Adakveo (crizanlizumab-tmca) is an intravenous humanized monoclonal antibody that binds to P-selectin. P-selectin is an adhesion molecule expressed on vascular endothelial cells and platelets and is a key modulator in the vaso-occlusive process. The inhibition of P-selectin is believed to reduce sickle red blood cell adhesion, thereby improving blood flow and minimizing vaso-…
Horizon Scanning
It is a challenge for health plans and clinicians to know what emerging technologies are in the preapproval phase and what their clinical, financial, and risk impacts might be. This is where we come in.The Hayes Horizon Scanning Solution identifies innovative and potentially disruptive health technologies that are in various stages of the regulatory approval process. Our Research Analysts continually investigate new technologies that are on the horizon or in the early stages of clinical investigation, as well as the testing of existing technologies that may be seeking approval for new clinical applications. Hayes Horizon Scanning helps you track emerging technologies before you need to make decisions. Accessed through the Hayes Knowledge Center, Hayes Horizon Scanning addresses the following fundamental questions to help our clients forecast emerging technologies:
- Is this emerging technology new or replacing an existing technology?
- Where is this technology in the FDA regulatory process?
- When will this technology be commercially available in the market?
- What is the projected use, cost, and financial impact of this technology?
Ruzurgi (Amifampridine) for Pediatric Lambert-Eaton Myasthenic Syndrome
Ruzurgi (amifampridine, base form) is an oral drug intended for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in children aged 6 to < 17 years. Ruzurgi is a voltage-gated potassium channel inhibitor that prolongs the depolarization of the pre-synaptic cell membrane, allowing for enhanced calcium influx into the neuron. This facilitates increased release of acetylc...
Ruzurgi (Amifampridine) for Pediatric Lambert-Eaton Myasthenic Syndrome
Ruzurgi (amifampridine, base form) is an oral drug intended for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in children aged 6 to < 17 years. Ruzurgi is a voltage-gated potassium channel inhibitor that prolongs the depolarization of the pre-synaptic cell membrane, allowing for enhanced calcium influx into the neuron. This facilitates increased release of acetylc...
Firdapse (Amifampridine Phosphate)
Firdapse (amifampridine phosphate) is an oral voltage-gated potassium channel inhibitor for the symptomatic treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults. Firdapse increases the release of acetylcholine from nerve cells, prolonging the depolarization of the pre-synaptic cell membrane and allowing for enhanced calcium influx into the neuron. This, in turn, fa…
Firdapse (Amifampridine Phosphate)
Firdapse (amifampridine phosphate) is an oral voltage-gated potassium channel inhibitor for the symptomatic treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults. Firdapse increases the release of acetylcholine from nerve cells, prolonging the depolarization of the pre-synaptic cell membrane and allowing for enhanced calcium influx into the neuron. This, in turn, fa…
Zolgensma (Onasemnogene Abeparvovec-xioi) for Spinal Muscular Atrophy
Zolgensma (onasemnogene abeparvovec-xioi; formerly AVXS-101) is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients < 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma is intended to replace the defective
Zolgensma (Onasemnogene Abeparvovec-xioi) for Spinal Muscular Atrophy
Zolgensma (onasemnogene abeparvovec-xioi; formerly AVXS-101) is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients < 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma is intended to replace the defective
Inebilizumab for Neuromyelitis Optica Spectrum Disorder
Inebilizumab is an investigational anti-CD19 monoclonal antibody. It was developed in response to the unmet treatment needs of patients with neuromyelitis optica spectrum disorder (NMOSD), a rare and debilitating autoimmune demyelinating disease that primary affects the optic nerves and spinal cord. Inebilizumab is administered every 26 weeks by intravenous infusion.
Viaskin Epicutaneous Immunotherapy (EPIT) for Peanut Allergy
Viaskin Peanut is an epicutaneously delivered immunotherapy in development for the treatment of peanut allergy. The Viaskin patch contains antigen in dry form, which adheres to a central backing surrounded by an adhesive crown that secures the patch to the skin, creating an occlusive chamber. The patch is applied once daily. The Viaskin patch works by gradually exposing the p…
Beovu (Brolucizumab) for Neovascular Age-Related Macular Edema
Beovu is a human vascular endothelial growth factor (VEGF) inhibitor indicated for the treatment of neovascular (wet) age-related macular degeneration (AMD). Inhibition of the VEGF pathway is believed to impede the growth of neovascular lesions, resolve retinal edema, and improve vision in patients with neovascular AMD.