Dual FDA designations represent important regulatory milestones in the development of ASP-001 for Alternating Hemiplegia of Childhood
[Toronto, ON] - [Aug. 10, 2026] - Aspartes Pharmaceuticals, Inc., a clinical-stage pharmaceutical company focused on advancing therapies for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to ASP-001, the Company's investigational orally disintegrating tablet (ODT) formulation of flunarizine, for the treatment of Alternating Hemiplegia of Childhood (AHC).
"Receiving both Orphan Drug Designation and Rare Pediatric Disease Designation represents two important regulatory milestones for Aspartes and for the ASP-001 development program," said Denny Medjedovic, Founder and Chief Executive Officer of Aspartes Pharmaceuticals. "AHC is a serious and deeply debilitating neurological movement disorder that presents within the first 18 months of life and causes lifetime morbidity and increased mortality. There are currently no FDA-approved treatments available for this condition, representing a substantial unmet need. Aspartes has the support of key physicians and patient advocacy groups to pursue the development of ASP-001 as a treatment for AHC. We look forward to continued collaboration with the FDA as we further our development plans for ASP-001 and work toward our goal of providing patients and families with a treatment developed specifically for this rare disease.”
Flunarizine is a CNS-acting calcium channel inhibitor that has a long history of clinical use in the management of AHC and remains an important treatment option for many patients internationally, but it is not currently approved or commercially available in the United States. Published literature suggests that flunarizine is safe and well-tolerated by patients and may be effective in reducing the frequency, duration, and severity of hemiplegic attacks associated with AHC.
ASP-001 is a novel pediatric orally disintegrating tablet formulation of flunarizine being developed by Aspartes Pharmaceuticals as a standardized age-appropriate treatment for patients with AHC. The program is intended to build upon the existing clinical experience with flunarizine while providing a standardized dosage form designed to support the dosing needs of pediatric patients with AHC. ASP-001 is being developed in 2mg and 5mg ODT strengths to provide greater flexibility for age- and weight appropriate dosing and is currently undergoing a Phase 1 trial in healthy adult volunteers.
FDA's Orphan Drug Designation is intended to support the development of therapies for rare diseases affecting fewer than 200,000 people in the United States. The designation provides potential development incentives, including tax credits for qualified clinical trials, exemption from certain FDA user fees and, following approval and satisfaction of applicable requirements, the potential for seven years of orphan-drug market exclusivity.
Rare Pediatric Disease Designation is granted to therapies intended to treat serious or life-threatening diseases that primarily affect children aged 18 years or younger and meet statutory rarity criteria. Upon approval of a qualifying marketing application, the sponsor may be eligible for a Priority Review Voucher (PRV), subject to FDA requirements.
The dual designations further support Aspartes' continued regulatory and clinical development of ASP-001 as the Company works to establish a treatment option specifically developed for patients with AHC.
About Alternating Hemiplegia of Childhood
Alternating Hemiplegia of Childhood (AHC) is a rare and severe neurological disorder that typically begins in infancy or early childhood. The disease is characterized by recurrent episodes of weakness or paralysis affecting one or both sides of the body, as well as other paroxysmal neurological manifestations that may include dystonia, abnormal eye movements and autonomic symptoms. Patients may also experience epilepsy, developmental impairment and persistent motor and neurological difficulties.There is currently no specific FDA-approved therapy for AHC, and treatment is directed toward managing the diverse symptoms of the disease. Flunarizine is commonly used in the management of AHC and has been reported to reduce the duration, severity and frequency of hemiplegic attacks in some patients.
About Aspartes Pharmaceuticals
Aspartes Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company focused on advancing promising therapies for rare diseases and patient populations with significant unmet medical needs. Aspartes seeks to build upon established clinical experience with promising therapeutic agents through optimized formulations and focused development programs.
The Company's lead program, ASP-001, is an investigational orally disintegrating tablet formulation of flunarizine being developed for the treatment of Alternating Hemiplegia of Childhood.
For more information, visit www.aspartes.com
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Media Contact Denny Medjedovic Aspartes Pharmaceuticals, Inc. |
