Onasemnogene abeparvovec


New Medicines

Zolgensma (US) · Spinal muscular atrophy, type 1


Zolgensma (US)
Advanced therapy medicinal product (ATMP)

Development and Regulatory status

Pre-registration (Filed)
Pre-registration (Filed)
Pre-registration (Filed)

Mar 19: A new market research report identifies onasemnogene abeparvovec as one of seven new drugs it believes will hit sales of $1 billion or more, the so-called “blockbuster” mark, by 2023 [11].

Dec 18: FDA grants priority review for onasemnogene abeparvovec, which means it could be approved in May 2019.[9]

Nov 18: marketing authorisation applications have been made in the EU, the US, and Japan, based on the results of the PI trial and the ongoing PIII STR1VE trial. The company expects regulatory approvals in the first half of 2019 and launch mid-2019 [8].

Sep 18: Filings for IV formulation now planned for 2018 [7].

Also has breakthrough paediatric therapy and fast track designations in the US [5].

May 18: AveXis has been acquired by Novartis who have announced plans to submit regulatory application in the EU in 2019. A new manufacturing plant is to be built in North Carolina in the US [4,5].

Sep 17: pivotal PIII US trial initiated (NCT03306277) [2].

Oct 14: orphan status was granted in the US [2].

Jan 17: AVXS 101 granted PRIority MEdicines (PRIME) status in EU. Also has orphan drug status in EU [2].


A self-complementary adeno-associated virus, serotype 9 (scAAV9) carrying the cDNA of the human survival motor neuron (SMN) gene under the control of cytomegalovirus (CMV) enhanced chicken beta-actin hybrid promoter
The estimated incidence is between 1 in 6,000 and 1 in 10,000 live births and the carrier frequency is between 1 in 40 and 1 in 60. SMA type II is the most common form; SMA type 1 (acute infantile) is the most acute form with onset before six months and median survival of 7 months, 95% die before 18 months [1].
Spinal muscular atrophy, type 1

Further information

To be confirmed

Evidence based evaluations