ZURICH (Reuters) - Novartis is confident it has adequate production capacity for its Zolgensma gene therapy should regulators this month approve the drug for multiple forms of the genetic disease spinal muscular atrophy (SMA), the Swiss drugmaker said on Wednesday.
Basel-based Novartis has several clinical trials ongoing, including against SMA Type 1, which often kills infants before the age of two, as well as SMA Type 2, a slower-moving but still disabling form of the inherited disease where victims lack functioning copies of a gene needed for motor neuron development.
The U.S. Food and Drug Administration (FDA) is expected to make a decision on the gene therapy by May 31.
"Ultimately, they will have to weigh in on exactly where we land on the label," Novartis's David Lennon, who heads the Zolgensma program, told analysts on a call. "We remain very confident around our ability to supply the market under a range of scenarios for what the FDA might grant us in terms of approval."
Lennon also said a media report this week suggesting Zolgensma would be priced at $2 million per patient was "complete speculation." Novartis has said it is considering a range between $1.5 million and $5 million, a price tag that would put Zolgensma among the most-expensive treatments and which has grabbed insurers', doctors' and patient groups' attention.
Zolgensma, which Novartis bought with its $8.7 billion acquisition of U.S.-based AveXis last year, is a key piece of Chief Executive Vas Narasimhan's push into treating rare diseases with innovative medicines. SMA affects about one in every 10,000 live births, amounting to up to 500 new cases annually in the United States, 600 in Europe and 50 in Japan.
(Reporting by John Miller; editing by Brenna Hughes Neghaiwi)
