FDA Accepts BLA & Gives Priority Review to Novartis' AVXS-101

An image of a stock chart
Credit: Shutterstock photo

Novartis AG NVS announced that the FDA has accepted the company's Biologics License Application (BLA) for AVXS-101, now known as Zolgensma (onasemnogene abeparvovec-xxxx).The FDA also granted a Priority Review to Zolgensma and regulatory action is expected in May 2019.

Priority Review designation from the FDA is generally granted to drugs with potential to provide significant improvements in the safety and effectiveness of the treatment along with prevention or diagnosis of a serious disease.

Zolgensma, an investigational gene replacement therapy, is being developed for the treatment of spinal muscular atrophy (SMA), a neurodegenerative disease accounting for the highest genetic cause of death in infants.

The filing was supported by data from the START study, which demonstrated a dramatic increase in survival and transformative improvement in achieving developmental milestones compared to the natural history of SMA Type 1.

Notably, both the FDA and the European Medicines Agency (EMA) have granted Zolgensmaan Orphan Drug designation for the treatment of SMA. The FDA has also granted Zolgensmaa fast track status for the treatment of SMA Type 1. The regulatory agency has also granted a Breakthrough Therapy designation to AVXS-101 for addressing SMA Type 1 in pediatric patients.

The company also secured PRIME and Sakigake designations for the candidate in Europe and Japan, respectively. In Europe, a decision on the Marketing Authorization Application (MAA) is expected in mid-2019.

If approved, Zolgensmawould be the first-ever one-time gene replacement therapy for SMA in the United States.

We remind investors that Zolgensma was added to Novartis' portfolio when the company acquired U.S.-based clinical-stage gene therapy company, AveXis, Inc. in April 2018.

Significantly, an approved product for the cure of SMA in the United States is Biogen's BIIB , Spinraza (nusinersen), the first and the only treatment to get an FDA nod in the country for this indication.

Other companies like Axovant Sciences AXON and uniQure N.V. QURE have been steadily developing their pipeline candidates using gene therapy.

The company's shares have gained 12.9% in the year so far, compared with the industry 's growth of 13.8%.

Further, Novartis also announced the initiation of phase III studies on ligelizumab (QGE031) - a high-affinity monoclonal anti-IgE antibody - in chronic spontaneous urticaria (CSU) patients whose symptoms are inadequately controlled by H1-antihistamines. The phase III studies, PEARL1 and PEARL 2 are planned to include more than 2,000 CSU patients. The studies will establish efficacy and safety of ligelizumab in adolescent and adult patients aged 12 years or above with CSU who remain symptomatic despite the use of H1-antihistamines.

Results from the phase IIb study showed that ligelizumab met the primary endpoint by demonstrating a clear dose-response relationship and improvements over Xolair (omalizumab) in CSU patients.

Novartis AG Price

Novartis AG Price | Novartis AG Quote

Zacks Rank

Novartis carries a Zacks Rank #3 (Hold). You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.

Today's Stocks from Zacks' Hottest Strategies

It's hard to believe, even for us at Zacks. But while the market gained +21.9% in 2017, our top stock-picking screens have returned +115.0%, +109.3%, +104.9%, +98.6%, and +67.1%.

And this outperformance has not just been a recent phenomenon. Over the years it has been remarkably consistent. From 2000 - 2017, the composite yearly average gain for these strategies has beaten the market more than 19X over. Maybe even more remarkable is the fact that we're willing to share their latest stocks with you without cost or obligation.

See Them Free>>

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report

Novartis AG (NVS): Free Stock Analysis Report

Biogen Inc. (BIIB): Free Stock Analysis Report

Axovant Sciences Ltd. (AXON): Free Stock Analysis Report

uniQure N.V. (QURE): Free Stock Analysis Report

To read this article on click here.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

In This Story


Other Topics


Latest Markets Videos


    Zacks is the leading investment research firm focusing on stock research, analysis and recommendations. In 1978, our founder discovered the power of earnings estimate revisions to enable profitable investment decisions. Today, that discovery is still the heart of the Zacks Rank. A wealth of resources for individual investors is available at

    Learn More