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.
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