Drug approvals in 2023

Following is a list of drugs for neurologic conditions that received approval from the U.S. Food and Drug Administration in 2023.

Alpha-mannosidosis

Lamzede® (velmanase alfa-tycv), an alimentary tract and metabolism product
Approval granted: February 16, 2023
Indication: Treatment of non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients
Manufacturer's press release

Alzheimer disease

LEQEMBI® (lecanemab-irmb), an amyloid beta-directed antibody
Accelerated approval granted: March 6, 2023
Approval granted: July 6, 2023
Indication: Treatment of Alzheimer disease
Manufacturer’s press release

REXULITI® (brexpiprazole), an atypical antipsychotic
Approval granted on: July 10, 2015
Approval for new indication granted on: May 10, 2023
Indication: Treatment of agitation associated with dementia due to Alzheimer disease
Manufacturer’s press release

Amyotrophic lateral sclerosis

QALSODY™ (tofersen), an antisense oligonucleotide
Accelerated approval granted: April 24, 2023
Indication: Treatment of adults with amyotrophic lateral sclerosis who have a mutation in the superoxide dismutase 1 gene
Manufacturer’s press release

Candidiasis

REZZAYO™ (rezafungin for injection), an echinocandin antifungal
Approval granted: March 22, 2023
Indication: Treatment of candidemia and invasive candidiasis in adults with limited or no alternative treatment options
Manufacturer’s press release

Cataplexy or excessive daytime sleepiness

LUMRYZ™ (sodium oxybate), a central nervous system depressant
Approval granted: May 1, 2023
Indication: Treatment of cataplexy or excessive daytime sleepiness in adults with narcolepsy
Manufacturer’s press release

Cervical dystonia

DAXXIFY® (daxibotulinumtoxinA-lanm), an acetylcholine release inhibitor and neuromuscular blocking agent
Approval granted: September 8, 2022
Approval for expanded indication granted on: August 15, 2023
Indication: Treatment of cervical dystonia in adults
Manufacturer’s press release

Chorea associated with Huntington disease

INGREZZA® (valbenazine), a vesicular monoamine transporter 2 inhibitor
Approval granted: April 11, 2017
Approval for expanded indication granted on: August 18, 2023
Indication: Treatment of adults with chorea associated with Huntington disease
Manufacturer’s press release

Chronic inflammatory demyelinating polyneuropathy

HYQVIA ®, (Ig Infusion 10% [Human] with rHuPH20), subcutaneous immunoglobulin infusion
Approval granted for primary immunodeficiency in adults: September 12, 2014
Approval granted for primary immunodeficiency in children 2 to 16 years old: April 11, 2023
Approval for expanded indication for CIDP in adults granted: January 16, 2024
Indication: Treatment of chronic inflammatory demyelinating polyneuropathy in adults
Manufacturer’s press release

Duchenne muscular dystrophy

AGAMREE® (vamorolone), a corticosteroid
Approval granted: October 26, 2023
Indication: Treatment of Duchenne muscular dystrophy in patients 2 years of age and older
Manufacturer’s press release

ELEVIDYS (delandistrogene moxeparvovec-rokl), an adeno-associated virus vector-based gene therapy
Accelerated approval granted: June 22, 2023
Indication: Treatment of ambulatory pediatric patients 4 to 5 years of age with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene
Manufacturer’s press release

Fabry disease

ELFABRIO® (pegunigalsidase alfa-iwxj), a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme
Approval granted: May 10, 2023
Indication: Treatment of adults with confirmed Fabry disease
Manufacturer's press release

Friedreich ataxia

SKYCLARYS® (omaveloxolone), an activator of the nuclear factor (erythroid-derived 2)-like 2 (Nrf2) pathway
Approval granted: February 28, 2023
Indication: Treatment of Friedreich ataxia in patients 16 years of age and older
Pharmaceutical Technology news release
Biogen acquisition of Reata Pharmaceuticals news release

Migraine

QULIPTA™ (atogepant), a calcitonin gene-related peptide receptor antagonist
Approval granted: September 28, 2021
Approval of expanded indication granted on: April 17, 2023
Indication: Preventive treatment of chronic migraine in adults
Manufacturer’s press release

RizaFilm® (rizatriptan), a serotonin receptor agonist (triptan)
Approval granted: April 18, 2023
Indication: Acute treatment of migraine with or without aura in adults and in pediatric patients 12 to 17 years of age weighing 40 kg or more
Manufacturer’s press release

ZAVZPRET™ (zavegepant), a calcitonin gene-related peptide receptor antagonist
Approval granted: March 9, 2023
Indication: Treatment of migraine with or without aura in adults
Manufacturer’s press release

Multiple sclerosis

Tyruko® (natalizumab-sztn), an integrin receptor antagonist
Approval granted: August 24, 2023
Indication: Treatment of relapsing forms of multiple sclerosis, to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults. For inducing and maintaining clinical response and remission in adult patients with moderately to severely active Crohn disease.
Manufacturer’s press release

Myasthenia gravis

RYSTIGGO® (rozanolixizumab-noli), a humanized monoclonal antibody that binds with high affinity to human neonatal Fc receptor
Approval granted: June 26, 2023
Indication: Treatment of generalized myasthenia gravis in adults who are anti-acetylcholine receptor or anti-muscle-specific tyrosine kinase antibody positive
Manufacturer’s press release

VYVGART® Hytrulo (efgartigimod alfa and hyaluronidase-qvfc), a neonatal Fc receptor blocker and endoglycosidase combination
Approval granted: June 20, 2023
Indication: Treatment of generalized myasthenia gravis in adult patients who are anti-acetylcholine receptor antibody positive.
Manufacturer’s press release

ZILBRYSQ® (zilucoplan), a macrocyclic peptide inhibitor of complement component 5
Approval granted: October 17, 2023
Indication: Treatment of generalized myasthenia gravis in adult patients who are anti-acetylcholine receptor antibody-positive
Manufacturer’s press release

Mydriasis induction

Mydcombi™ (tropicamide and phenylephrine hydrochloride ophthalmic spray), an anticholinergic + alpha-1 adrenergic receptor agonist
Approval granted: May 8, 2023
Indication: To induce mydriasis for diagnostic procedures and in conditions where short term pupil dilation is desired
Manufacturer’s press release

Opioid overdose

RiVive™ (naloxone HCI), an opioid antagonist
Approval granted: July 28, 2023
Indication: Emergency treatment of known or suspected opioid overdose
Manufacturer’s press release

Polymyalgia rheumatica

Kevzara® (sarilumab), an interleukin-6 receptor antagonist
Approval granted: February 28, 2023
Indication: Treatment of adults with polymyalgia rheumatica who have had an inadequate response to corticosteroids or who cannot tolerate corticosteroid taper.
Manufacturer’s press release

Pompe disease

Pombiliti™ (cipaglucosidase alfa-atga) plus Opfolda™ (miglustat), a hydrolytic lysosomal glycogen-specific enzyme plus enzyme stabilizer
Approval granted: September 28, 2023
Indication: Treatment of late-onset Pompe disease in patients weighing at least 40kg and who are not improving on their current enzyme replacement therapy
Manufacturer’s press release

Rett syndrome

DAYBUE™ (trofinetide), a synthetic analogue of glycine-proline-glutamate
Approval granted: March 10, 2023
Indication: Treatment of Rett syndrome in adults and pediatric patients 2 years of age and older
Manufacturer’s press release