There is big money in orphan drugs.
Today, biotech company Ionis Pharmaceuticals announced they earned an additional $40 Million from Biogen following the announcement that Spinraza (nusinsersen) was approved in Japan for treating infantile-onset spinal muscular atrophy (SMA).
The drug was approved
in the United States by the U.S. Food and Drug Administration (FDA) on December 23, 2016 and in Europe by the European Medicines Agency (EMA) on June 1, 2017.
SMA is a genetic condition that leads to a deficiency in the spinal motor neuron (SMN) protein due to mutations of the survival motor neuron 1 (SMN1
Generally, the muscles most affected in SMA patients are those near the shoulders, hips, thighs and upper back. Muscles used for breathing and swallowing may also be affected. The life expectancy for those born with early-onset SMA is less than 2 years without treatment.
Nusinersen is an antisense oligonucleotide designed to alter the splicing of pre-mRNA from the SMN2
gene in order to increase production of fully functional SMN protein.
In August of 2016, Biogen licensed the global rights to develop, manufacture and commercialize Spinraza from Ionis Pharmaceuticals. Part of the agreement between the 2 companies is that Biogen will pay Ionis certain fees following each milestone reached.
To date, Ionis has earned over $435 million from Biogen related to Spinraza.
Stanley T. Crooke, chairman and chief executive officer of Ionis Pharmaceuticals said, "Spinraza is now approved in the U.S., EU, Japan, Canada and Brazil, which brings us closer to achieving our shared goal with Biogen that all patients with SMA will have access to this life-changing medicine."
B. Lynne Parshall, chief operating officer at Ionis Pharmaceuticals added, "Biogen is working to expand access to Spinraza, with marketing authorization applications currently under review in Switzerland, Israel, South Korea and Australia, and additional filings planned.
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