Drug Profile

AVXS 201

Alternative Names: AVXS-201; OAV-201; scAAV9.MeCP2

Latest Information Update: 28 Jan 2022

Price :

$50 ^*

Note:

Adis is an information provider. We do not sell or distribute actual drugs.
Final gross price and currency may vary according to local VAT and billing address.
Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase.
A link to download a PDF version of the drug profile will be included in your email receipt.

At a glance

Originator AveXis
Developer Novartis Gene Therapies
Class Gene therapies
Mechanism of Action Gene transference; Methyl-CpG-binding protein 2 gene replacements

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Rett syndrome
New Molecular Entity No

Highest Development Phases

No development reported Rett syndrome

Most Recent Events

28 Jan 2022 No recent reports of development identified for phase-I development in Rett syndrome in USA (Intrathecal, Injection)
02 Sep 2020 AveXis Inc is now called Novartis Gene Therapies
22 Jul 2020 Novartis announces intention to submit regulatory application for Rett syndrome in or after 2025 (Novartis pipeline, February 2021)

You need to be a logged in or subscribed to view this content

If your organization or you do not have a subscription, try one of the following:

Contacting your organization’s admin about adding this content to your AdisInsight subscription
Buying a PDF version of any individual profile
Request a free trial (opens in a new window)

If your organization has a subscription, there are several access options, even while working remotely:

Working within your organization’s network
Login with username/password or try to access via your institution
Persisted access using your organization’s identifier stored in your user browser for 90 days

For assistance, contact us at asktheexpert.adisinsight@springer.com