AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy biotech company focused on developing gene therapy for patients with rare and life-threatening neurological genetic diseases, announced on Wednesday, July 20, 2016, that the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to AVXS-101, its proprietary gene therapy candidate for the treatment of pediatric spinal muscular atrophy (SMA) Type 1. AVXS-101 had previously been granted orphan drug designation for the treatment of all types of spinal muscular atrophy (SMA) and Fast Track designation for the treatment of SMA Type 1.
Breakthrough Therapy Designation is given to a drug intended alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. The FDA expedites the development and review of any drug given such designation.
The Breakthrough Therapy Designation granted to AVXS-101 is based on an interim analysis of data from the ongoing Phase 1 trial indicating that AVXS-101 continues to demonstrate a favorable safety profile in patients studied. AveXis has been granted exclusive rights to the spinal muscular atrophy gene therapy program developed at The Research Institute at Nationwide Children’s Hospital and The Ohio State University in Columbus, Ohio.
“We are encouraged to have received Breakthrough Therapy Designation for AVXS-101, and look forward to collaborating with the FDA to determine next steps in the development pathway for AVXS-101,” said Sean P. Nolan, president and chief executive officer of AveXis. “By this action the FDA recognizes the high unmet need for effective treatment options for patients suffering from SMA.”
AveXis has been asked by the FDA to submit a request for a Type B meeting. Type B meetings are scheduled to occur within 60 days of FDA receipt of the written meeting request and include pre-investigational new drug application meetings, certain end-of-phase 1 meetings, end-of-phase 2 and pre-phase 3 meetings, and pre-new drug application/biologics license application meetings. The company plans to submit the meeting request later this month.
SMA is a muscular disease transmitted genetically when both parents carry a copy of the recessive SMA gene. In such unions, one out of four children develops SMA. Two-and-one-half percent of the U.S. population carry the recessive SMA gene, leading to an incidence of SMA that is approximately one in 10,000 live births. The earlier the condition occurs, the more dangerous it is. Type 1 is the most severe, affecting children while still in the womb or shortly after birth. It is the leading genetic cause of infant mortality, and a child diagnosed with SMA Type 1 has a life expectancy of less than two years. Sixty percent (60%) of SMA patients have Type 1, with observation of disease symptoms within six months of birth. Type 4 is the least severe, affecting adults. At present, no FDA or European Medicines Agency (EMA) approved treatment for SMA currently exists.
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 SMA 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.
AveXis is a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases. The company’s initial proprietary gene therapy candidate, AVXS-101, is in an ongoing Phase 1 clinical trial for the treatment of SMA Type 1.
For additional information, please visit www.avexis.com
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