Neurological disorders

ALS

• Gold Nanocrystals
• Ibudilast
• Lenzumestrocel

• Pridopidine

  • Amyotrophic lateral sclerosis (ALS)

Dementia

• Blarcamesine
• Donanemab
• Hydromethylthionine mesylaat
• Lecanemab
• Valiltramiprosate

Depression

• Psilocybine
• Seltorexant
• Zuranolone

Duchenne

• Casimersen
• Delandistrogene moxeparvovec
• Delpacibart zotadirsen
• Deramiocel

• Givinostat

  • Duvyzat is indicated for the treatment of Duchenne muscular dystrophy (DMD) in ambulant patients, aged 6 years and older, and with concomitant corticosteroid treatment

• Vamorolone

  • Extension of indication to include treatment of 2 to <4 year olds with Duchenne muscular dystrophy (DMD)

Epilepsy

• Bexicaserin
• Opakalim
• Zonisamide ^IND

Eye disorders

• Acoltremon
• Atropine sulfate ^IND
• Bimatoprost
• Botaretigene sparoparvovec
• Brimonidine tartrate/carbachol
• Citicoline
• IBI311

• Mercaptamine

  • Cystadrops is indicated for the treatment of corneal cystine crystal deposits in adults and children from 6 months of age with cystinosis.
• Midazolam/ketamine
• OCU400

• Ranibizumab

  • Treatment of neovascular age-related macular degeneration (AMD).
• Revakinagene Taroretcel
• Ripasudil
• Sonpiretigene Isteparvovec

• Tarcocimab tedromer

  • Macular edema due to retinal vein occlusion (RVO).
• Timbetasine acetaat
• Tinlarebant

Headache

• Atogepant

  • Acute Migraine in adults

• Eptinezumab

  • Eptinezumab monotherapy for preventive treatment of Chronic migraine in children over 6 years of age and adolescents with or without aura.

• Fremanezumab

  • Fremanezumab monotherapy for preventive treatment of Chronic migraine, Episodic migraine in children over 6 years of age and adolescents.
• Meloxicam / rizatriptan

Multiple sclerosis

• Tolebrutinib

Muscular diseases other

• Baclofen / naltrexone / sorbitol
• Bidridistrogene xeboparvovec
• Condoliase
• Doxecitine / doxiribtimine
• Empasiprubart
• Incobotulinumtoxina

• Inebilizumab

  • Inebilizumab monotherapy for treatment of myasthenia gravis in adults and elderly with anti-AChR or anti-MuSK antibody.
• Kyv-101
• Mexiletine ^IND
• Nipocalimab
• Omaveloxolon
• Rozanolixizumab ^IND

• Sugammadex

  • Extension of indication to include treatment the paediatric population: sugammadex is only recommended for routine reversal of rocuronium induced blockade in paediatric patients from birth to 17 years.
• Tanruprubart
• Vatiquinone
• Zilucoplan

Neurological disorders other

• Cannabidiol

  • Children and Adolescents With Fragile X Syndrome aged 3 to < 18 years.
• Deutetrabenazine (extended release)
• Diflunisal
• Ecopipam
• Efgartigimod alfa ^IND

• Eplontersen

  • Wainzua is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with stage 1 or stage 2 polyneuropathy.
• Gefurulimab
• Nepla-tem-cel

• Pridopidine

  • Early stage Huntington Disease.

• Tiratricol

  • Emcitate is indicated for the treatment of peripheral thyrotoxicosis in patients with monocarboxylate transporter 8 (MCT8) deficiency (Allan-Herndon-Dudley Syndrome), from birth.
• Trofinetide
• Ulixacaltamide

• Vutrisiran

  • Treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.
• Zilganersen

Other psychiatry

• Soclenicant
• Troriluzol

Pain

• Adezunap
• Basimglurant
• Chloroprocaine ^IND
• Sufentanil / ketamine

Parkinson's

• Levodopa / carbidopa

  • Levodopa/carbidopa monotherapy for treatment of Parkinson's disease in adults over 30 years of age and elderly who are experiencing motor fluctuations.

SMA

• Apitegromab

• Nusinersen

  • Nusinersen monotherapy for treatment of 5q Spinal muscular atrophy in newborns, infants, toddlers, children, adolescents and adults administered at a higher dose

• Onasemnogene abeparvovec

  • Patients up to 18 years of age with 5q spinal muscular atrophy (SMA) SMA type 1, 2 and 3 or with 1-4 copies of the SMN2-gene.

Schizophrenia, psychosis, bipolar disorder

• Brexpiprazol ^IND
• Iloperidon
• Olanzapine (long-acting injectable)

Sleep disorders

• Melatonine

  • Extension of indication for the treatment of insomnia in children and adolescents aged 6-17 years with attention-deficit hyperactivity disorder (ADHD) where sleep hygiene measures have been insufficient.

• Tasimelteon

  • Extension of indication to include the treatment of nighttime sleep disturbances in adults with Smith Magenis Syndrome (SMS).