Date: 2018-03-27
Type of information: Granting of the Breakthrough Therapy status
Product name: AVXS-101 - adeno-associated virus serotype 9 expressing the human Survival Motor Neuron (SMN) gene
Compound: adeno-associated virus serotype 9 expressing the human Survival Motor Neuron gene
Therapeutic area: Rare diseases - Neuromuscular diseases
Action mechanism:
- gene therapy. AVXS-101 is a proprietary gene therapy candidate of a one-time treatment for SMA Type 1 and is the only clinical-stage gene therapy in development for SMA. AVXS-101 is designed to address the monogenetic root cause of SMA and prevent further muscle degeneration by addressing the defective and/or loss of the primary SMN gene. AVXS-101 also targets motor neurons providing rapid onset of effect, and crosses the blood brain barrier allowing an IV dosing route and effective targeting of both central and systemic features.
- AVXS-101 - ChariSMA™ is a recombinant AAV9 in which most of the AAV9’s naturally-occurring components have been removed and replaced with the SMN transgene and other elements such as the CB promoter and the Bovine Growth Hormone polyadenylation signal (BGH pA) added for hydrolytic enzymatic protection and mRNA stability. The viral vector will deliver the SMN transgene into the motor neurons. Protein synthesis from the SMN transgene will produce full-length, functional SMN protein.
Company: AveXis (USA - TX)
Disease: spinal muscular atrophy
Latest news:
- • On March 27, 2018, AveXis announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) awarded AVXS-101, SAKIGAKE Designation for the treatment of spinal muscular atrophy Type 1. The designation was based on data from the Phase 1 clinical trial of the company’s proprietary gene therapy.
- SAKIGAKE is intended to promote research and development in Japan for innovative new medical products that satisfy certain criteria, such as the severity of the intended indication, by providing prioritized consultation review during the early stages of development and by shortening the target review period for license applications from 12 months to as few as six months. The benefits of SAKIGAKE Designation are similar to the Breakthrough Therapy Designation in the United States and access into the PRIority MEdicines (PRIME) scheme in the EU, both of which have already been granted to AVXS-101.
- • On January 31, 2017, AveXis announced that the European Medicines Agency (EMA) has granted access into its PRIority MEdicines (PRIME) program for AVXS-101, for the treatment of spinal muscular atrophy (SMA) Type 1. The PRIME application was based on data from both preclinical evaluations and the ongoing Phase 1 clinical trial of AVXS-101 as of September 15, 2016.
- PRIME is intended to enhance support for the development of medicines – specifically those that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options – through early and proactive support by EMA to optimize the generation of robust data and development plans, and potentially expedite the assessment of the Marketing Authorization Application (MAA) so these medicines may reach patients sooner.
- • On July 20, 2016, AveXis announced the FDA has granted Breakthrough Therapy Designation for AVXS-101, the company’s lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1 in pediatric patients. The FDA has requested the company submit a Type B meeting request for a multidisciplinary, comprehensive discussion of the development program for AVXS-101. The company plans to submit the meeting request later this month.
- • On 12-13 May 2015, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for adeno-associated viral vector serotype 9 containing the human SMN gene for treatment of spinal muscular atrophy. AveXis is currently sponsoring a US-based Phase 1 open-label, single-dose, dose-escalation study, which will enroll nine infants under the age of nine months who have been diagnosed with SMA Type 1. The study is being conducted at The Research Institute at Nationwide Children’s Hospital (NCT02122952).
- • On September 30, 2014, the FDA has granted orphan drug designation for adeno-associated virus serotype 9 expressing the human Survival Motor Neuron gene for the treatment of spinal muscular atrophy.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2014-09-30
Orphan status UE: 2015-06-19
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
