According to AveXis president David Lennon, the company, which was acquired by Novartis earlier this year, hopes to become the second biotechnology with a marketed gene therapy in the US, reported Investor's Business Daily.
Also in 2019, Lennon expects the gene therapy for spinal muscular atrophy, dubbed Zolgensma, to gain approvals in Europe and Japan.
The news source said that if successful, AveXis would follow Spark Therapeutics, which gained US approval late last year for a gene therapy that treats a rare eye disorder.
When asked what he expects for gene therapy in 2019, Lennon said "I think what you'll see in gene therapy is you're getting approvals like ours or (those that are) seeking approval. I think you'll also see the shakeout of a number of companies."
He noted "there are actually over 280 programs in cell and gene therapy, not all of those will work," adding that "a lot of that comes down to the ability of companies to move the lab-based processes to commercial and manufacturing processes, and that takes a level of investment that is not insignificant."
In terms of plans for commercializing Zolgensma, Lennon said "we have a commercial team on the ground in the US that is already getting prepared for that launch," adding the company is "in conversations with payers and specialty pharmacy and (pharmacy benefit managers) in the US to get prepared to introduce this new therapy to market. We're doing the same for Europe and Japan."
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