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Biogen, Ionis win FDA OK for spinal muscular atrophy drug Spinraza

michel-vounatsosBiogen $BIIB and Ionis $IONS got an early Christmas present Friday evening as the FDA announced that it had provided an accelerated approval for Spinraza (nusinersen), their new drug — and the first — for rare cases of spinal muscular atrophy.

Peak sales estimates hover around $2 billion, with some of the new estimates coming in over $3 billion by 2026.

“Our previous forecast included sales of $75mm in 2017 growing to $2.8bn by 2026,” noted Leerink’s Geoffrey Porges in early November, with the arrival of more Phase III data. “We now assume a launch in Q1 2017 from Q2 2017 previously and our current forecast is for sales of $92mm in 2017 increasing to $1.56bn by 2020 and then $3.2bn by 2026.”

Biogen says that it will be ready to ship the drug in a week.

Shares of Ionis shot up 10% and Biogen saw its stock climb 2% on the news.

The approval comes a little less than two months after researchers posted a fresh round of promising data on the drug. Using the Hammersmith Functional Motor Scale Expanded report card for patients’ motor functions, the drug arm witnessed a mean 4.0 improvement on scores while the placebo arm dropped by 1.9 points, a 5.9 point overall spread in the drug’s favor.

Ionis CEO Stanley Crooke

Ionis CEO Stanley Crooke

Both companies badly needed this win after a year of upheavals. The approval will help set the stage for Biogen’s new CEO, Michel Vounatsos, to take the helm. Ionis, meanwhile, can boast of a year-end victory after setbacks in the clinic. And analysts were impressed by the broad label applied by the FDA.

“Labeling is not restricted to any specific SMA subtype, indicating that it will likely be broadly prescribed for type 1, 2, and 3 patients and while the epidemiology in SMA is not entirely definitive, a label making the drug available to all patient types represents significant expansion potential to the initial market opportunity (though given the unmet need in SMA, this positive development is not entirely unsurprising),” notes Jefferies Brian Abrahams.

The approval also comes with a rare disease priority voucher, which can be used to accelerate review of another program, or sold on the open market.

“There has been a long-standing need for a treatment for spinal muscular atrophy, the most common genetic cause of death in infants, and a disease that can affect people at any stage of life,” said Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “As shown by our suggestion to the sponsor to analyze the results of the study earlier than planned, the FDA is committed to assisting with the development and approval of safe and effective drugs for rare diseases and we worked hard to review this application quickly; we could not be more pleased to have the first approved treatment for this debilitating disease.”


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