Extended indication Treatment of paediatric patients diagnosed with spinal muscular atrophy Type 1.
Therapeutic value No judgement
Registration phase Clinical trials

Product

Active substance Adeno-associated viral vector serotype 9 containing the human SMN gene (AVXS-101)
Domain Neurological disorders
Main indication Muscular diseases
Extended indication Treatment of paediatric patients diagnosed with spinal muscular atrophy Type 1.
Manufacturer AveXis
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional remarks Non-replicating recombinant AAV9 containing the complimentary deoxyribonucleic acid (cDNA) of the human SMN gene under the control of the cytomegalovirus (CMV) enhancer/chicken-β-actin-hybrid promoter (CB). The AAV inverted terminal repeat (ITR) has been modified to promote intramolecular annealing of the transgene, thus forming a double-stranded transgene ready for transcription.

Registration

Registration route Centralised (EMA)
Type of trajectory Accelerated assessment
Particularity New medicine with Priority Medicines (PRIME)
Submission date 2020
Expected Registration 2020
Orphan drug Yes
Registration phase Clinical trials
Additional remarks PRIME granted. AveXis: "AVXS-101, has been granted Orphan Drug Designation for the treatment of all types of spinal muscular atrophy (SMA) and Breakthrough Therapy Designation, as well as Fast Track Designation, for the treatment of SMA Type 1—one of the most life-threatening neurological genetic disorders."

Therapeutic value

Current treatment options Nusinersen (sluis-product).
Therapeutic value No judgement
Substantiation Ondanks de hele gunstige effecten van dit medicijn op het bewegen en de motoriek, zijn op dit moment de lange termijn effecten en de duurzaamheid van het effect nog niet bekend.
References NCT03306277; smaonderzoek.nl/2017/11/09/resultaten-avxs-101-fase-1/
Additional remarks Eenmalige toediening

Expected patient volume per year

Patient volume

32 - 104

Market share is generally not included unless otherwise stated.

References Vergaderstuk Adviescommissie Pakket 26 januari 2018, beoordeling nusinersen (Spinraza®) bij de behandeling van SMA.
Additional remarks In de budget impact analyse van nusinersen wordt het volume van patiënten met SMA type 1 bij therapeutische meerwaarde voor 2020 ingeschat op 32. Bij bredere indicatiestelling die alle SMA types omvat kan dit aantal oplopen tot 104.

Expected cost per patient per year

Additional remarks Het enige geneesmiddel voor SMA type 1 betreft nusinersen, dit wordt echter nog niet vergoed vanuit het basispakket (sluis). De kosten van dit middel worden geschat tussen de €210.000-270.000 p.p.p.j.. De verwachting is dat AVXS-101 duurder zal zijn gezien dit gaat om een gentherapie met eenmalige toediening.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension Yes
Indication extensions SMA Type 2 (intrathecal treatment).
References AveXis.com (Phase 1 Trial of AVXS-101 in SMA Type 2 (STRONG))
Additional remarks Planned Trials in SMA: Pivotal Trial of AVXS-101 in SMA Type 1 in Europe (STR1VE EU), Pre-Symptomatic SMA Types 1, 2, 3 (SPRINT), Pediatric “All Comers” with SMA Types 1, 2, 3 (REACH).

Other information

There is currently no futher information available.