discomfort.
To report SUSPECTED ADVERSE REACTIONS, contact Neurelis, Inc. at 1-866-696-3873 or FDA at 1-800-FDA-1088 (www.fda.gov/medwatch).
Please read full Prescribing Information, including Boxed Warning, for additional important safety information.
Contacts:
Brittany Bradrick, Chief Operating Officer and Chief Financial Officer, +1 858 251 2135
Media:
Erich Sandoval, Finn Partners, + 1 917 497 2867
SOURCE Neurelis, Inc.
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